Unproven ‘stem cell’ therapies for COVID-19 pose harm to public, says UB expert – UB Now: News and views for UB faculty and staff – University at…

Be wary of stem cell therapy as a preventative treatment for COVID-19, warns Laertis Ikonomou, a UB expert on stem cell and gene therapies.

While stem cell therapy, such as bone marrow transplantation, may be used to treat a limited number of diseases and conditions, there are currently no clinically tested or government-approved cell therapies available for the treatment or prevention of COVID-19, says Ikonomou, associate professor of oral biology in the School of Dental Medicine.

He urges the public to exercise caution as the nation experiences a rise in businesses offering direct-to-consumer, unproven and unsafe stem cell therapies that promise to prevent COVID-19 by strengthening the immune system or improving overall health.

What these patients are actually sold is false hope, he says. These businesses are continuously transforming and reinventing themselves, but the common thread is that they offer potentially dangerous treatments based on unproven science.

Ikonomou is also the chair of the International Society for Cell and Gene Therapy (ISCT) Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapy.

Stem cell therapy involves the conversion of stem cells into specific types of cells, such as heart or blood cells. These cells are then transplanted into a patient to promote healing.

While there are companies that carefully develop cell-based treatments following established regulatory and ethical standards, there has also been an explosion of businesses since the mid-2000s that advertise directly to consumers and evade regulations to provide unsafe and ineffective treatments, he says.

These businesses operate in gray regulatory areas, frequently branding stem cell therapies as medical interventions rather than therapeutic drugs to avoid the need for U.S. Food and Drug Administration (FDA) approval, Ikonomou says, adding that according to published research, there are more than 1,000 of these unsafe businesses in the U.S.

They offer purported stem cell therapies for nearly every condition imaginable, from diabetes and autism to Alzheimers disease. There are also reports of people suffering physical harm including blindness and death from unsafe stem cell interventions, such as drawing and reinjecting patients with their own fat cells, he says.

Im not surprised that a lot of these businesses went into COVID treatments, says Ikonomou. They went where the money is and took advantage of peoples fears.

The treatments range in price from a few thousand to tens of thousands of dollars, and often patients are encouraged to receive the expensive infusions every few months. Many people go into severe debt to acquire these ineffective treatments, he says.

This year, the FDA has issued several letters to offending businesses, including those advertising cell therapies for COVID-19, says Ikonomou. The Federal Trade Commission has also cracked down on misleading advertising from stem cell therapy clinics, he says.

However, many of these clinics are small and difficult to track. Patient prudence is key to avoiding harmful interventions, he says.

Ikonomou shares a list of steps the public can take to ensure a stem cell therapy is safe, proven and ethical.

Ikonomou also urges patients to share any questions they have with their physicians, who often are the gatekeepers for medical treatment. His best advice to patients: If something sounds too good to be true, it probably isnt true.

For information on safe and ethical cell therapies, visit the ISCT website.

Read more from the original source:
Unproven 'stem cell' therapies for COVID-19 pose harm to public, says UB expert - UB Now: News and views for UB faculty and staff - University at...

Gilead’s Kite snares a landmark FDA OK on a $373000 CAR-T therapy which comes with bragging rights to the first true franchise op – Endpoints News

Bob Nelsen had been quietly wondering how to eradicate viruses for years, before one day in 2015, he welcomed a pair of immunologists into the ARCH Venture Partners offices on the 34th floor of Seattles Wells Fargo Building.

Louis Picker and Klaus Frh, professors at Oregon Health & Science University, had by then spent 5 years running around the country in search of funding for their startup, TomegaVax, and Frh, at least, was nearing wits end. The Gates Foundation was interested but told them they needed other investors. Investors told them to come back with more data, pharmaceutical executives said theyre in the wrong game too little money to be made fighting infectious disease. Still, a well-connected board member named Bob More landed them a meeting with the coveted venture capitalist, and so, in a narrow conference room overlooking the Puget Sound, Picker prepared to again explain the idea he had spent 15 years on: re-engineering a benign microbe into the first vaccines for HIV and better ones for hepatitis and tuberculosis.

This lightbulb went on his head, Picker recalled in a recent interview. Most of them just didnt get it. And Bobs hit.

By that point, Nelsen was more than just a venture capitalist. Scraggly and greying but no less opinionated at 52, he was mobbed at biotech conferences, having earned a reputation for crass wisdom and uncanny foresight, for making big bets on big ideas that changed medicine. Those ideas included DNA sequencing, which he first cut a check for in the 90s, and leveraging the immune system to tackle cancer. He earned millions making billion-dollar companies.

Yet for years he had harbored an almost singular obsession: I hate viruses, he told Forbes in 2016. He told me he was pissed off at them. The obsession drove him to his first biotech investment in 1993, for an inhalable flu vaccine approved a decade later and still in use. And it drove him to invest in CAR-T as a potential cure for HIV, years before it proved a wildly effective treatment for some cancers.

Now, listening to Picker talk about T cells and antibodies and the curious biology of cytomegalovirus, Nelsen began wondering if it was time for another bet. Pickers technology was not only promising, he reasoned, it could be the basis of a company that changed how researchers approached viruses. Instead of trying to come up with an antidote for every pathogen, you could do what cancer researchers had learned to do, and harness the immune system to do the work for you.

This wasnt a popular opinion at the time. Its like the least trendy idea in the world, Nelsen told me. People would say, Why the hell are you going into infectious disease?

Unlock this article along with other benefits by subscribing to one of our paid plans.

SUBSCRIBE SIGN IN

More:
Gilead's Kite snares a landmark FDA OK on a $373000 CAR-T therapy which comes with bragging rights to the first true franchise op - Endpoints News

National Institutes of Health Selects Humanigen’s Lenzilumab for its COVID-19 Big Effect Trial – Business Wire

BURLINGAME, Calif.--(BUSINESS WIRE)--Humanigen, Inc., (HGEN) (Humanigen), a clinical stage biopharmaceutical company focused on preventing and treating cytokine storm, announced that the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), which is part of the United States Government Department of Health and Human Services (HHS) as represented by the Division of Microbiology and Infectious Diseases (DMID), and Humanigen have executed a clinical trial agreement for lenzilumab, the companys proprietary Humaneered anti-human granulocyte macrophage-colony stimulating factor (GM-CSF) monoclonal antibody drug candidate, as an agent to be evaluated in the NIAID-sponsored Big Effect Trial (BET) in hospitalized patients with COVID-19.

BET will help advance NIAIDs strategic plan for COVID-19 research, which includes conducting studies to advance high-priority therapeutic candidates.1 Identification of agents with novel mechanisms of action for therapy is a strategic priority.

This trial builds on initial data from NIAIDs Adaptive COVID-19 Treatment Trial (ACTT) that demonstrated Gileads investigational antiviral, remdesivir, may improve time to recovery in hospitalized patients with COVID-19. BET will evaluate the combination of lenzilumab and remdesivir on treatment outcomes versus placebo and remdesivir in hospitalized COVID-19 patients. The trial is expected to enroll 100 patients in each arm of the study with an interim analysis for efficacy after 50 patients have been enrolled in each arm.

We have been encouraged by the lenzilumab efficacy and safety data demonstrated in the compassionate use series in COVID-19 patients and are thrilled that NIH selected lenzilumab to be part of its Big Effect Trial, said Cameron Durrant, MD, MBA, chief executive officer of Humanigen. With data from the BET and our ongoing Phase III study, we will have data from approximately 500 hospitalized COVID-19 patients.

Experience with SARS-CoV-2 indicates that infection of the respiratory tract is rapid and damage is primarily mediated by the host inflammatory response. These conditions may make it difficult to modify COVID-19 with a pathogen-directed therapeutic. Host-directed strategies that target the immune response may exert additional therapeutic benefit. Having previously published data demonstrating the ability of lenzilumab to prevent and/or treat cytokine storm, Humanigen believes lenzilumab may be synergistic in the treatment of patients with COVID-19 when used in combination with a direct-acting antiviral, like remdesivir, given the differing mechanisms of action.

More details on Humanigens programs in COVID-19 can be found on the companys website at http://www.humanigen.com under the COVID-19 tab, and details of the US Phase III potential registration study can be found at clinicaltrials.gov using ClinicalTrials.gov Identifier NCT04351152.

1 NIAID Strategic Plan for COVID-19 Research FY2020 FY2024. National Institute of Allergy and Infectious Diseases. April 22, 2020. https://www.niaid.nih.gov/sites/default/files/NIAID-COVID-19-Strategic-Plan-2020.pdf.

About Humanigen, Inc.

Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. We believe that our GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection. The companys immediate focus is to prevent or minimize the cytokine release syndrome that precedes severe lung dysfunction and ARDS in serious cases of SARS-CoV-2 infection. The company is also focused on creating next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, the company is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders. The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T cell engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Additionally, Humanigen and Kite, a Gilead Company, are evaluating lenzilumab in combination with Yescarta (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma in a clinical collaboration. For more information, visit http://www.humanigen.com.

Forward-Looking Statements

This release contains forward-looking statements. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual events or results may differ materially from those contained in the forward-looking statements. Words such as "will," "expect," "intend," "plan," "potential," "possible," "goals," "accelerate," "continue," and similar expressions identify forward-looking statements, including, without limitation, statements regarding our expectations for the Phase III study and the potential future development of lenzilumab to minimize or reduce the severity of lung dysfunction associated with severe and critical COVID-19 infections or to be approved by FDA for such use or to help CAR-T reach its full potential or to deliver benefit in preventing GvHD. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the risks inherent in our lack of profitability and need for additional capital to conduct the Phase III study and grow our business; our dependence on partners to further the development of our product candidates; the uncertainties inherent in the development and launch of any new pharmaceutical product; the outcome of pending or future litigation; and the various risks and uncertainties described in the "Risk Factors" sections and elsewhere in the Company's periodic and other filings with the Securities and Exchange Commission.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You should not place undue reliance on any forward-looking statements, which speak only as of the date of this release. We undertake no obligation to revise or update any forward-looking statements made in this press release to reflect events or circumstances after the date hereof or to reflect new information or the occurrence of unanticipated events, except as required by law.

View original post here:
National Institutes of Health Selects Humanigen's Lenzilumab for its COVID-19 Big Effect Trial - Business Wire

Cell and Gene Therapy Tools, and Reagents: Global Markets – PRNewswire

NEW YORK, July 27, 2020 /PRNewswire/ -- Report Scope: The scope of this study encompasses an investigation of the markets cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors.The analyst examines each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

Technological issues, including the latest trends, are discussed. The analyst examined the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

Report Includes: - 77 data tables - An overview of the global market for immunoassay-based cell and gene therapy tools, and reagents within the life sciences industry - Analyses of the global market trends, with data corresponding to market size for 2017-2019, and projections of compound annual growth rates (CAGRs) through 2024 - Information about in vitro diagnostics (IVD), its background and importance of IVD testing - Discussion on prevalence of infectious disease, metabolic disorders and chronic ailments as drivers of immunoassay testing market - Description of recent technologies such as chemiluminescent immunoassays (CLIA), fluorescent immunoassays, multiplex assays and rapid diagnostic technologies - Evaluation of market potential for immunoassay-based cell and gene therapy tools and reagents, current market size and market forecast, and market share analysis of the leading players engaged - Patent review and new developments, R&D efforts, industrial changes with emphasis on recent investments, and current state of the immunoassay technology - Company profiles of the key market participants, including Bio-Rad Laboratories, Cytiva Life Sciences, Luminex Corp., Millipore Sigma, Thermo Fisher Scientific and STEMCELL Technologies

Reasons for Doing this Study: Gene and cell therapy are emerging as important tools to treat human health.Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers.

The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene- based diseases.As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging.

This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

Contact Clare: [emailprotected] US: (339)-368-6001 Intl: +1 339-368-6001

SOURCE Reportlinker

http://www.reportlinker.com

Continue reading here:
Cell and Gene Therapy Tools, and Reagents: Global Markets - PRNewswire

Global Stem Cell Therapy Market Analysis Highlights the Impact of COVID-19 2020-2024| Increase in Awareness of Stem Cell Therapy to Boost Market…

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the global stem cell therapy market size and it is poised to grow by USD 588.22 million during 2020-2024, progressing at a CAGR of almost 7% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Although the COVID-19 pandemic continues to transform the growth of various industries, the immediate impact of the outbreak is varied. While a few industries will register a drop in demand, numerous others will continue to remain unscathed and show promising growth opportunities. Technavios in-depth research has all your needs covered as our research reports include all foreseeable market scenarios, including pre- & post-COVID-19 analysis. Download a Free Sample Report on COVID-19 Impacts

Frequently Asked Questions:

The market is fragmented, and the degree of fragmentation will accelerate during the forecast period. Gilead Sciences Inc., Holostem Terapie Avanzate Srl, Lineage Cell Therapeutics Inc., Lonza Group Ltd., Novartis AG, Nuvasive Inc., Organogenesis Inc., Osiris Therapeutics Inc., RTI Surgical Holdings Inc., and Vericel Corp. are some of the major market participants. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

Buy 1 Technavio report and get the second for 50% off. Buy 2 Technavio reports and get the third for free.

View market snapshot before purchasing

An increase in awareness of stem cell therapy has been instrumental in driving the growth of the market.

Technavio's custom research reports offer detailed insights on the impact of COVID-19 at an industry level, a regional level, and subsequent supply chain operations. This customized report will also help clients keep up with new product launches in direct & indirect COVID-19 related markets, upcoming vaccines and pipeline analysis, and significant developments in vendor operations and government regulations.

Stem Cell Therapy Market 2020-2024: Segmentation

Stem Cell Therapy Market is segmented as below:

To learn more about the global trends impacting the future of market research, download a free sample: https://www.technavio.com/talk-to-us?report=IRTNTR43432

Stem Cell Therapy Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. The stem cell therapy market report covers the following areas:

This study identifies the limited number of FDA-approved stem cell therapies as one of the prime reasons driving the stem cell therapy market growth during the next few years.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Technavios in-depth research has direct and indirect COVID-19 impacted market research reports.

Register for a free trial today and gain instant access to 17,000+ market research reports. Technavio's SUBSCRIPTION platform

Stem Cell Therapy Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Type

Customer Landscape

Geographic Landscape

Market Drivers

Market Challenges

Market Trends

Vendor Landscape

Vendor Analysis

Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

Originally posted here:
Global Stem Cell Therapy Market Analysis Highlights the Impact of COVID-19 2020-2024| Increase in Awareness of Stem Cell Therapy to Boost Market...

Enthera Pharmaceuticals Raises 28 Million in Series A Financing to Advance Restorative Therapy for Type 1 Diabetes and Inflammatory Bowel Disease -…

MILAN, Italy--(BUSINESS WIRE)--Enthera Pharmaceuticals (Enthera), a biotech company developing disease-modifying biologics to transform the therapeutic paradigm of specific autoimmune conditions by re-establishing stem cell capabilities in a non-traditional way, today announces the completion of a 28 million Series A financing round.

The Series A was co-led by seed investor Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan, and incoming investor AbbVie, with further investment from the JDRF T1D Fund (the venture capital fund of JDRF) and several Italian investors including a group coordinated by Banor SIM, a group coordinated by Banca Profilo through Arepo Fiduciaria and Indaco Venture Partners SGR. The 28 million raised is the largest VC-backed Series A funding round for an Italian biotechnology company to date.

The Series A marks AbbVies first venture investment into an Italian company. Enthera was also the first European investment of the JDRF T1D fund, who previously invested in the Company during its 4 million seed extension funding round in 2018.

The proceeds from the Series A financing will be used to obtain clinical proof-of-concept through a first-in-human study of Ent001, Entheras lead candidate. Ent001 is the only drug in development with the potential to restore the pancreatic beta cell compartment in type 1 diabetes as well as the original intestine structure in inflammatory bowel disease, in order to re-establish organ function. The funds will also be used to further develop Entheras pipeline.

Commenting on the successful Series A financing, Giovanni Amabile, CEO of Enthera, said: Having renowned investors Sofinnova Partners, AbbVie and JDRF T1D Fund choosing to support us on our journey is great validation of our company strategy and scientific method, and gives credit to the innovative science that is currently underway in Italy.

We have a revolutionary approach that offers a new perspective for intractable diseases such as type 1 diabetes and inflammatory bowel disease, and the funds raised will enable us to accelerate our lead program Ent001 to clinical proof-of-concept. Through this and the development of our wider pipeline, we have the opportunity to bring real hope to patients in need and to provide effective and safe treatments.

Graziano Seghezzi, Managing Partner of Sofinnova Partners, who co-seeded the company, stated: Entheras Series A financing is a fantastic milestone enabled by the leadership of Giovanni who, since his arrival in the company, has expanded its activities into novel therapeutic areas. This is also an opportunity for us to applaud BiovelocITAs role in transforming the breakthrough science originating from Paolo Fiorina and his team into a full-fledged enterprise. Enthera is a salient example of what can be achieved when the critical components of a biotech ecosystem are brought together to accelerate innovation in Italy: World-class science, experienced management, and dedicated financial capital.

Margarita Chavez, Managing Director of AbbVie Ventures and new addition to the Enthera Board of Directors, added: AbbVie Ventures investment in Entheras Series A demonstrates our confidence in the team and their unique approach to treating underserved autoimmune disorders. I look forward to working with Giovanni and rest of the team as we drive the Company and its innovation forward.

Enthera was formed in 2016 on the basis of research by scientific founders Prof. Paolo Fiorina and Dr. Francesca DAddio. The Company is the first spin-off of the Italian biotech accelerator BiovelocITA, founded by Sofinnova Partners, Silvano Spinelli and Gabriella Camboni, which provided the initial seed fund. A seed extension was financed in 2018 by Sofinnova Partners, JDRF T1D fund and the other existing investors.

ENDS

Notes to Editors

About Enthera

Enthera Srl is a biotech company developing first-in-class biologics to transform the treatment paradigm of specific autoimmune conditions by re-establishing stem cell capabilities in a non-traditional way. The Companys primary target indications are type 1 diabetes (T1D) and inflammatory bowel disease (IBD).

Enthera's pioneering approach capitalizes on the key discovery of the IGFBP3/TMEM219 pathway, which is involved in beta cell and stem cell apoptosis in pancreas and gut, respectively.

The Company is building a pipeline of inhibitory monoclonal antibodies (mAbs) and fusion proteins targeting the pathway via multiple angles. Its lead program Ent001 is the only drug in development with the potential to restore the endogenous pancreatic stem cell compartment in T1D as well as the original intestine structure in IBD, in order to re-establish organ function.

Enthera is a private company headquartered in Milan, Italy and founded in 2016 by Prof Paolo Fiorina and Dr Francesca DAddio at BiovelocITA, an Italian biotech accelerator. The Company is backed by Sofinnova Partners and JDRF T1D fund. Entheras discovery engine and assets are protected by a broad portfolio of patents.

For more information, visit https://www.entherapharmaceuticals.com/

Connect with us on LinkedIn.

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

For more information, please visit: http://www.sofinnovapartners.com

About AbbVie Ventures

AbbVie Ventures is the corporate venture capital group of AbbVie. We are a strategic investor, investing exclusively in novel, potentially transformational science aligned with AbbVie's core R&D interests. We measure success primarily by the extent to which our investments foster innovation with potential to transform the lives of patients that AbbVie serves. AbbVie Ventures enables its portfolio companies with both funding as well as access to AbbVie's internal network of experts across all phases of drug development, from drug discovery through commercialization.

For more information, please visit http://www.abbvie.com/ventures

For more information about AbbVie, please visit us at http://www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.

About the JDRF T1D Fund

The JDRF T1D Fund is a venture philanthropy fund accelerating life-changing solutions to cure, prevent and treat type 1 diabetes (T1D) through catalytic equity investments. Through its investments in partnership with private capital, including venture capital, corporations and foundations, the T1D Fund seeks to attract the private investment necessary to advance therapeutics, devices, diagnostics and vaccines into the hands of those living with T1D. The T1D Fund invests in areas strategically aligned with JDRF, the leading global organization funding T1D research, with an exclusive focus on supporting the best commercial opportunities. The T1D Fund reinvests any realized gains into new investments to further its mission.

http://www.t1dfund.org

BiovelocITA

BiovelocITA S.r.l. is the first Italian accelerator dedicated to biotech. It was founded by Silvano Spinelli, Gabriella Camboni and Sofinnova Partners. Thanks to BiovelocITA, entrepreneurs, scientists and investors can work together to accelerate biotech projects with an international scope and reach the proof of concept that precedes the clinical phase. Set up to offer innovative solutions to the medical community and to patients, BiovelocITA supports the creation and development of innovative biotech companies in all therapeutic areas.

For more information, see: http://www.biovelocita.com

See the original post:
Enthera Pharmaceuticals Raises 28 Million in Series A Financing to Advance Restorative Therapy for Type 1 Diabetes and Inflammatory Bowel Disease -...

21 Existing Drugs Identified Appear to Block SARS-CoV-2 Replication in The Lab – ScienceAlert

In an industry where getting one drug developed from lab to clinic can cost hundreds of millions of dollars and decades of time, it helps when scientists can be thrifty and make use of substances that already exist.

A global team of researchers has now done just that, by searching through one of the world's largest collections of already developed drugs to discover 21 potential treatments for COVID-19.

We will need considerably more research to determine if any of these drugs can be used to help COVID-19 patients, but it's an excellent first step, bypassing some of the barriers for producing completely new drugs.

"The development of a vaccine is likely to require at least 12-18months, and the typical timeline for approval of a novel antiviral therapeutic can exceed 10years," the team writes in their new paper.

"Thus, repurposing of known drugs could significantly accelerate the deployment of novel therapies for COVID-19."

The researchers took the ReFRAME Drug Repurposing Library - 11,987 compounds which have either received FDA approval or made it to clinical-trials and began testing them against SARS-CoV-2 ina line of cells called Vero, cultured from the kidney of an African green monkey (Chlorocebus sp.).

"We realised early in the COVID-19 pandemic that ReFRAME would be an invaluable resource for screening for drugs to repurpose against the novel coronavirus," says medical chemist and study co-author Arnab Chatterjeefrom Calibr, the drug discovery division of Scripps Research that created ReFRAME.

As was expected, the vast majority of the tests didn't result in much, but the team did find 100 compounds that inhibited viral replication of SARS-CoV-2 in Vero cells.

The team then demonstrated that 21 of those 100 compounds had a dose-response relationship meaning the dose required to be effective wasn't also likely to cause harm to humans. One of these was remdesivir, so that leaves 20 yet to be tested for COVID-19 treatment.

Of those 21 drugs, 13 were found to be effective at concentrations that could be safely used in COVID-19 patients and had previously entered clinical trials.

"This study significantly expands the possible therapeutic options for COVID-19 patients, especially since many of the molecules already have clinical safety data in humans," said virologist Sumit Chandafrom Sanford Burnham PrebysMedical Discovery Institute in California.

"This report provides the scientific community with a larger arsenal of potential weapons that may help bring the ongoing global pandemic to heel."

The 21 drugs are currently being tested further using small animal models or mini lungs that can mimic human tissue. However, the team has already found three that worked in human stem cell-derived lung tissue, and one showed antiviral effects in a lab-based lung culture system.

This is all exciting stuff, but as we've seen in manyexamples so far, caution is needed before rushing into the administration of new drugs, even during a pandemic - not all promising compounds will actually work against COVID-19, even if those drugs have been approved for other ailments in the past. The more treatments we have however, the better.

"While some of these drugs are currently in clinical trials for COVID-19, we believe it's important to pursue additional drug candidates so we have multiple therapeutic options," says Chanda.

The research has been published in Nature.

Read the original post:
21 Existing Drugs Identified Appear to Block SARS-CoV-2 Replication in The Lab - ScienceAlert

Paralyzed man walks after 11 years with the help of Plexus Neuro and Stem Cell Research Centre, Bengaluru – PR Newswire India

Anand met with an unfortunate road accident and suffered spinal cord injury. He was suddenly propelled from the world of the able-bodied into the world of the disabled. He was paralyzed from below the neck and couldn't even wipe his tears, which emphasized his helplessness. Finally, Plexus entered Anand's life as a glimmer of hope and Anand rehabilitation was seen as a triumphant fight where the treating team played an imperative role.

Dr Naeem Sadiq, Consultant Neurologist, Founder & Director of Plexus Neuro and Stem Cell Research Centre said, "In Anand's 11 years struggle, I saw a possibility of recovery and his father entrusted his hopes and desires on Plexus and then his journey towards recovery started. At Plexus, he was subjected to a customized treatment approach, which was planned after carrying out a critical evaluation of his strengths and weaknesses."

He further said, "A group of Rehabilitation professionals focused on every aspect of his recovery starting from his poor posture to the use of his hands and walking."

His treatment comprised of stem cell therapy & comprehensive rehabilitation program which focused on improving his strength, sensation, daily activities and bowel and bladder control. After an arduous excursion of 120 days, Anand was able to use his hands to hold a spoon and eat, write, wear his clothes and hold a walker to walk independently. His trunk became steady and regained its balance, his legs started bearing weight and he even started feeling his bowel and bladder sensations. Dr Sadiq added, "Anand's recovery defied science and at every step challenged his treating team. Anand's unwavering belief in self, motivation and determination became the highlights of his journey in Plexus."

Anand's 11-year long tussle finally ended at Plexus and this end marked the beginning of a new life for him and his family.

About Plexus

At Plexus,we are committed to providing advanced treatment to effectively treat a host of chronic diseases. Our comprehensive services such as stem cell therapy, physiotherapy, occupational therapy, speech & swallowing therapy, cognitive behaviour therapy, customised hand splinting, sensory integration therapy, cognitive rehabilitation therapy to help, treat and rehabilitate patients suffering from neurological and neuropsychiatric disorders. When you choose Plexus, you can expect swift diagnosis, a warm and friendly environment, and thorough treatment all at an affordable price.

Visithttp://www.plexusnc.com/or call+91-8904842087for more details

Media Contact :

Abhishek Saran [emailprotected] +91-9535046900 The PRview

Photo: https://mma.prnewswire.com/media/1217269/Plexus_Dr_Naeem_Sadiq.jpg

SOURCE Plexus

Read more:
Paralyzed man walks after 11 years with the help of Plexus Neuro and Stem Cell Research Centre, Bengaluru - PR Newswire India

Can progenitor cells go further than gene therapy in retinal disorders? A biotech’s answer shines in PhIIb – Endpoints News

By the time the FDA approved Luxturna the pioneering gene therapy for an inherited retinal disease Henry Klassen had been researching and treating the group of diseases known as retinitis pigmentosa for more than 20 years.

Spark Therapeutics success there had not just inspired a $4.3 billion takeover by Roche, it had also emboldened other biotechs pursuing a therapy that would deliver one of the genes tied to different variations of the disorder. Nightstar Therapeutics subsequently scored its own buyout with Biogen, and MeiraGTx recently posted early but exciting data on its J&J-partnered program.

But Klassen went a different way.

Catching on the stem cell craze right at the turn of the millennium, he took inspiration from scientists who transplanted neural progenitor cells into the retina and developed a method to grow retinal progenitor cells instead as a therapy. Starting out as the director of stem cell research at the Childrens Hospital of Orange County, he continued the work at the University of California, Irvine, eventually spinning out a biotech dubbed jCyte in 2012.

Over the weekend jCyte reported positive Phase IIb results from what they call one of the largest studies ever conducted in RP, suggesting that patients on the treatment saw improved functional vision compared to the placebo group.

The credit to the gene therapies is that theyre actively trying to fix the gene underlying the problem. Thats very commendable, and were not doing that, Klassen told Endpoints News. But our treatment as it stands should have impact across a variety of different genotypes.

The study enrolled a total of 84 patients, of whom 74 were included for the final analysis. For each patient on the primary endpoint of best corrected visual acuity (measured with glasses on), the mean change from baseline to month 12 for the sham, low dose and high dose arms were +2.81, +2.96, and +7.43 letters, respectively.

In a post hoc analysis for a target subgroup, the difference was even more prominent: +1.85, -0.15, and +16.27 letters, respectively.

There was one serious adverse event in the low dose arm, but jCyte said the grade 3 ocular hypertension resolved with treatment and other side effects were generally minor.

CEO Paul Bresge noted that the target subgroup analysis was intended to hammer out the criteria they might use to recruit patients into Phase III which would likely have a similar design and use the same primary endpoint of BCVA, the gold standard in the context of FDA. The late-stage trial is slated for 2021.

We did enroll a very wide patient population into our Phase IIb, including patients that had vision anywhere from 20/80 to 20/800, just to learn which patients would potentially be the best responders, he said.

The target subgroup is characterized by having reliable fixation on the study eye, and a study eye that does not have significantly worse BCVA (15 letters) than the fellow eye.

He added that investigators also observed encouraging results with the secondary endpoints such as low light mobility, contrast sensitivity kinetic visual fields and a vision function questionnaire, although the data werent disclosed.

Typically people think about the disease as a narrowing of this peripheral vision in a very nice granular way, but thats actually not what happens, he said about the visual fields finding. What happens in the disease is that patients lose like islands of vision. So what were doing in our tests is actually measuring [] islands that the patients have at baseline, and then what were seeing after treatment is that the islands are expanding. Its similar to the way that one would track, lets say a tumor, in oncology of course were looking for the opposite effect. Were looking for the islands of vision to expand.

The therapy works primarily by preserving photoreceptors, Klassen posits, not by generating new ones. But what he thinks is happening is that photoreceptors are regenerating the outer segment if photoreceptors are radios, these would be the antenna thereby regaining some function.

That could position it as a treatment for a different stage of the disease than Sparks or Nightstars. Klassen, whos also researching retinal reconstruction using stem cells, is happy to not view it through the competitive lens.

If you look into the future, one could imagine that gene therapies will be most effective very early in the course of a disease before photoreceptors are lost, he said. Then as photoreceptors begin to be lost anyway, if that happens, then a therapy like ours would become extremely valuable. And if ours starts to lose power late in the course of a disease, maybe cell transplantation under the retina could have a role.

Go here to see the original:
Can progenitor cells go further than gene therapy in retinal disorders? A biotech's answer shines in PhIIb - Endpoints News

BrainStorm Cell Therapeutics to Announce Second Quarter Financial Results and Provide a Corporate and R&D Update – Stockhouse

NEW YORK, July 27, 2020 /PRNewswire/ -- BrainStorm-Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company will hold a conference call to update shareholders on financial results for the second quarter ended June 30, 2020, and provide a corporate update, at 8:00 a.m., Eastern Daylight Time (EDT), on Wednesday, August 5 2020.

On the call, BrainStorm CEO Chaim Lebovits will present a corporate update, including details on the timeline for the data readout of the Company's Phase 3 pivotal trial studying the safety and efficacy of NurOwn® (MSC-NTF cell) in people with ALS. In addition, Dr. Revital Aricha. Brainstorm's Vice President of Research and Development, will provide an R&D update including recently announced data from the Company's groundbreaking preclinical study evaluating NurOwn-derived exosomes for the treatment of COVID-19 ARDS.

Thereafter, senior management officers will join the call for a Q&A session. Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com. Questions should be submitted by 5:00 p.m. EDT, Monday, August 3, 2020.

Teleconference Details BRAINSTORM CELL THERAPEUTICS 2Q 2020

The investment community may participate in the conference call by dialing the following numbers:

Participant Numbers:

Toll Free: 877-407-9205 International: 201-689-8054

Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

Replay Number:

Toll Free: 877-481-4010 International: 919-882-2331 Replay Passcode: 36017

Teleconference Replay Expiration:

Wednesday, August 19, 2020

About NurOwn®

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and initiated enrollment in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations: Preetam Shah, MBA, PhD Chief Financial Officer BrainStorm Cell Therapeutics Inc. Phone: +1-862-397-1860 pshah@brainstorm-cell.com

Media: Paul Tyahla SmithSolve Phone: +1-973-713-3768 Paul.tyahla@smithsolve.com

View original content:http://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-to-announce-second-quarter-financial-results-and-provide-a-corporate-and-rd-update-301100141.html

SOURCE Brainstorm Cell Therapeutics Inc

More:
BrainStorm Cell Therapeutics to Announce Second Quarter Financial Results and Provide a Corporate and R&D Update - Stockhouse