Category Archives: Cell Biology

Cell Biology

Groundbreaking Treatment for Severe COVID-19 Using Stem Cells It’s Like Smart Bomb Technology in the Lung – SciTechDaily

Umbilical cord-derived mesenchymal stem cells naturally migrate directly to the lung where they begin repair to COVID-19 damage. Credit: Dr. Camillo Ricordi

Study looked at treating severe COVID-19 with umbilical-cord derived mesenchymal stem cells.

University of Miami Miller School of Medicine researchers led a unique and groundbreaking randomized controlled trial showing umbilical cord derived mesenchymal stem cell infusions safely reduce risk of death and quicken time to recovery for the severest COVID-19 patients, according to results published inSTEM CELLS Translational Medicinein January 2021.

The studys senior author, Camillo Ricordi, M.D., director of the Diabetes Research Institute (DRI) and Cell Transplant Center at the University of Miami Miller School of Medicine, said treating COVID-19 with mesenchymal stem cells makes sense.

The paper describes findings from 24 patients hospitalized at University of Miami Tower or Jackson Memorial Hospital with COVID-19 who developed severe acute respiratory distress syndrome. Each received two infusions given days apart of either mesenchymal stem cells or placebo.

It was a double-blind study. Doctors and patients didnt know what was infused, Dr. Ricordi said. Two infusions of 100 million stem cells were delivered within three days, for a total of 200 million cells in each subject in the treatment group.

Researchers found the treatment was safe, with no infusion-related serious adverse events.

Camillo Ricordi, M.D., director of the Diabetes Research Institute (DRI) and Cell Transplant Center at the University of Miami Miller School of Medicine. Credit: University of Miami Health System

Patient survival at one month was 91% in the stem cell treated group versus 42% in the control group. Among patients younger than 85 years old, 100% of those treated with mesenchymal stem cells survived at one month.

Dr. Ricordi and colleagues also found time to recovery was faster among those in the treatment arm. More than half of patients treated with mesenchymal stem cell infusions recovered and went home from the hospital within two weeks after the last treatment. More than 80% of the treatment group recovered by day 30, versus less than 37% in the control group.

The umbilical cord contains progenitor stem cells, or mesenchymal stem cells, that can be expanded and provide therapeutic doses for over 10,000 patients from a single umbilical cord. Its a unique resource of cells that are under investigation for their possible use in cell therapy applications, anytime you have to modulate immune response or inflammatory response, he said. Weve been studying them with our collaborators in China for more than 10 years in Type 1 Diabetes, and there are currently over 260 clinical studies listed in clinicaltrials.gov for treatment of other autoimmune diseases.

Mesenchymal cells not only help correct immune and inflammatory responses that go awry, they also have antimicrobial activity and have been shown to promote tissue regeneration.

Our results confirm the powerful anti-inflammatory, immunomodulatory effect of UC-MSC. These cells have clearly inhibited the cytokine storm, a hallmark of severe COVID-19, said Giacomo Lanzoni, Ph.D, lead author of the paper and assistant research professor at the Diabetes Research Institute. The results are critically important not only for COVID-19 but also for other diseases characterized by aberrant and hyperinflammatory immune responses, such as autoimmune Type 1 Diabetes.

When given intravenously, mesenchymal stem cells migrate naturally to the lungs. Thats where therapy is needed in COVID-19 patients with acute respiratory distress syndrome, a dangerous complication associated with severe inflammation and fluid buildup in the lungs.

It seemed to me that these stem cells could be an ideal treatment option for severe COVID-19, said Dr. Ricordi, Stacy Joy Goodman Professor of Surgery, Distinguished Professor of Medicine, and professor of biomedical engineering, microbiology and immunology. It requires only an intravenous (IV) infusion, like a blood transfusion. Its like smart bomb technology in the lung to restore normal immune response and reverse life-threatening complications.

When the pandemic emerged, Dr. Ricordi asked collaborators in China if they had studied mesenchymal stem cell treatment in COVID-19 patients. In fact, they and Israeli researchers reported great success treating COVID-19 patients with the stem cells, in many cases with 100% of treated patients surviving and recovering faster than those without stem cell treatment.

But there was widespread skepticism about these initial results, because none of the studies had been randomized, where patients randomly received treatment or a control solution (placebo), to compare results in similar groups of patients.

We approached the FDA and they approved our proposed randomized controlled trial in one week, and we started as quickly as possible, Dr. Ricordi said.

Dr. Ricordi worked with several key collaborators at the Miller School, the University of Miami Health System, Jackson Health System, and collaborated with others in the U.S. and internationally, including Arnold I. Caplan, Ph.D., of Case Western Reserve University, who first described mesenchymal stem cells.

The next step is to study use of the stem cells in COVID-19 patients who have not yet become severely ill but are at risk of having to be intubated, to determine if the infusions prevent disease progression.

The findings have implications for studies in other diseases, too, according to Dr. Ricordi.

Hyper-immune and hyper-inflammatory responses in autoimmune diseases might share a common thread with why some COVID-19 patients transition to severe forms of the disease and others dont.

Autoimmunity is a big challenge for healthcare, as is COVID-19. Autoimmunity affects 20% of the American population and includes over 100 disease conditions, of which Type 1 Diabetes can be considered just the tip of the iceberg. What we are learning is that there may be a common thread and risk factors that can predispose to both an autoimmune disease or to a severe reaction following viral infections, such as SARS-CoV-2, he said.

The DRI Cell Transplant Center is planning to create a large repository of mesenchymal stem cells that are ready to use and can be distributed to hospitals and centers in North America, he said.

These could be used not only for COVID-19 but also for clinical trials to treat autoimmune diseases, like Type 1 Diabetes, Dr. Ricordi said. If we could infuse these cells at the onset of Type 1 Diabetes, we might be able to block progression of autoimmunity in newly diagnosed subjects, and progression of complications in patients affected by the disease long-term. We are planning such a trial specifically for diabetes nephropathy, a kidney disease that is one of the major causes of dialysis and kidney transplantation. We are also planning to do a study on umbilical cord mesenchymal stem cell transplantation in combination with pancreatic islets to see if you can modulate the immune response to an islet transplant locally.

Funding by The Cure Alliance made launching the initial trial possible, while a $3 million grant from North Americas Building Trades Unions (NABTU) allowed Dr. Ricordi and colleagues to complete the clinical trial and expand research with mesenchymal stem cells.

North Americas Building Trades Unions (NABTU) has been a major supporter of the Diabetes Research Institute since 1984, when they started a campaign to fund, and build, our state-of-the-art research and treatment facility. NABTU has continued to support our work through the years, including our mesenchymal stem cell research that helped lead the way to this clinical trial, he said.

Reference: 5 January 2021, STEM CELLS Translational Medicine.

All the organizations funding the research are nonprofit entities, including the Barilla Group and Family, The Fondazione Silvio Tronchetti Provera, the Simkins Family Foundation and the Diabetes Research Institute Foundation. The National Center for Advancing Translational Sciences also provided funding.

Coauthors on the NEJM paper include: Giacomo Lanzoni, Ph.D., assistant research professor, DRI; Elina Linetsky, Ph.D., DRI director of quality assurance and regulatory affairs; Diego Correa, M.D., Ph.D., assistant professor (Research) Dept. of Orthopaedics and the DRI, adjunct assistant professor of biology at Case Western Reserve University; Shari Messinger Cayetano, Ph.D., associate professor of Public Health Sciences at the Miller School; Roger A. Alvarez, D.O., M.P.H., a pulmonologist with UHealth Pulmonary and Sleep Medicine; Antonio C Marttos, M.D., a UHealth general surgeon; Ana Alvarez Gil, DRI; Raffaella Poggioli, M.D., DRI; Phillip Ruiz, M.D., Ph.D., department of Surgery at the Miller School and the UHealth Anatomic Pathology department; Khemraj Hirani, M.Pharm., Ph.D., R.Ph., CCRP, CIP, RAC, M.B.A., director of regulatory affairs and quality assurance at the DRI; Crystal A. Bell, department of medicine at the Miller School; Halina Kusack, department of Medicine, Miller School; Lisa Rafkin, research assistant professor, DRI; Rodolfo Alejandro, M.D., professor of Medicine at the Miller School, co-director of the Cell Transplant Center, and director/attending physician of the Clinical Cell Transplant Program at the DRI; David Baidal, M.D., assistant professor of Medicine in the division of Endocrinology, Diabetes & Metabolism at the Miller School and member of the DRIs Clinical Islet Transplant Program; Andrew Pastewski, M.D., Jackson Health System; Kunal Gawri, Miller School and University of Miami Health System; Dimitrios Kouroupis, postdoctoral research fellow at the Miller School; Clarissa Leero, DRI; Alejandro M.A. Mantero, Ph.D., lead research analyst, department of Health Sciences at the Miller School; Xiaojing Wang, DRI; Luis Roque, DRI; Burlett Masters, DRI; Norma S. Kenyon, Ph.D., deputy director and the Martin Kleiman professor of Surgery, Microbiology and Immunology and Biomedical Engineering at the DRI; Enrique Ginzburg, M.D., chief of Surgery at University of Miami Hospital and Trauma Medical Director at Jackson South Community Hospital; Xiumin Xu, DRI; Jianming Tan, M.D., Ph.D., Fuzhou General Hospital, Fujian, China; Arnold I. Caplan, Ph.D., professor of Biology at Case Western Reserve University; and Marilyn Glassberg, M.D., division chief of Pulmonary Medicine, Critical Care and Sleep Medicine at the University of Arizona College of Medicine.

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Groundbreaking Treatment for Severe COVID-19 Using Stem Cells It's Like Smart Bomb Technology in the Lung - SciTechDaily

Cell Biology

New clues on why pregnancy may increase risk of organ transplant rejection – Newswise

Newswise A research study at the University of Chicago has found that in pregnancy, while the T cell response to a fetus becomes tolerant to allow for successful pregnancy, the part of the immune system that produces antibodies (known as the humoral response) becomes sensitized, creating memory B cells that can later contribute to the rejection of a transplanted organ.

The results help to clarify why it is that the immune system can tolerate a fetus during pregnancy, but later may be more likely to become sensitized to and reject an organ transplant. The study was published on January 4, 2021 in the Journal of Clinical Investigation.

The immune system is designed to respond to and protect against foreign invaders; it does this by recognizing molecules on foreign cells, known as antigens, and mounting an immune response that produces T cells to target and attack foreign cells directly, as well as memory B cells that produce antibodies to tag foreign cells for destruction by other blood cells.

In most cases, this system is extremely beneficial but in pregnancy, some adaptation is required to prevent the rejection of a fetus, which only shares half its genes with the mother and therefore presents foreign antigens to the mothers immune system.

This also has the paradoxical effect of increasing the risk of a rejection for a transplanted organ (or allograft) after a person has given birth, particularly if the transplanted organ such as a kidney is from the father of their children.

This new research was inspired by prior work showing that T cells become tolerized during pregnancy, meaning they dont respond to fetal antigens. This was paradoxical to the transplant field, where we consider pregnancy a sensitizing event, said co-senior author Anita Chong, PhD, a professor of surgery at UChicago. I wanted to know why it was that pregnancy resulted in sensitization to an allograft (transplanted organ) from the male partner, but enhanced tolerance to a fetus expressing the same antigens.

In the study, the investigators examined the immune response of female mice after receiving a transplanted heart from one of their offspring. By tracking both the T cell response and the humoral response, they could follow both arms of the immune response and study their effects on transplant rejection. They saw that the T cells did not react to the allograft, but the memory B cells did, producing antibodies against foreign antigens from the transplanted heart.

Our assumption was that both arms of the immune system would be sensitized to the offspring-matched transplanted organ, said Chong, But theres something about the fetus promoting T cell tolerance that is also preserved for the allograft. On the other hand, the antibodies that are produced to the fetus do not harm the fetus, but cause the rejection of the allograft.

Given the biology of pregnancy, the investigators say, these results make sense.

Pregnancy cannot evolve to completely eliminate the humoral response because its critical for a mother to be able to produce antibodies against infectious pathogens during pregnancy and breastfeeding; its the only immunity a mother can pass to their child. So, the immune system is primed to make antibodies against anything foreign during this period, including those expressed by the fetus, said Chong. As a result, the placenta has evolved ways to handle these antibodies in order to prevent fetus rejection in subsequent pregnancies.

These results are a promising start for preventing transplant rejection in people after pregnancies in the future.

There is potential for applying therapies that would eliminate memory B cells and antibodies that now make it more difficult for these women to accept a transplant, said co-senior author Maria-Luisa Alegre, MD/PhD, a professor of medicine at UChicago. This would level the playing field for women with children. We could eliminate antibodies and B cells before transplantation and eliminate the problem, while T cell responses to antigens shared by the fetus and the transplant would already be spontaneously partially suppressed.

What is not yet clear is how the sensitized humoral response overrides the T cell tolerance to reject an allograft in people after pregnancy, or how the T cell tolerance might be induced in non-mothers in order to prevent rejection in other populations.

As part of their ongoing collaboration, Chong and Alegre hope to continue working on this puzzle. One aspect of future research is to see if we can exploit this ability of pregnancy to tolerize T cells to have better acceptance not only in people who have been pregnant, but in everybody, said Alegre. Outside of pregnancy, people can get sensitized prior to transplantation in different ways, from disease or environmental antigens, and it can be difficult to protect the transplant from cross-reactive memory T cells. Now were looking at how pregnancy can tolerize these memory T cells that are otherwise difficult to immunosuppress with current drugs.

The study, Pregnancy-induced humoral sensitization overrides T cell tolerance to fetus-matched allografts in mice, was supported by NIH/NIAID grants R01AI142747 and P01AI097113. Additional authors include Ashley N. Suah, Dong-Kha V. Tran, Stella H.W. Khiew, Michael S. Andrade, Jared M. Pollard, Dharmendra Jain, James S. Young, and Dengping Yin of UChicago; and Geetha Chalasani of the University of Pittsburgh.

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About the University of Chicago Medicine & Biological Sciences

The University of Chicago Medicine, with a history dating back to 1927, is one of the nations leading academic health systems. It unites the missions of the University of Chicago Medical Center, Pritzker School of Medicine and the Biological Sciences Division. Twelve Nobel Prize winners in physiology or medicine have been affiliated with the University of Chicago Medicine. Its main Hyde Park campus is home to the Center for Care and Discovery, Bernard Mitchell Hospital, Comer Childrens Hospital and the Duchossois Center for Advanced Medicine. It also has ambulatory facilities in Orland Park, South Loop and River East as well as affiliations and partnerships that create a regional network of care. UChicago Medicine offers a full range of specialty-care services for adults and children through more than 40 institutes and centers including an NCI-designated Comprehensive Cancer Center. Together with Harvey-based Ingalls Memorial, UChicago Medicine has 1,296 licensed beds, nearly 1,300 attending physicians, over 2,800 nurses and about 970 residents and fellows.

Visit UChicago Medicines health and science news blog at http://www.uchicagomedicine.org/forefront.

Twitter @UChicagoMed Facebook.com/UChicagoMed

Facebook.com/UChicagoMedComer

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New clues on why pregnancy may increase risk of organ transplant rejection - Newswise

Cell Biology

This Was the Busiest Year for IPOs Since the Dot-Com Bubble. Expect Another Busy Year in 2021. – Barron’s

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In 2020, a soaring IPO market not only rebounded from the Covid-19 pandemic that nearly killed it, but also posted its busiest year since 1999. Initial public offerings arent taking much of a holiday breather, with some deals expected to launch the week of Jan. 4

More than twice as many companies went public this year compared with 2019, while valuations jumped 168%. According to Dealogic, 456 U.S. IPOs raised $167.4 billion as of Dec. 24, compared to $62.5 billion collected by 211 companies for the same period in 2019.

Thats the most new issues produced by the IPO market in 20 years. In 1999, a rousing time for new issues when hundreds of companies sought to tap the public equities markets, 547 offerings collected $107.9 billion, Dealogic said.

The IPO door opened and a flood of activity went through. The outperformance of early offerings in the aftermarket created a huge degree of momentum for others to follow, said Jim Cooney, head of Americas equity capital markets at Bank of America.

The increase in IPOs this year was due to a surging U.S. stock market that powered through Covid-19 volatility and jitters around the U.S. presidential election. Public markets rewarded growth over profitability in 2020, Christina Roupas, a partner and co-chair of capital markets at law firm Winston & Strawn, said. High growth, net-loss companies and other start-ups that never would have been viable IPO candidates in prior years, all of a sudden had investors willing to throw money at them that perhaps would only have been available previously in the private markets and at less desirable valuations, she said.

By far the biggest story of this years IPO market was the success of special purpose acquisitions, or SPACs. There were 248 blank check companies that went public in 2020more than half the number of all IPOs this yearraising $82.3 billion. Thats nearly 50% of the $167.4 billion raised this year by the IPO market.

SPACs also delivered the years biggest offering: Pershing Square Tontine Holdings (ticker: PSTH), a $4 billion so-called blank-check company headed by Bill Ackman, the founder of Pershing Square Capital Management. Investors continue to provide significant capital to a diverse group of sponsors given [that] these offerings are working incredibly well, Cooney said of SPACs. With few exceptions, the majority of clients should be evaluating this opportunity.

The technology sector was quiet during the first half of 2020, but came roaring out of the gate during the last six months of the year. There were 65 tech IPOs this year, valued at $38.7 billion, Dealogic said. Tech also delivered four of the five biggest IPOs this year: Snowflake (SNOW), which raised $3.86 billion; Airbnbs (ABNB) $3.83 billion offering; DoorDashs (DASH) $3.37 billion deal; and Lufax Holding (LU), which raised $2.69 billion.

The other big story in the IPO market? Health care. The sector produced 102 IPOs this year, valued at $24.8 billion, the second most by number and third biggest by value. Much of health cares success was due to the biotech industry, which saw a surge in deals. Three-fourths of health-care IPOs were biotechs, raising $20.1 billion, Dealogic said. Investors, and the public, realized the importance of biotechnology and life science research as companies raced to develop a cure for Covid-19 this year, executives said. Its the golden age of drug discovery and thats been recognized broadly by the investment community, said BofAs Cooney.

Covid-19 buzz helped some biotechs and life sciences surge, delivering three of the top five best performing offerings this year. CureVac (CVAC), a German biotech that develops vaccines for infectious disease, soared nearly 250% in August. Berkeley Lights (BLI), a digital cell biology company, rocketed nearly 198% in July, while Seer (SEER), which develops tools to analyze proteomics, jumped 197.16% earlier this month.

Anything that is going to help solve the current health crisis, anything health-related is in high demand right now, said Chris Malik, a managing director at KeyBanc Capital Markets.

Next year, expect technology and health care IPOs to continue dominating the market and SPACs to remain aggressive, executives said. Several well-known companies are anticipated to go public in 2021 including Marqeta, Bumble, Roblox, Robinhood Markets, Affirm Holdings, Coinbase, Poshmark and Oscar Health.

The IPO market isnt taking much time off. Due to speedy virtual roadshows, management teams can now market their companies to investors through virtual meetings in three to five days, down from the eight to 10 days typically needed for in-person roadshows. We expect the strength and momentum in IPOs to continue in 2021, with numerous clients targeting IPO launches as early as the first week of the year, BofAs Cooney said.

Write to Luisa Beltran at luisa.beltran@dowjones.com

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This Was the Busiest Year for IPOs Since the Dot-Com Bubble. Expect Another Busy Year in 2021. - Barron's

Cell Biology

LyGenesis Receives FDA Clearance to Begin Phase 2a Trial of its Cell Therapy for Patients with End Stage Liver Disease, and also Closes $11 Million…

PITTSBURGH, Dec. 30, 2020 /PRNewswire/ --LyGenesis, Inc., a biotechnology company developing cell therapies that enable organ regeneration, announced today that the U.S Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application.Under the IND, LyGenesis will be conducting a Phase 2a study on the safety, tolerability, and efficacy of its first-in-class novel cell therapy for patients with end stage liver disease (ESLD).

In addition, LyGenesis just completed over $11 million in private financing of convertible notes led by Juvenescence, Ltd. and Longevity Vision Fund. Proceeds will be used to fund LyGenesis's Phase 2a clinical trial with a first patient in targeted for early 2021, as well as to push forward on their other cell therapies using lymph nodes as bioreactors to regrow functioning organs, including pancreas, kidney, and thymus regeneration.

"The FDA clearance for our IND and the start of our Phase 2a study in patients with ESLD is a testimony to our robust preclinical research program, the unmet need in advanced liver disease, and our novel approach to organ regeneration. Moreover, the lack of genetic manipulation, ease of administration, and low cost of goods for our cell therapy forms the foundation for a promising and scalable first commercial product," said Michael Hufford, PhD, Co-Founder and CEO of LyGenesis.

"LyGenesis's progress has been simply extraordinary. They have recently published 4 peer-reviewed papers on their regenerative medicine technology, filed and received clearance for their first IND, identified a primary site for their Phase 2a clinical trial, and now have closed a round of financing to ensure they have the cash to run their trial and also progress additional cell therapies toward the clinic," said Jim Mellon, Co-Founder of Juvenescence, and Chair of LyGenesis's Board of Directors. Sergey Young, founder of Longevity Vision Fund, said "We are excited to support LyGenesis in its vision to tackle some of the most challenging unmet medical needs of our time with a unique organ regeneration technology. By enabling one donated organ to act as a source of therapies for dozens of patients, LyGenesis is on the cusp of disrupting the supply-demand calculus of organ donation, and this regulatory clearance from the FDA is a definitive milestone in their evolution."

About LyGenesis, Inc.LyGenesis is a biotechnology company with an organ regeneration technology platform enabling a patient's lymph nodes to be used as bioreactors to regrow functioning ectopic organs. LyGenesis's lead allogeneic cell therapy program is focused on liver regeneration for patients with end stage liver disease. Its drug development pipeline includes thymus, pancreas, and kidney regeneration. Privately held, LyGenesis is headquartered in Pittsburgh, Pennsylvania. To learn more, please visit lygenesis.com.

About Juvenescence, Ltd.Juvenescence Ltd. is a life sciences company developing therapies to modify aging increasing human health span and longevity. It was founded byJim Mellon,Dr. Gregory BaileyandDr. Declan Doogan. The Juvenescence team are highly experienced drug developers, entrepreneurs and investors with a significant history of success in the pharmaceutical and consumer health sectors. Juvenescence will create, partner with or invest in new companies with longevity-related therapeutics, by in-licensing compounds from academia and industry, or forming joint ventures to develop therapeutics for longevity. Juvenescence believes that recent advances in science have greatly improved our understanding of the biology of aging and seeks to develop therapeutics with the possibility of slowing, halting or potentially reversing elements of aging. To learn more, please visit juvlabs.com.

About Longevity Vision FundLongevity Vision Fund is a venture capital fund that invests in technologies with the potential to disrupt life sciences to help people live longer and healthier lives. The fund is focused on early diagnostics, AI in healthcare, and advanced therapeutic platforms targeting aging and age-related diseases. It was founded by Sergey Young, a venture capital investor with a mission to make longevity more accessible and affordable. Sergey is also an Innovation Board member at XPRIZE Foundation and a development sponsor of Age Reversal XPRIZE. To learn more, please visit lvf.vc.

Contact InformationMichael Hufford(858) 603-2514262126@email4pr.com

View original content:http://www.prnewswire.com/news-releases/lygenesis-receives-fda-clearance-to-begin-phase-2a-trial-of-its-cell-therapy-for-patients-with-end-stage-liver-disease-and-also-closes-11-million-convertible-debt-financing-301199042.html

SOURCE LyGenesis, Inc.

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LyGenesis Receives FDA Clearance to Begin Phase 2a Trial of its Cell Therapy for Patients with End Stage Liver Disease, and also Closes $11 Million...

Cell Biology

COVID-19 Impact on Live Cell Imaging Consumables Market Size, Share, Trends and Analysis 2020 to 2026| Carl Zeiss AG (Germany), Olympus Corporation…

Global Live Cell Imaging Consumables Market Forecast to 2026.

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Cell Biology

Global Live Cell Imaging Consumables Market Tendencies, Revenue Forecast and Interesting Opportunities from 2020 to 2026 – LionLowdown

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Global And Regional Prospects:

Some of the following top market players profile included in this report: Carl Zeiss AG (Germany), Olympus Corporation (Japan), Molecular Devices, LCC (US), Leica Microsystems (Germany), Becton, Dickinson and Company (US), Nikon Corporation (Japan), Thermo Fisher Scientific, Inc.(US), GE Healthcare (U.K.), PerkinElmer, Inc. (US), Sigma Aldrich Corporation (US)

By the product type, the market is primarily split into: Assay Kits, Reagents, Media, Others,

By the end-users/application, this report covers the following segments: Cell Biology, Stem Cells, Developmental Biology, Drug Discovery,

The report covers different geographical areas: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina, Colombia), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

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Moreover, the report contains the discussion over the market regarding the total revenue generation by the global Live Cell Imaging Consumables market at the global level, along with the manufacturing techniques and cost, demand, and supply of a generated product or offered services, capacity, consumption, and other related information. The total cost spent on manufacturing the product and analysis of its assembling procedure is also mentioned in the report. The analysis study includes market size and forecast by type and by application segment in terms of sales and revenue for the period 2015-2025.

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Global Live Cell Imaging Consumables Market Tendencies, Revenue Forecast and Interesting Opportunities from 2020 to 2026 - LionLowdown

Cell Biology

Scientists Discover a Gene to Stay Thin Protects Against Diet-Induced Obesity – SciTechDaily

By Institute of Molecular Biotechnology of the Austrian Academy of SciencesDecember 27, 2020

Why some people eat as much as they want and stay lean while others cant. In mice, genetic deletion of ALK resulted in thin animals with marked resistance to diet induced obesity while food intake was not changed. The researchers found that ALK in the hypothalamus controls energy expenditure via sympathetic control of adipose tissue lipolysis. The drawing illustrates how our genetic make-up determines body weight (gain). Credit: IMBA/IMP graphics

A noticeable impact on the waistline of many people is a side-effect of the quarantine due to the global COVID-19 outbreak. Reduced activity and lack of sports while consuming the same, or even elevated amounts of calories can quickly cause a substantial weight gain.

Strikingly, some individuals can make it through this period without gaining any weight we all know these people who can eat what they want but do not appear to gain weight.

A consortium of international researchers including scientists from IMBA, the University of British Columbia, Medical University of Vienna, and the Estonian Biobank have now taken a unique approach: thus far, the regulation of fat metabolism has mainly focused on finding genes linked to obesity. The team, however, went on a quest to discover genes linked to thinness, or the resistance to weight gain.

In order to identify candidate thinness genes, the research team conducted genome-wide association studies in an Estonian population cohort, profiling over 47,000 people. They compared thin to control individuals and were thereby able to pinpoint ALK, which codes for Anaplastic Lymphoma Kinase, as a candidate gene for thinness. ALK was mainly known due to its involvement in cancer, as it is frequently mutated in multiple cancers. However, its physiological function was largely elusive.

To test the hypothesis of ALK being involved in thinness, the researchers inactivated the ALK gene in mice. Strikingly, despite normal food intake and activity, ALK deficient mice were skinnier because of a much-reduced fat mass and strikingly protected against diet-induced obesity as opposed to littermate controls. Interestingly, when knocking down the ALK orthologues in the fruit fly Drosophila melanogaster, they also found significantly lower triglyceride fat accumulation, even when flies were fed a high-sucrose diet.

First author Michael Orthofer from the Penninger lab explains: By using a technique called indirect calorimetry, we could show that ALK deficient mice exhibit increased energy expenditure. This means that they burn more calories than normal mice and explains why they remain thin even if they eat the same amount of food. In addition to that, these animals also show improved glucose tolerance.

ALK is highest expressed in a very specific brain region called the paraventricular nucleus (PVN) of the hypothalamus. When the scientists depleted ALK in this brain area, a similar body weight reduction was observed compared to full-body ALK knockout models. The PVN is known to be involved in the regulation of energy homeostasis, both via hormonal pathways and the sympathetic nervous system, which uses norepinephrine as neurotransmitter. Indeed, levels of the neurotransmitter norepinephrine were elevated in both white and brown fat of the mutant mice, indicating that ALK deficiency increases sympathetic tone to adipose tissues. Consequently, ALK knockout mice showed increased breakdown of fat, which explains their low body adiposity and resistance to weight gain.

This strengthens the notion that ALK is indeed part of a larger brain circuit involved in energy expenditure. We are very excited about these results on the genetics of thinness and will further investigate the mechanisms of how ALK-expressing neurons are able to control weight. Our results also highlight the important therapeutic potential of ALK inhibition, says Josef Penninger, IMBA group leader and founding director, who is now director of the Life Sciences Institute of the University of British Columbia.

For more on this research, read Scientists Identify Gene Linked to Thinness.

Reference: Identification of ALK in Thinness by Michael Orthofer, Armand Valsesia, Reedik Mgi, Qiao-Ping Wang, Joanna Kaczanowska, Ivona Kozieradzki, Alexandra Leopoldi, Domagoj Cikes, Lydia M. Zopf, Evgenii O. Tretiakov, Egon Demetz, Richard Hilbe, Anna Boehm, Melita Ticevic, Margit Nukas, Alexander Jais, Katrin Spirk, Teleri Clark, Sabine Amann, Maarja Lepamets, Christoph Neumayr, Cosmas Arnold, Zhengchao Dou, Volker Kuhn, Maria Novatchkova, Shane J.F. Cronin, Uwe J.F. Tietge, Simone Mller, J. Andrew Pospisilik, Vanja Nagy, Chi-Chung Hui, Jelena Lazovic, Harald Esterbauer, Astrid Hagelkruys, Ivan Tancevski, Florian W. Kiefer, Tibor Harkany, Wulf Haubensak, G. Gregory Neely, Andres Metspalu, Jorg Hager, Nele Gheldof and Josef M. Penninger, 21 May 2020, Cell.DOI: 10.1016/j.cell.2020.04.034

IMBA Institute of Molecular Biotechnology is one of the leading biomedical research institutes in Europe focusing on cutting-edge stem cell technologies, functional genomics, and RNA biology. IMBA is located at the Vienna BioCenter, the vibrant cluster of universities, research institutes and biotech companies in Austria. IMBA is a subsidiary of the Austrian Academy of Sciences, the leading national sponsor of non-university academic research. The stem cell and organoid research at IMBA is being funded by the Austrian Federal Ministry of Science and the City of Vienna.

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Scientists Discover a Gene to Stay Thin Protects Against Diet-Induced Obesity - SciTechDaily

Cell Biology

Global Live Cell Imaging Market Proceeds To Witness Huge Upswing Over Assessment Period by 2025 – Factory Gate

The globalLive Cell Imaging Marketresearch report enlists the vital and practical information with regards to market situation. The present scenario of Live Cell Imaging market, along with its previous performance as well as future scope are covered in the report. This eases the users understanding of the market thoroughly, while also gaining knowledge about market opportunities and the dominant players Becton, Dickinson and Company (U.S.), PerkinElmer, Inc. (U.S.), Olympus Corporation (Japan), Nikon Corporation (Japan), GE Healthcare (U.K.), Thermo Fisher Scientific, Inc.(U.S.), Sigma Aldrich Corporation (U.S.), Leica Microsystems (Germany), Carl Zeiss AG (Germany), Molecular Devices, LCC (U.S.) in the Live Cell Imaging market.

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The Global Live Cell Imaging Market Research Report Details

The beginning of the report summarizes the market with the definition of the overall Live Cell Imaging market.

The following section includes Live Cell Imaging market segmentation Equipment, Consumables. Segmentation is done on the basis of application, type, end-user industries, and several such factors among others.

We have strived to include sub-segments Cell Biology, Stem Cells, Developmental Biology, Drug Discovery in segmentation section, wherever possible. Also included are details regarding the dominant segments in the worldwide Live Cell Imaging market.

The global Live Cell Imaging market has also been classified on the basis of regions. On the basis of the regional diversification, details regarding market share and size have also been obtained.

In the succeeding part, growth factors for the Live Cell Imaging market have been elucidated. This section also explains the technological advancements made to improve market size and position. Also enlisted is the information pertaining to the end-use industries for the Live Cell Imaging market.

Read Detailed Index of full Research Study at::https://www.marketresearchstore.com/report/global-live-cell-imaging-market-report-2018-industry-366843

Live Cell Imaging Market COVID-19 Impact Analysis

As the world is still dealing with COVID-19 situation, many of the countries have slowly started to revive its economic situation by starting its trade and businesses. There has been enormous loss in these few months both in terms of economy and human lives. As the WHO has already suggested that there are very less chances that the virus will completely go, hence we will have start living with it. Many of the drug companies are getting positive response of their COVID-19 vaccines, but there is still time for its availability in the global market.

There are 15 Sections to show the global Live Cell Imaging market

Sections 1, Definition, Specifications and Classification of Live Cell Imaging , Applications of Live Cell Imaging , Market Segment by Regions;Section 2, Assembling Cost Structure, Crude Material and Providers, Assembling Procedure, Industry Chain Structure;Sections 3,Technical Data and Manufacturing Plants Analysis of Live Cell Imaging , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Sections 4, Generally Market Analysis, Limit Examination (Organization Fragment), Sales Examination (Organization Portion), sales Value Investigation (Organization Section);Sections 5 and Six, Regional Market Investigation that incorporates United States, China, Europe, Japan, Korea and Taiwan, Live Cell Imaging segment Market Examination (by Sort);Sections 7 and Eight, The Live Cell Imaging Segment Market Analysis (by Application) Major Manufacturers Analysis of Live Cell Imaging ;Sections Nine, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Equipment, Consumables Market Trend by Application Cell Biology, Stem Cells, Developmental Biology, Drug Discovery;Sections 10, Regional Promoting Type Investigation, Worldwide Exchange Type Examination, Inventory network Investigation;Sections 11, The Customers Examination of global Live Cell Imaging;Sections 12, Live Cell Imaging Research Findings and Conclusion, Appendix, system and information source;Sections 13, 14 and 15, Live Cell Imaging deals channel, wholesalers, merchants, traders, Exploration Discoveries and End, appendix and data source.

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Particulars Of The Global Live Cell Imaging Market Research Report

Further part in the report enlists the restraining factors for the Live Cell Imaging market growth. The restraints are explained comprehensively and with details in order that the client can comprehend how these factors are affecting the global Live Cell Imaging market and how such factors can be tackled effectively using suitable measures.

Also, regional study and analysis of global Live Cell Imaging market focused on in the report. Here, the major regions with Live Cell Imaging market establishment have been explained thoroughly. Due to this, our clients will have clarity in understanding the booming markets as well as the potential Live Cell Imaging markets in the near future.

The concluding section relates to the conclusions and observations regarding the global Live Cell Imaging market.

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Global Live Cell Imaging Market Proceeds To Witness Huge Upswing Over Assessment Period by 2025 - Factory Gate

Cell Biology

Follow the Money: Spatial Omics, CAR-NK Cells, AI-Powered Biology – Bio-IT World

December 21, 2020 | Funding updates around the life sciences including cash for a handheld mass spec device, Series D for oncolytic immunotherapy, a new company launch in allogenic CAR-NK cells, AI-powered cell classification, a 3-D sequencing platform, and more.

$170M: Israeli Digital Health First VC Firm

OTV (formerly Olive Tree Ventures), Israels digital health first venture capital firm, today announced the closing of a fund with a total value of $170M. OTV also announced their new name and the appointment of a new Head of Asia Pacific to spearhead the funds expansion into the regions market. OTV is the only venture capital fund in Israel whose primary focus is digital health, specializing in supporting their portfolio companies reach maturity, refine execution, tackle regulatory hurdles and ensure a global imprint on validated products. Over the course of the past five years, OTV has prioritized investment in digital health companies that develop cutting-edge solutions to todays most pressing healthcare problems. OTVs portfolio includes some of the worlds highest-profile digital health leaders, including TytoCare, Lemonaid Health, Emedgene, Scopio and Donisi Health.

$116M: Handheld, Desktop Mass Spec

908 Devices, which provides mass spectrometry devices for forensic and scientific research, raised the proposed deal size for its upcoming IPO. The Boston, MA-based company now plans to raise $116 million by offering 6.3 million shares at a price range of $18 to $19. The company had previously filed to offer the same number of shares at a range of $15 to $17. At the midpoint of the revised range, 908 Devices will raise 16% more in proceeds than previously anticipated. 908 Devices provides handheld and desktop mass spectrometry devices that are used to interrogate unknown and invisible materials, providing actionable answers to directly address critical problems in life sciences research, bioprocessing, industrial biotech, forensics and adjacent markets. Since its inception, the company has sold more than 1,200 handheld and desktop devices to over 300 customers in 32 countries, including 18 of the top 20 pharmaceutical companies by 2019 revenue, as well as numerous domestic and foreign government agencies and leading academic institutions.

$91M: Series C for Digital, Decentralized Trials

Medable has announced $91 million in Series C funding to accelerate the life sciences industrys shift to digital and decentralized clinical trials. The round was led by Sapphire Ventures, with follow-on investment from existing investors GSR Ventures, PPD, and Streamlined Ventures. The funding brings Medables total capital raised to more than $136 million. Medables flexible and modular software platform enables clinical leaders to shift from clinic-centric to patient-centric research strategies. The platform provides a unified experience for patients and clinicians, enabling recruitment, remote screening, electronic consent, clinical outcomes assessment (eCOA), eSource, telemedicine, and connected devices. Medable has seen rapid eCOA adoption, driving the field forward with enhancements including connected devices and telemedicine. The COVID-19 pandemic has driven demand for remote clinical trial technologies and Medable is enabling complex research protocols to be conducted remotely through its platform. By minimizing the need for in-person site visits, Medable customers have achieved unprecedented results including 3X faster enrollment and over 90% retention rates.

$58M: Series B for Healthcare Ecosystem Platform

H1, a global platform for the healthcare ecosystem, announced today that it has closed a $58 million Series B round of funding. The round was co-led by IVP and Menlo Ventures, which led the Series A round in April 2020. Transformation Capital, Lux Capital, Lead Edge Capital, Novartis dRx and YC also participated. H1 has created the largest healthcare platform to forge connections in the healthcare ecosystem. The H1 team has taken a unique approach to building the platform that combines AI, human powered engineering, third-party data sources, and government partnerships, to create the largest platform of healthcare professionals, currently spanning over 9 million healthcare professions around the globe.

$50M: Series C for Somatic Cancer, Wellness Platform

Congenica has announced the completion of its Series C funding round, raising $50 million. The round was co-led by Tencent and Legal & General and included other new investors Xeraya, Puhua Capital and IDO Investments. Existing investors Parkwalk, Cambridge Innovation Capital and Downing also participated. The funding is aimed at accelerating international market development and driving further expansion of Congenicas product platform into somatic cancer, wellness and through partnerships with pharmaceutical companies. Furthermore, the company will deliver capabilities including the ability to integrate with existing electronic health systems and deliver automated interpretation.

$47M: Series D for Novel Oncolytic Immunotherapies

CG Oncology has closed a $47 million Series D preferred stock financing led by new investor Kissei Pharmaceutical Co., Ltd., with participation from existing investors ORI Healthcare Fund, Camford Capital and Perseverance Capital Management. The financing will support the advancement of CG Oncologys late-stage clinical programs for its lead oncolytic immunotherapy, CG0070, including an ongoing global Phase 3 trial (BOND3) with CG0070 as a monotherapy for the treatment of BCG-unresponsive, Non-Muscle Invasive Bladder Cancer (NMIBC), and a combination Phase 2 study (CORE1) of CG0070 with KEYTRUDA (pembrolizumab) in the same indication. In addition, a Phase 1b study (CORE2) is currently ongoing with CG0070 in combination with OPDIVO (nivolumab) as a neoadjuvant immunotherapy for Muscle-Invasive Bladder Cancer (MIBC) in cisplatin-ineligible patients.

$42M: Series A for Allogeneic CAR-NK Cell Therapies

Catamaran Bio has launched with $42 million in financing. Sofinnova Partners and Lightstone Ventures co-led the Series A round that is part of the launch financing, with participation by founding investor SV Health Investors, as well as Takeda Ventures and Astellas Venture Management. Proceeds will be used to advance the companys two lead chimeric antigen receptor (CAR)-NK cell therapy programs. In addition, funding will expand the companys TAILWIND Platform, an integrated and proprietary suite of technologies for designing, genetically engineering, and manufacturing allogeneic CAR-NK cell therapies.

$34M: Series B for glycoproteomic powered ovarian cancer diagnostic

InterVenn Biosciences has raised $34M in a Series B fundraising. The latest round was led by Anzu Partners with full participation of Genoa Ventures, Amplify Partners, and True Ventures; Xeraya Capital and the Ojjeh Family joined the syndicate as well. Funds will be used to commercialize the companys High-Throughput-Glycoproteomic powered diagnostic for ovarian cancer; to service increasing partnership platform demand; and to accelerate development efforts for the immuno-oncology treatment response and colorectal cancer indications. InterVenn has demonstrated that analysis of protein glycosylation, the most common and most complex form of post-translational protein modification, is a highly effective way of discovering uniquely informative biomarkers. This breakthrough was made possible given the companys development of its AI neural network for high throughput analysis (PiP) and enabling software that powers the proprietary Vista research product for over a dozen different oncology indications.

$24M: Series A for Molecular Cartography

Resolve Biosciences has completed a $24 million Series A financing round and appointed Co-founder Jason T. Gammack as Chief Executive Officer. The Series A financing round was led by PS Capital Management and MasterMind Advisory Services and included participation from Alafi Capital, John Shoffner, and High Tech Grnderfonds. The company will use the proceeds to accelerate product development and drive industry adoption of its Molecular Cartography technology. The company's Molecular Cartography platform is a groundbreaking multi-analyte and highly multiplex spatial analysis technology that enables scientists to resolve the most daunting biological challenges in areas such as oncology, neuroscience, and infectious disease. It produces deep contextual datasets that illuminate molecular interactions at subcellular resolution, while preserving the sample tissue. The initial applications of Resolve's Molecular Cartography platform deliver the highest-resolution view of transcriptomic activity and provide the ability to interrogate hundreds of genes in a single run. Future solutions will add DNA, protein, and metabolomic data layers. Unlike current approaches, Resolve's technology provides the required sensitivity, specificity, and workflow convenience to elucidate the cell's complex transcriptional landscape.

$23M: Series A for Traumatic Brain Injury Test

BRAINBox Solutions announced the initial closing on a $23 million Series A financing to support the clinical development of the company's BRAINBox TBI (Traumatic Brain Injury) Test to aid both in the diagnosis and prognosis of mild TBI (concussion). BioVentures Investors led the financing round and was joined by the Tauber Foundation, the Virginia Tech Carilion Innovation and Seed Funds, Genoa VC, Pharmakon Holdings LLC, Astia Angels and additional qualified investors, including Kevin Love, professional basketball player and mental health advocate. BRAINBox TBI is the first test designed to assist in both the diagnosis and prognosis of concussion. The multi-marker and multi-modality test, which can be used in either point-of-care or clinical laboratory settings, includes a panel of blood biomarkers as well as advanced digital neurocognitive testing in partnership with BrainCheck, Inc. Using proprietary AI algorithms, BRAINBox TBI combines the results of the test components and patient reported outcomes to generate a single, objective score for diagnosis up to 96 hours from the time of injury and a prognosis report for likely injury-related symptoms up to three months post-event.

$23M: Series B for Digital, Computational Pathology Solutions

Proscia has secured $23 million in Series B funding led by Scale Venture Partners, with participation from Hitachi Ventures, the strategic corporate venture capital arm of Hitachi, Ltd., bringing its funding total to $35 million. The company will use the investment to accelerate its global growth and strengthen its position of leadership in transforming cancer research and diagnosis at a time when demand for modernizing pathology is higher than ever. With its Concentriq software platform, Proscia is accelerating the transformation to digital pathology, which centers around high-resolution images of tissue biopsies, as the new standard of care. Concentriq combines enterprise scalability with powerful AI applications to help laboratories, health systems, and life sciences companies unlock new insights, accelerate breakthroughs, and improve patient outcomes.

$21.5M: Series B for Software, Data Products for Biomarker Discovery

Ovation.io has raised $21.5 million in Series B funding led by SignalFire with participation from Madrona Venture Group, Borealis Ventures, StageDotO Ventures and industry veteran David Shaw. Ovations suite of software and data products make it easier for molecular diagnostic labs to bring innovative tests to the patients that need them. Developed by scientists to help laboratories accelerate adoption of molecular diagnostics, Ovation is a turn-key, cloud-based platform with configurable, out-of-the-box workflows for molecular tests and seamless integrations to support the needs of a rapidly growing lab. With this latest round of funding, Ovation will work with its network of participating labs to develop insights that can help life-science companies reduce the time and cost associated with biomarker discovery.

$20M: Series A for AI-Powered Cell Classification

Deepcell has closed its Series A round of financing with $20 million, led by Bow Capital and joined by Andreessen Horowitz, which led its $5 million seed round. The new funding will allow Deepcell to develop its microfluidics-based technology, continue building a cell morphology atlas of more than 400 million cells, and drive a hypothesis-free approach to cell classification and sorting. Spun out of Stanford University in 2017, Deepcell is using deep learning and big data to classify and isolate individual cells from a sample. The technology combines advances in AI, cell capture, and single-cell analysis to sort cells based on detailed visual features, delivering novel insights through an unprecedented view of cell biology. The Deepcell platform maintains cell viability for downstream single-cell analysis and can be used to isolate virtually any type of cell even those occurring at frequencies as low as one in a billion to offer access to rare cells and atypical cell states that will help advance precision medicine research.

$20M: Series B for Spatial Omics

Rebus Biosystems has closed a $20 million Series B financing round, led by Illumina Ventures and joined by Lifecore Partners, Ncore Ventures, Xolon Invest, CTK Investments, Ray Co., Ltd., Seegene Medical Foundation, LabGenomics Co., Ltd., and Timefolio Asset Management. At the heart of the Rebus Biosystems spatial omics solution is the companys patented Synthetic Aperture Optics (SAO) system, which provides the resolution and sensitivity of a 100X oil lens, but with the breadth and depth of a 20X air lens. Data is captured 100 times faster than with other imaging based spatial omics methods that rely on 100x lenses and z-stacking. Speed and ease of use of the system is further improved by integration with custom microfluidics and image processing.

$14.8M: Scaled Up Production of Portable PCR Diagnostic Device

QuantuMDx Group Limited, a UK-based life sciences company, is investing over 11 million to scale up production to mass manufacture its flagship diagnostic device, Q-POC and disposable test cassette. QuantuMDx accelerated development, scale-up and manufacture of Q-POCits rapid point-of-care testing systemearlier this year in response to the COVID-19 pandemic.Q-POC is a portable, PCR device offering rapid, sample-to-answer, molecular diagnostic testing at the point of care, with results in approximately 30 minutes. The Q-POC system comprises a sample collection kit, single-use test cassette and analyzer. The companys first commercial assay for Q-POC will detect SARS-CoV-2, the virus causing COVID- 19, and Flu A & B, providing a powerful rapid PCR diagnostics and surveillance tool for clinicians and public health officials.QuantuMDx has worked with British development partner Cambridge Design Partnership to undertake pilot manufacturing of Q-POCTM, and is now working with Cogent Technology, as the company scales for volume manufacturing.

$14M: Novel Treatment for Ischemic Stroke

BrainsGate has secured $14 million in a new investment round at a pre-money valuation of $147 million. New investor, BNP Joint Capital Fund, and the Impact investment and consulting firm SPERO led the round alongside existing investors, Elron, Medtronic, Agate, Pitango, and Cipio. The new investment is expected to fund BrainsGate through its pre-market approval (PMA) and enable it to achieve volume production readiness and apply for coverage from the Centers for Medicare & Medicaid Services (CMS) for its Ischemic Stroke System (ISS). BrainsGates therapy involves a less than 5 minutes procedure in which a neurostimulator implant is injected into an existing canal. The implant stimulates a nerve center that augments collateral blood flow to improve stroke patients outcomes in a 24-hour window. In May 2020, BrainsGate received marketing approval in Europe (CE marking) of its ISS product. BrainsGates PMA application was filed with the US FDA in February 2020. The company plans to initiate commercialization activities subject to the PMA being approved.

$10.6M: UK Investment Firm for Life Sciences

Intuitive Investments Group, a closed-end investment company focused on the life sciences sector, has raised 7.85 million (before expenses) in its AIM float by placing 39,250,000 new Ordinary Shares at 20p. The net proceeds of the Placing will be used by the Company to invest in fast growing and/or high potential Life Sciences businesses, based predominantly in the UK, wider Europe and the US, chosen from an identified pipeline of investment opportunities. Investments will be focused on diagnostics and healthcare, medical devices, tools and technologies and bio-therapeutics and pharmaceuticals

$7.4M: BARDA Extension For Point-of-Care Infection Diagnostics

Inflammatix has announced a contract extension of $7.4 million from the Biomedical Advanced Research and Development Authority (BARDA) to further develop its point-of-care test and system to diagnose infection by reading the immune system. The contract is part of a BARDA contract worth up to $72 million, if all options are exercised. The new funding will support continued development and commercialization of Inflammatixs sample-to-answer, point-of-care Myrna test system, which is designed to read RNA using machine learning and produce results in under 30 minutes, as well as continued development of the ViraBac EZ test (formerly known as HostDx Fever), which reads gene expression patterns in the immune system to identify whether a suspected infection is bacterial or viral, enabling physicians to quickly and accurately determine when to prescribe antibiotics. The test will use a fingerstick collection and capillary blood sample, and is designed for use in primary care, urgent care and other outpatient clinical settings.

$6.6M: Series A for Robotics, Machine Learning in Biology

Trailhead Biosystems announced its $6.6 million Series A financing. The company has developed a proprietary platform to perform systems-level interrogation of complex biological problems, ranging from the generation of industrially-scalable manufacturing conditions for specialized human cells to combinatorial drug discovery in cancer and anti-viral therapies. Trailhead robotically conducts the largest dimensioned experiments in biology, using machine learning to discover critical process parameters and combinatorial inputs that explain biological responses. Trailhead aims to rapidly develop the capability of creating specialized human cell types at high purity for regenerative medicine and therapeutic purposes at an industrial scale, addressing current industry needs for highly specialized cells used in drug discovery and modeling of human diseases. Through strategic partnerships, the cells provided by the company will be used to address multiple areas of clinical need. The company currently develops products that target type I diabetes, Parkinson's disease, Multiple Sclerosis, Alzheimer's disease, and blood disorders, among others.

$6.1M: 3-D Sequencing Platform

DNA sequencing instigator Single Technologies announced the completion of a heavily oversubscribed 5 million share issue to existing and new shareholders. Among the new investors are Jens von Bahr, Rothesay Ltd, Carl-Henric Svanberg funded Cygnus Montanus Trust, Professor Ulf Landegren and Andreas Ehn. The funds will be used to accelerate development of the companys 3-D sequencing platform. Stockholm Corporate Finance acted as financial advisor. The new funding will enable the company to finalize automating its 3-D sequencing process, make it more robust and improve quality for both Whole Genome Sequencing (WGS) and transcriptomics applications. The ambition is to open the first data sequencing production site in Stockholm by 2022.

$5M: Gates Grant for At-Home COVID-19 Test

Sherlock Biosciences has received a $5 million grant from the Bill & Melinda Gates Foundation to continue to advance INSPECTR, its instrument-free, synthetic biology-based molecular diagnostics platform. In addition to advancing the INSPECTR platform development to be as sensitive as gold-standard PCR tests, the funding will support the development of an over-the-counter disposable product, similar to an at-home pregnancy test, that can be used to detect SARS-CoV-2, the virus that causes COVID-19. Pending approval, the company says they will launch this product in mid-2021. INSPECTR, which stands for Internal Splint-Pairing Expression Cassette Translation Reaction, uses synthetic biology to enable the creation of instrument-free diagnostic tests that can be conducted at home, at room temperature. INSPECTR can be adapted to work on a simple paper strip test or to provide an electrochemical readout that can be read with a mobile phone. It can also be adapted for use in laboratory or point-of-care settings.

$4M: Prostate Health Center at Mount Sinai

Mount Sinai has received a $4 million donation from Lizzie and Jonathan Tisch to support prostate health and the Milton and Carroll Petrie Department of Urology at Mount Sinai. The prostate program will be named The Lizzie and Jonathan Tisch Center for Prostate Health. The medical services provided at the Lizzie and Jonathan Tisch Center for Prostate Center include state-of-the-art prostate cancer screening and imaging technologies, precision urology, focal therapy, targeted biopsies, robotics for prostate cancer surgery, prostate cancer fusion biopsy, and active surveillance. Additionally, prostate cancer experts such as Dr. Tewari; Avinash Reddy, MD; Sujit Nair, PhD; Robert Valenzuela; MD; Michael Palese, MD; and Steven Kaplan, MD, are available to see patients at the newly named Center.

$2.9M: NIH Grant for Human Placental Stem Cells

Human placental stem cells may have the potential to regenerate heart tissue after a heart attack, according to Mount Sinai researchers who have received a $2.9 million grant from the National Institutes of Health to study them. Their findings could lead to new therapies for repairing the heart and other organs. Hina W. Chaudhry, MD, Director of Cardiovascular Regenerative Medicine at the Icahn School of Medicine at Mount Sinai, is the Principal Investigator for this four-year award. Dr. Chaudhry and a team of investigators previously discovered that mouse placental stem cells can help the hearts of mice recover from injury that could otherwise lead to heart failure. They identified a specific type of placental stem cells, called Cdx2 cells, as the most effective in making heart cells regenerate.

$2M: Cryo Solutions for Cell, Gene Therapy

GlycoNet has secured a $2-million USD equity investment for PanTHERA CryoSolutions (PanTHERA), a Canadian biotechnology start-up. The investment came from US-based investor Casdin Capital and bioproduction tools supplier BioLife Solutions Inc. In addition to an up-front investment, subject to closing conditions, BioLife will provide an additional $2 million to support product development over the next 24 months in exchange for exclusive, worldwide marketing and distribution rights to the technology for use in cell and gene therapy applications. The core technology from PanTHERA was created out of an academic research collaboration between the University of Ottawa and the University of Alberta. During cryopreservation of biological materials, the uncontrolled growth of ice causes cell injury and death. PanTHERA's solution is to develop ice recrystallization inhibitors (IRIs) to control the growth of ice and prevent this damage from occurring, ultimately resulting in superior cellular products after thawing.

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Follow the Money: Spatial Omics, CAR-NK Cells, AI-Powered Biology - Bio-IT World

Cell Biology

Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) – GlobeNewswire

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire