StemCell Therapy

ARLINGTON, Va. & REYKJAVIK, Iceland--(BUSINESS WIRE)--Kerecis, the company pioneering the use of fish skin and fatty acids for tissue regeneration and protection, is donating its Kerecis Omega3 Burn product for burn victims of the fires in California, Oregon and Washington. Qualified medical personnel wanting to take advantage of this offer should contact wildfires@kerecis.com.

These horrific fires are having a devastating effect on human lives and habitat, said G. Fertram Sigurjonsson, founder and CEO of Kerecis. We want to help those who have been burned to heal as quickly and easily as possible. We encourage medical professionals to contact us to get a supply of Kerecis Omega3 Burn for their patients.

About Kerecis Omega3 Burn

Kerecis Omega3 Burn is intact fish skin that, when grafted onto damaged human tissue, recruits the bodys own cells and ultimately is converted into living tissue. Because no disease-transfer risk exists between cold-water fish and humans, the Kerecis fish skin is only gently processed and retains its similarity to human skin, making it an ideal skin substitute. The fish skin contains Omega3 fatty acids and multiple proteins that help the product to become incorporated into the body quickly, while providing a much-needed bacterial barrier to protect the wound bed. Clinical studies have found that the Kerecis products heal wounds faster than competitive products, so patients can be ready for additional tissue building and/or move directly to split-thickness skin grafting in record time. Kerecis Omega3 Burn is available for the treatment of humans as well as for animals.

About Kerecis

Kerecis is pioneering the use of fish skin and fatty acids in the globally expanding cellular- therapy and regenerative-medicine market. The Kerecis fatty-acid-rich intact fish skin protects the bodys tissues and enables the body to regenerate tissues. The Kerecis sprayable fatty-acid topical and oral formulations protect the body from bacterial and viral infections.

The Kerecis products, which are based on fish skin and fatty acids, are currently being used to regenerate tissue in diabetic and trauma wounds (including burns), and for infection control. Kerecis is also developing products for areas such as oral surgery, plastic surgery and neurological applications.

The companys mission is to extend human life by supporting the bodys own ability to regenerate, and its vision is to become the world leader in tissue regeneration by sustainably harnessing natures own remedies. For more information, visit http://www.kerecis.com

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Kerecis to Donate its FDA-Approved Fish Skin Treatment for Burn Victims of West Coast Fires - Business Wire

TOKYO--(BUSINESS WIRE)--SanBio Co., Ltd. (headquarters: Chuo-ku, Tokyo, Representative Director and President: Keita Mori, hereafter SanBio) hereby announces that it has obtained new analytical results from the Phase 2b clinical trial (the trial) of SB623 for the treatment of chronic motor deficit resulting from ischemic stroke the SanBio Group (SanBio Co., Ltd. and its subsidiary SanBio, Inc.) conducted in the US. It also announces that based on the newly obtained results, it has updated its development plans, including in regard to late-stage clinical trials for the ischemic stroke and hemorrhagic stroke programs of SB623 in Japan.

The trial evaluated efficacy and safety of SB623 in 163 patients suffering from chronic motor dysfunction from ischemic stroke. On January 29, 2019, SanBio announced that the trial did not meet its primary endpoint, as it failed to demonstrate statistical significance in the difference in the proportion of patients whose Fugl-Meyer Motor Scale (FMMS) score improved by 10 or more points from the baseline (primary endpoint) between the treatment group that received SB623 and the control group. Since then, the SanBio Group had continued to work on additional analysis of the trial data, and results of the additional analysis are as follows.

In conducting the additional analysis, from the perspective of minimal clinically important difference (MCID, or the minimal change in scores or other metrics that could be interpreted to mean the change in a patient is clinically meaningful) and based on the results of the Phase 2 clinical trial of SB623 for the treatment of chronic motor deficit from traumatic brain injury (TBI; STEMTRA trial), the company reevaluated trial data using composite FMMS. Of the total 163 patients enrolled in the trial, the company specifically looked at 77 patients who had infarct areas smaller than a certain size (47% of all patients enrolled in this trial). The SanBio Group evaluated the proportion of patients that met one or more of the following FMMS score improvement criteria 24 weeks after treatment: 6-point improvement on FMMS score for upper extremity, 4-point improvement on FMMS score for lower extremity, and 9-point improvement on FMMS total score (all from the baseline). Of the 51 patients in the treatment group that received SB623, improvement was seen in 49%, versus in 19% of 26 patients in the control group that received sham surgery, the difference between the two groups being statistically significant (p-value of 0.02). SanBio Group thinks that even compared to the primary endpointthe proportion of patients whose FMMS score improved by 10 or more points over the baseline six months after treatmentthe endpoint using composite FMMS can adequately explain clinical significance of the treatment efficacy. Details of the additional analysis results will be announced at the financial results briefing for institutional investors and the media held on September 15, 2020. The briefing video will be made available to the public on our website on the 16th of September or thereafter.

Based on the above results, the SanBio Group has begun preparations for the next late-stage clinical trials in the ischemic stroke and hemorrhagic stroke programs of SB623. 2021. Specific designs of the clinical trials and the contents of development for those two programs will be announced promptly upon being finalized. To maximize the value of SB623 at an early stage by selecting areas to focus the Groups management resources on, the SanBio Group plans to prioritize the development of the ischemic stroke and hemorrhagic stroke programs in Japan at the same time as it prepares to file for approval of SB623 for the treatment of chronic motor deficit resulting from TBI in Japan by the end of the current fiscal year (ending January 2021). The Group, however, postponed the global Phase 3 clinical trial for the TBI program of SB623 it had planned to commence this fiscal year to the next or subsequent fiscal years.

Many patients suffering from the chronic effects of ischemic stroke are said to be regularly taking drugs to prevent recurrence. However, because there is no drug that can fundamentally cure motor dysfunction, there is high unmet need for therapeutic drugs to restore motor functions for patients in the chronic phase of stroke. The SanBio Group aims to contribute to improving the lives of these patients, as well as of their family members, suffering from motor impairment and difficulties it causes in carrying out their daily lives through SB623.

About SB623

SB623 is an allogeneic mesenchymal stem cell produced by modifying and culturing bone marrow derived from healthy donors. Implantation of SB623 cells into nerve tissues is expected to promote regeneration of damaged nerve cells. Because SB623 is made from allogeneic cells, large-scale production is possible and there is no need for complex cell processing required for treatments using autologous cells, e.g., cell preparation for each patient at medical institutions. Hence, pharmaceutical products made from allogeneic cells, such as SB623, can be provided to many patients in uniform quality.

About SanBio Co., Ltd. and SanBio, Inc.

SanBio Group is engaged in the regenerative cell medicine business, spanning research, development, manufacture, and sales of regenerative cell medicines. The Companys propriety regenerative cell medicine product, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from traumatic brain injury and ischemic stroke. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

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Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit...

"On behalf of our entire board of directors, we wholeheartedly welcome Terry to his new role as President and CEO of Vizgen," said Dr. David R. Walt, Cofounder of Vizgen; Hansjrg Wyss Professor of Biologically Inspired Engineering, Harvard Medical School; Professor of Pathology, Brigham and Women's Hospital; Core Faculty, Wyss Institute for Bioinspired Engineering, Harvard University; HHMI Professor. "Terry's technical savvy combined with his demonstrated business acumen in building out product and service offerings on a global scale will benefit Vizgen as the Company enters the next phase of commercial development."

Prior to joiningVizgen, Terry was President of Akoya Biosciences, a post he assumed after an acquisition from PerkinElmer where he served as General Manager, Quantitative Pathology Solutions. Previously, he held several executive positions at global biopharma and diagnostic companies including Roche, Hologic, and Bristol-Myers Squibb, and has extensive experience in launching innovative technologies in new markets. He holds an MBA from the University of Chicago Booth School of Business, an MS in Microbiology from Virginia Tech, and dual BS degrees in Molecular Genetics and Psychology from The Ohio State University.

"I'm excited to take the helm at Vizgen to work together with an incredibly talented team of scientists and innovators to bring an unsurpassed spatial profiling technology to market," said Mr. Lo. "Gene expression with spatial context has now become the new research frontier in unlocking core biological questions, and Vizgen's technology is regarded as a premier solution to gain insight into the molecular underpinnings of health, the progression to disease, and the development of new therapies and vaccines."

Vizgen's MERFISHtechnology was developed in the laboratory of Dr. Xiaowei Zhuang, a Howard Hughes Medical Institute Investigator and David B. Arnold, Jr. Professor of Science at Harvard University. Dr. Zhuang and Dr. Jeffrey Moffitt, a former postdoctoral fellow in Dr. Zhuang's lab andnow an Assistant Professor at the Program in Cellular and Molecular Medicine at Boston Children's Hospital and the Department of Microbiology at Harvard Medical School, are also cofounders of Vizgen. MERFISH enables spatially resolved, single-cell genomic profiling at extremely high levels of throughput and accuracy. The novel technology is used as a tool for several Human Cell Atlasprojects and was named a "Technology to Watch" by Nature for mapping the transcriptome.

Vizgen launched in January 2020 with a $14M Series A Financing led by ARCH Venture Partners and Northpond Ventures. Last month the Company announced an early release program for its spatial genomics platformto provide scientific investigators an opportunity to gain access to the proprietary technology to accelerate their research. Vizgen's technology is already being employed by world-leading academic research institutions including the Broad Institute of MIT and Harvard and The Rockefeller University.

For more information email: [emailprotected]

About Vizgen Vizgen is developing the next generation of spatially resolved genomic profiling tools that enable researchers to gain new insight into the biological systems that underlie human health and disease. The company's patented MERFISH technology enables massively multiplexed, genome-scale nucleic acid imaging with high accuracy and unrivaled detection efficiency at subcellular resolution. MERFISH provides transformative insight into a wide range of tissue-scale basic research and translational medicine in oncology, immunology, neuroscience, infectious disease, developmental biology, and regenerative medicine. For more information, go towww.vizgen.com, connect on social media

@Twitter,@LinkedInandFacebook, and join the MERFISH Group at: https://bit.ly/merfishgroup.

SOURCE Vizgen

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Vizgen Taps Akoya Biosciences Executive Terry Lo As New President and CEO - PRNewswire

VANCOUVER, B.C., Sept. 14, 2020 /PRNewswire/ -- The Global Femtech Marketis expected to reach USD 60.01 Billion by 2027, according to a new report by Emergen Research. Demand for the femtech industry is motivated mainly by the growing burden of both chronic and infectious diseases among the world's female population. An increase in the number of health problems relating to women would stimulate competition for technologically innovative healthcare solutions. Growing women's emphasis on reproductive health and sexual empowerment in developing economies would further encourage development in the industry.

Increasing awareness among women of the detection and management of early illness as part of the patient care program would improve the market outlook. Various efforts by government and other agencies in developing countries to raise awareness of women's health would accelerate the development of the industry. Furthermore, an increasing tendency towards daily preventive care check-ups, as well as the advancement of user-friendly technology to track individual health problems, may prove beneficial to the developments in the women's health industry.

While more and more people today choose to be more transparent about their health concerns and treatment, in some of the lesser developed regions, women's health issues remain stigmatized. For these places, Femtech applications are likely to be favored because the scanning is less invasive and more secure. Increasing population growth is related to being one of the main factors behind the case.

Request free sample of this research report at: https://www.emergenresearch.com/request-sample/37

Key Highlights From The Report

Read more at: https://www.emergenresearch.com/industry-report/femtech-market

For the purpose of this report, Emergen Research has segmented into the Global Femtech Market on the basis of type, end-use, application, and region:

Type Outlook (Revenue: USD Billion; 2017-2027)

End Use Outlook (Revenue: USD Billion; 2017-2027)

Application Outlook (Revenue: USD Billion; 2017-2027)

Regional Outlook (Revenue, USD Billion; 2017-2027)

Find more research reports on healthcare and pharmaceuticals industry, by Emergen Research:

Regenerative Medicine MarketRegenerative Medicine Market By Product (Tools, Therapeutics), By Therapeutic Category (Musculoskeletal, Dermatology, Immunology & Inflammation, Cardiovascular), and By Applications (Wound Care, Musculoskeletal Disorders, Ocular Disorders), Forecasts to 2027

Next-Generation Sequencing MarketNext-Generation Sequencing Market by Technology (Whole Exome, Whole Genome, Others), By Workflow (Sequencing, Pre-Sequencing, Others), By Application (Consumer Genomics, HLA Typing, Others) and By End-Use (Academic, Clinical, Others), Forecasts to 2027

RFID in Healthcare MarketBy Product (Tags, Systems & Software) and By Application (Asset Tracking, Patient Tracking, Pharmaceutical Tracking, Blood Tracking, Others), Forecasts to 2027

Non-Invasive Prenatal Testing MarketBy Method, By End-Use, By Application, By Region, Forecasts to 2017-2027

Viral Vector and Plasmid Manufacturing MarketBy Vector Type (Retrovirus, Adenovirus, Others), By Workflow (Upstream, Downstream), By Disease (Cancer, Genetic Disorders, Others), By Application (Gene Therapy, Retailers) and By End-User, Forecasts to 2027

Interoperability Solutions in Healthcare MarketBy Level (Foundational, Structural, Semantic), By Product Type (Services, Solutions), and By Application (Diagnostics, Treatments, Others), Forecasts to 2027

About Emergen Research

At Emergen Research, we believe in advancing with technology. We are a growing market research and strategy consulting company with an exhaustive knowledge base of cutting-edge and potentially market-disrupting technologies that are predicted to become more prevalent in the coming decade.

With market-leading insights and an in-depth understanding of leading and niche technologies, our solutions address the most pertinent questions for your business needs. A major technological shift has been witnessed towards creating a 'Circular Economy,' fuelled by factors, such as the increased adoption of bio-based materials, along with other methods for achieving carbon neutrality. We are conversant in technologies, viz., Artificial Intelligence (AI), Augmented Reality (AR), Virtual Reality (VR), Robotic Process Automation (RPA), Smart Manufacturing, Internet of Things (IoT), Big Data Analytics, Machine learning, Nanotechnology, Edge Computing, Blockchain Technology, Cloud Computing, Vehicle Electrification, Advanced Maintenance Analytics, and Predictive Maintenance, among other prevalent and emergent technologies.

Contact Us:Eric LeeCorporate Sales SpecialistEmergen Research | Web: https://www.emergenresearch.comE-mail: [emailprotected]

Read full Press Release at :https://www.emergenresearch.com/press-release/global-femtech-market

SOURCE Emergen Research

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Femtech Market to Reach USD 60.01 Billion By 2027 | CAGR of 15.6%: Emergen Research - PRNewswire

Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer

Experienced biotech executive to lead the next phase of growth

14 September 2020

LONDON, Silence Therapeutics plc, AIM:SLN and Nasdaq: SLN (Silence or the Company), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, today announces the appointment of Mark Rothera as President and Chief Executive Officer (CEO) and Board member, effective immediately. Iain Ross, who has been Executive Chairman since December 2019, has today assumed his previous position of Non-Executive Chairman.

Mr. Rothera brings more than 30 years of experience in the biopharmaceutical industry, with a strong record of commercial and operational leadership, including driving the successful build of multiple biotech companies, predominantly in the field of rare or specialty diseases. Prior to joining Silence, Mr. Rothera served as CEO of Orchard Therapeutics (Orchard), where he oversaw its transformation from a small U.K.-based, privately held company with two clinical-stage programmes into a leading gene therapy company with seven clinical-stage programmes and fully integrated capabilities. Under his leadership, Orchard completed an initial public offering of American Depositary Shares on the Nasdaq Global Market and during his tenure that company secured more than $600 million in financing and grew from a market capitalization of $250 million to more than $1.7 billion at its peak.

Prior to Orchard, Mr. Rothera served as Chief Commercial Officer of PTC Therapeutics (PTC), where he helped transition that company from a privately held R&D biotechnology company to a publicly traded, commercial-stage company with a global footprint, including the successful launch of two rare disease therapies. He also previously served as Global President of Aegerion Pharmaceuticals Inc. and Vice President and General Manager of commercial operations at Shire Human Genetic Therapies for Europe, Middle East and Africa. Mr. Rothera received an M.A. in Natural Sciences from Cambridge University and an M.B.A. from the European Institute for Business Administration (INSEAD).

Based out of Silences New York City office, Mr. Rothera will lead the continued global expansion of the Company. His appointment follows the completion of Silences Nasdaq listing on 8 September 2020 and aligns with the strategy of increasing the Companys presence in the United States.

Iain Ross, Chairman of Silence Therapeutics plc, said: "On behalf of the Silence Board and the entire Silence team, I welcome Mark to the Company. Following a thorough search, Marks appointment reflects his proven leadership skills and strong track record in growing successful biotechnology companies and building shareholder value. I believe he will now provide the leadership necessary to grow Silence into a leading international biotechnology company built upon our innovative siRNA technology platform, proprietary product pipeline and validating industry partnerships.

On a personal note, and on behalf of the Board, I would like to thank the management team and staff at Silence for their support, hard work and tremendous resilience during the current COVID-19 pandemic and over the past nine months whilst I have been Executive Chairman. The Company has made great strides during this period, and is now in a strong position, both operationally and financially, and ready for Mark to take the helm.

Mark Rothera, President and CEO of Silence Therapeutics plc, added: It is an honour to take the role of leading Silence at this time in the Companys history. I believe the Company is poised to capitalise on its important siRNA technology platform, pipeline and research capabilities built over 18 years, and position itself as a leader in the RNAi field. The Company has made great strides under Iains leadership and I look forward to working with the Board, the management team and Silence employees to build upon this momentum.

Director disclosures

The following information is being disclosed pursuantto Rule 17 and paragraph (g) of Schedule 2 of the AIM Rules for Companies.

Mark Rothera

Full name and age: Mark Andrew Rothera (aged 58)

Current Directorships or Partnerships:Genpharm

Previous Directorships or Partnerships in the last 5 years:Orchard Therapeutics plcPTC Therapeutics International LimitedAlliance for Regenerative Medicine

No further information in connection with his appointment is required to be disclosed under Schedule Two, paragraph (g) of the AIM Rules for Companies.

Enquiries:

About Silence TherapeuticsSilence Therapeutics is developing a new generation of medicines by harnessing the bodys natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet medical need. Silences proprietary technology can be used to engineer short interfering ribonucleic acids (siRNAs) that bind specifically to and silence, through the RNAi pathway, almost any gene in the human genome to which siRNA can be delivered. Silences wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of Lipoprotein(a) and SLN124 to address beta-thalassemia and myelodysplastic syndrome. Silence is also developing SLN500 in partnership with Mallinckrodt Pharmaceuticals to reduce the expression of the C3 protein for the treatment of complement pathway-mediated diseases. Silence maintains ongoing research and collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals and Takeda. For more information, please visit: https://www.silence-therapeutics.com/

The person who arranged for the release of this announcement on behalf of the Company was Rob Quinn, Chief Financial Officer.

Forward-Looking StatementsCertain statements made in this announcement are forward-looking statements, including with respect to the Companys clinical and commercial prospects. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

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Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer - GlobeNewswire

Daniel Dennett has inspired many people, but perhaps none more than his former student Jeff Stibel, A95, an entrepreneur and brain scientist. Now Stibel is ensuring that the influential philosophers legacy will continue at Tufts, with a generous gift to create a consortium at the university focused on cognitive and brain science.

Stibels gift will launch the Stibel Dennett Consortium for Brain and Cognitive Science, which will bring together important research and teaching in the field. The consortium will cross university departments and schools, including psychology, biology, philosophy, education, engineering, and medicine, and will serve students, faculty, alumni, and the wider community.

I was inspired to create a new consortium at Tufts that will serve as a center of gravity, to explore important and groundbreaking cognitive and brain science issues through teaching and research, said Stibel, who is the author of two books and a USA Today column on the workings of the mind.

With his fellow partners Stibel has also given to the university BrainGate, Inc., a company that holds intellectual property enabling technologies to read and translate brain signals through a computer interface. The brain's motor cortex sends out electrical pulses that can be recorded by this technology and decoded into motor commands. In the future, the companys technology could help people with spinal injuries or locked-in syndrome control devices, such as a robotic arm or an exoskeleton that would allow a paralyzed person to walk.

The donation of BrainGate, Inc., combined with Stibels support for faculty, promises to spur new research at the university. Stibels gift includes funds to endow two professorships in the School of Arts and Sciences. Gina Kuperberg has been appointed the inaugural Dennett Stibel Professor of Cognitive Science, and Stephanie Badde has been recruited to the faculty as the Stibel Family Assistant Professor of Brain and Cognitive Science.

Kuperberg and Badde plan to build upon the BrainGate technology, exploring a deeper understanding of how the brain processes language as well as how the brain gathers information from our senses and tells our body how to move. Other faculty at Tufts also plan to explore research opportunities related to the BrainGate intellectual property.

What Jeff Stibel has given us is priceless, said James Glaser, dean of the School of Arts and Sciences. The two professorships have enabled us to recognize the excellence of Gina Kuperberg, an important cognitive science faculty member, and to recruit a talented new colleague to the program. And the gift of BrainGate, Inc., will help solidify Tufts international reputation as a locus of excellence in cognitive science.

In just one of its potential applications, the Stibel gift may lead to a deeper understanding of the very nature of human memory, said Michael Levin, A92, the Vannevar Bush Professor of Biology and director of the Allen Discovery Center at Tufts . Levin studies regenerative biologythe process of replacing or "regenerating" human or animal cells, tissues, or organs to restore or establish normal function.

Advances in regenerative medicine depend on understanding electrical anatomical memory, which is like memory in the brain. BrainGates technology could offer key insights into how cells communicate to signal growth, adaptation to trauma, or even the storage of memories.

Using the technology to interpret the communication between cells, we could find out how memories are stored and encoded in tissue, and learn to decode them, Levin said. We also could learn how memories can survive remodeling of the tissue, how memories can be moved or copied, and how memories can belong to a unified self. Levin is currently collaborating with Dennett on research and a publication related to cellular memory and cognition

Tufts will launch the Stibel Dennett Consortium in the fall, through an online event for the Tufts community with other programming to follow.

Angela Nelson can be reached at angela.nelson@tufts.edu.

Stephanie Badde, Stibel Family Assistant Professor of Brain and Cognitive Science: We think our senses give us a good impression of the physical reality around us, but that's just not true. The information were getting is actually very spottyit's as if were wearing blurry glasses with a small hole in the middle. The brain performs a lot of work to take in this input and give us the impression we have. My research asks, How does the brain do this miracle?

With the BrainGate patents, we hope to find out more about how movements and sensory information are connected in the brain, and how this might be leveraged so that people can regain the sense of touch and sense of body posture where theyve lost them.

Gina Kuperberg, Dennett Stibel Professor of Cognitive Science: We take it for granted that as we talk to one another, we are literally transferring thoughts from one mind to another. Language is what enables humans to communicate, and ultimately, language is a code. My research program is aimed at understanding this code. We're trying to figure out not only where, when, and how the brain uses language to communicate, but also the nature of the neural code itself.

The whole idea of decoding brain activity is so important, not only medically for people in the future, but for understanding the nature of the human brain and thought. And were really on the cutting edge of being able to do that, particularly now with the BrainGate intellectual property.

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Getting Smarter About the Mind | Tufts Now - Tufts Now

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the currently available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

[Read: These tech trends defined 2020 so far, according to 5 founders]

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene-editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts as a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up our genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for the immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the number of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibodies against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually, the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology, provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

This article is republished from The Conversation by Samira Kiani, Associate Professor of Pathology, University of Pittsburghunder a Creative Commons license. Read the original article.

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How CRISPR is tackling the troubling immune response thats plagued gene therapy until now - TNW

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

SOURCE GreenLight Biosciences

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GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

JOHNSON CITY, Tenn., Sept. 10, 2020 /PRNewswire/ --LabConnect, the preeminent provider of clinical trial central lab services, today announced the expansion of its Johnson City facility to support its significant growth in cell and gene therapy and immuno-oncology studies. The company,which recentlyrelocated its headquarters to Tennessee, is doubling the capacity of its biorepository for sample storage and its clinical trial kit building capacity. LabConnect welcomed Tennessee Governor Bill Lee, Congressman Phil Roe, Economic Development Commissioner Bob Rolfe, elected officials, and other dignitaries to celebrate this milestone in the company's growth.

"While we have all had to adjust and adapt during these unprecedented circumstances, Tennessee's business climate has remained strong and welcoming to companies around the globe," said Gov. Lee. "I applaud LabConnect for continuing to invest and create jobs in our state and for choosing to bring its headquarters to Tennessee. I look forward to the many great things that will come from this facility in Johnson City."

"We are pleased that another company has chosen to expand its presence in Tennessee," Commissioner Rolfe said. "LabConnect is committed to leading the way in central laboratory services, which will have a global impact from its Tennessee=based facility. We appreciate LabConnect for its continued innovation and for creating high quality jobs."

"We are excited that the incentives with the State, Tennessee Valley Authority, and Northeast Tennessee Regional Economic Partnership have enabled LabConnect to expand its operations," said Tom Sellig, LabConnect CEO. "Our location offers several advantages which has allowed us to provide unique services to pharmaceutical and biotech clients. We are currently serving 200 leading biopharmaceutical clients and proud of the more than 20 products we have supported that are now FDA approved and used to treat patients around the world. The expanded capacity will allow us to scale our organization to meet LabConnect's growing demand for our clients' cell & gene therapy, rare and orphan diseases, and immuno-oncology projects."

For more information, visit http://www.labconnect.com.

About LabConnectConnect with LabConnectthe preeminent provider of central laboratory support services for analytically and logistically complex studies such as immuno-oncology, cell and gene therapies, and rare & orphan diseases. The company offers unique and innovative services that have been specifically designed to meet the exacting demands of today's clinical trials. The worldwide scope of services includes routine and specialized testing, real-time sample tracking, data integration, biorepository, sample processing and specialized functional outsourcing. Leading the evolution in central laboratory services since 2002, LabConnect's services are customized to fit the unique needs of biopharmaceutical clients. Get connected by requesting a proposal at http://www.labconnect.com or via email at [emailprotected].

SOURCE LabConnect

http://www.labconnect.com

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LabConnect Announces Expansion to Support Cell & Gene Therapy Growth - PRNewswire

New Jersey contract manufacturer Catalent has been right in the mix in the COVID-19 response effort, signing pacts to help produce frontrunners in the vaccine hunt. Now, the company is fleshing out a Maryland facility to aid in that effortand position Catalent's cell and gene therapy offerings well into the future.

Catalent will infuse $130 million into its cell and gene therapy manufacturing facility in Harmans, Maryland, to broaden the CDMO's late-stage production capacity, the company said Wednesday.

The newest investment will add five late-stage clinical and commercial manufacturing suites to the Harmans site, expected to go online in the first half of 2022, Catalent said.

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That expansion will bring the total number of manufacturing suites to 15 at the planned 350,000-square-foot complexnear the Baltimore/Washington International airport. The Harmans facility recently received FDA approval for commercial production, and its initial 10 manufacturing suites are set to be fully operational by the first quarter of 2021.

The five new suites will be located in a second building at the site that will also house cold-storage warehousing and added office space, Catalent said. The Harmans complex is one of five Maryland sites for Catalent's cell and gene therapy manufacturing portfolio.

Adding capacity at its Harmans site is a future play for Catalent in the bustling cell and gene therapy space, but the facility could also benefit the CDMO's immediate COVID-19 response efforts.

RELATED:AstraZeneca ropes in Catalent gene therapy site to produce viral vectors for COVID-19 vaccine

Last month, British drugmaker AstraZeneca tappedCatalent to help produce bulk drug substance and viral vectors at the Harmans facility for the University of Oxford's adenovirus-based COVID-19 vaccine.

Catalent will start production there this quarter,buildingon its previous pact with AstraZeneca for fill-finish and packaging duties at its Anagni, Italy, site.

Catalent's work will include production of viral vectors for a genetically modified form of the adenovirus used in Oxford's shot, dubbed AZD1222. The modified virus, known as ChAdOx1 nCoV-19, aims to induce a lasting immune response to spike proteins added to the virus's surface.

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Catalent injects $130M into Maryland cell and gene therapy site drafted into COVID-19 vaccine hunt - FiercePharma

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution - Endpoints News

CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C written response only meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), to obtain OTATs concurrence on the commencement of its next clinical trial for SRP-9001 using commercial process material. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) is Sareptas investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

Among other items, OTAT has requested that Sarepta utilize an additional potency assay for release of SRP-9001 commercial process material prior to dosing in a clinical study. Sarepta has several existing assays and data that it believes could be employed in response to OTATs request. However, additional dialogue with the Agency is required to determine the acceptability of the potency assay approach.

We look forward to working with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study. We will provide further updates as we are able, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. Every day, thousands of children degenerate from the irreversible damage caused by Duchenne muscular dystrophy. It is for that reason that we will work relentlessly with the Division to satisfy any requests of OTAT and continue the advancement of a potentially transformative therapy for these patients.

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: delays in the commencement of Sareptas next clinical study for SRP-9001 could delay, prevent or limit our ability to gain regulatory approval for SRP-9001; any inability to complete successfully clinical development could result in additional costs to Sarepta or impair Sareptas ability to generate revenues from product sales, regulatory and commercialization milestones and royalties; SRP-9001 may not result in a viable treatment suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -...

Collaboration partners,Neurocrine Biosciences (NASDAQ:NBIX) and Voyager Therapeutics (NASDAQ:VYGR) announced data from an early stage clinical trial with Parkinson's disease patients and NBIb-1817, an experimental gene therapy. Three years after a single administration, 14 out of 15 patients are still reporting motor function improvements.

At the moment, NBIb-1817 is in the middle of a phase 2 study that was put on hold in April due to the COVID-19 pandemic. If allowed to restart, the phase 2 RESTORE-1 trial will randomize patients to receive NBIb-1817 or a placebo, then measure for a change in "On" time without troublesome dyskinesia.

Image source: Getty Images.

This potential new treatment option uses a viral vector to deliver a gene encoding an enzyme that helps Parkinson's disease patients convert levodopa into the dopamine they need.

Administering NBIb-1817 requires magnetic resonance imaging (MRI) to guide a pair of infusions that deliver the therapy directly into the striatum, a structure deep in the center of the brain. Needles through the skull aren't anybody's idea of a good time, but the side effects that come with daily doses of levodopa aren't any fun either.

For patients with severe Parkinson's disease that has a diminishing response to levodopa, the temporary discomfort that comes with NBIb-1817 treatment seems like a trade-off most will be willing to accept. Three years after a single treatment with NBIb-1817, patients were able to reduce their daily levodopa doses from a baseline of 1500.9 milligrams per day to 1061.4 milligrams per day. Despite reducing their levadopa intake, patients receiving three different dosage strengths of NBIb-1817 improved average "On" time by up to 2.23 hours.

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Parkinson's Disease Patients Get Long-Term Benefits From Experimental Gene Therapy - The Motley Fool

CAMBRIDGE, Mass., Sept. 15, 2020 /PRNewswire/ --Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced that Bristol Myers Squibb (NYSE:BMY) has exercised its option to an exclusive worldwide license to a cell therapy candidate based on Obsidian's cytoDRiVE technology for the controlled expression of the immunomodulatory factor CD40L. This announcement marks the first opt-in decision by Bristol Myers Squibbsince the companies announced their collaboration to develop novel cell therapies in January 2019. Under the terms of the agreement, Obsidian is eligible to receive potential future milestone and royalty payments.

"We are very interested in exploring innovative approaches to developing engineered cell therapies, including the cytoDRiVE platform," said Rupert Vessey, D. Phil., Executive Vice President, Research and Early Development, Bristol Myers Squibb. "By controllingthe expression of armed payloads like CD40L, Obsidian's cell therapy candidates may have the potential to overcome tumor microenvironment resistance and unlock the power of cell therapy in solid tumors and other malignancies."

"This announcement marks an important milestone validating Obsidian's cytoDRiVE platform, and we look forward to continuing to work with Bristol Myers Squibbto bring powerful new immunotherapies to patients," said Paul K. Wotton, Ph.D., Chief Executive Officer of Obsidian Therapeutics. "We are also pleased with the pace with which our own pipeline programs are progressing as we continue to advance our lead controllable tumor infiltrating lymphocyte (TIL) therapy to the clinic."

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:

Maggie BellerRusso Partners, LLC[emailprotected]646-942-5631

SOURCE Obsidian Therapeutics

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Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE Cell Therapy Candidate - PRNewswire

Pfizer has tipped its pipeline to deliver new products that add more than $15 billion to revenues by 2025. The anticipated pipeline contributions led Pfizer to predict it will weather the next wave of patent expirations that is set to start in 2026.

At its virtual investor day Tuesday, Pfizer predicted (PDF) that JAK1 inhibitor abrocitinib, antisense therapy vupanorsen and a 20-valent pneumococcal conjugate vaccine can generate peak annual revenues of more than $3 billion each. Pfizer also tipped its Duchenne muscular dystrophy gene therapy to rake in an excess of $2 billion and identified six other potential blockbusters.

With Pfizer tipping another five pipeline prospects to generate revenues of $500 million to $1 billion, the Big Pharma said sales of as-yet-unapproved experimental products could come to $15 billion by 2025.

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The peak years of the experimental medicines likely lie beyond 2025. The timing of the anticipated peaks of those products coincides with the years in which Pfizer expects to face another patent cliff.

In 2026 and 2027, the U.S. basic product patents on Prevnar 13, Eliquis and Xtandi are set to expire, potentially exposing products that generated sales of $6.3 billion in Pfizers home market last year to off-patent competition. All told, analysts expect Pfizer to lose up to $20 billion in sales due to patent expirations starting in 2026. Pfizers internal forecast is roughly in line with analyst estimates.

After Lyrica went off patent last year, Pfizer entered a window in which its portfolio is virtually free from the loss of exclusivity through to 2026. The lull gives Pfizer a period to get some of its pipeline prospects to market and start growing their sales, leading management to predict the current slate of experimental assets will at least replace the revenues lost to off-patent rivals starting in 2026.

That prediction rests on Pfizers ability to get key pipeline products to market. Pfizer used the virtual event to claim a growing effectiveness in that regard. As of 2015, 5% of the drugs Pfizer took into the clinic went on to win approval. By 2019, that figure had increased to 9%, causing Pfizers success rate to go from well below the industry average to slightly above the performance of its peers.

Pfizer also noted an improved phase 2 success rate. In 2017, 17% of Pfizers phase 2 trials succeeded. Since then, Pfizer has averaged 46%. The success rate so far this year stands at 53%. Pfizer claims to be moving faster, too, stating it is on course to reduce development timelines by 2.5 years between 2017 and 2021. Automation of processes accounts for the biggest anticipated time saving.

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Pfizer tips pipeline to add $15B to sales in coming years - FierceBiotech

TEL AVIV, Israel, Sept. 15, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) will present today a corporate overview, including an update on the OVAL pivotal study, at the H. C. Wainwright 22nd Annual Global Investment Conference being held virtually on September 14-16, 2020.

"Our OVAL trial of VB-111 in ovarian cancer continues to progress well, with over a third of the study participants already enrolled," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "We are pleased that the high response rate seen in our interim analysis in March, continues to be high in the total patient population to date. With blinded data becoming more mature, we currently see a good correlation between the CA-125 and RECIST responses, as well as with preliminary PFS and OS data. So far, OVAL blinded data recapitulate what we have seen in our positive Phase 2 study, which is very encouraging."

Presentation Details:

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Michael RiceLifeSci Advisors, LLC(646) 597-6979

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VBL Therapeutics to Provide an Update on the OVAL Study Today at the H. C. Wainwright 22nd Annual Global Investment Conference - GlobeNewswire

As the global COVID-19 situation is rapidly changing, staying abreast with the latest news can be challenging. In this article, Sheraz Gul provides an overview of the broad range of potential treatments in development and discusses how the regulatory landscape can shift at any time.

IN EARLY AUGUST 2020, the World Health Organization (WHO) reported the number of confirmed cases of COVID-19 has exceeded 20 million including over 700,000 deaths. These staggering figures have come about despite global lockdown measures being in place for the past few months. The resulting flattening of confirmed cases and deaths is now being jeopardised because, as the lockdown measures are being relaxed, we are seeing a resurgence of cases in some locations. In order to prevent a possible second wave of COVID-19 cases and deaths, the wearing of masks in public places is now mandatory in many countries. At the start of August 2020, New Zealand, which had gone 102 days without recording a locally transmitted case of COVID-19, saw new cases emerging. This highlights that we are still vulnerable to the spread of the virus.

Thus far, no US Food and Drug Administration (FDA)-approved therapy for use to treat COVID-19 is available. In order to provide some respite, on 1 May 2020 the FDA, as part of its Coronavirus Treatment Acceleration Program (CTAP), issued an Emergency Use Authorization (EUA) for the investigational antiviral drug remdesivir to treat suspected or laboratory-confirmed COVID-19 in adults and children hospitalised with severe disease. Although there is limited information known about the safety and effectiveness of using remdesivir to treat people in the hospital with COVID-19, this investigational drug has been shown in a clinical trial to shorten the time to recovery in some patients. It is noteworthy that prior to this, on 28 March 2020, the FDA also issued an EUA for chloroquine and hydroxychloroquine. This was subsequently revoked on 15 June 2020, as the FDA considered that chloroquine and hydroxychloroquine no longer met the statutory criteria for the EUA. In conclusion, the use and revocation of COVID-19 drugs can change at any time, so referring to official guidance is essential.

A recent update of the US ClinicalTrials. gov website listed around 2,000 COVID-19 interventional studies that are underway, around 90 of which are now completed. These cover a broad range of potential treatments including: 1) antiviral drugs to prevent viruses from multiplying; 2) immunomodulators aimed at reducing the bodys own immune reaction to the virus; 3) neutralising antibody therapies to fight the virus (manufactured, animalsourced and blood-derived from people who have previously had COVID-19); 4) cell therapy products (cellular immunotherapies and other types of both autologous and allogeneic cells, such as stem cells, and related products); and 5) gene therapy products that modify or manipulate the expression of a gene or alter the biological properties of living cells for therapeutic use. This diversity of therapeutic approaches is important as it allows opportunities for all types of treatments to be discovered, whilst expanding our understanding of the disease. Bearing in mind the timescale to discover a therapy for any given disease, the scientific community has made major advances towards developing a COVID-19 therapy. We eagerly await the output of various clinical trials over the next few months with the expectation that some of these yield sufficiently efficacious and safe therapies that gain approval by the regulators.

As the global COVID-19 situation is rapidly changing, it is advisable to stay abreast with the latest news only from reputable sources (see suggested reading below).

Sheraz Gul is an expert in drug discovery with experience gained in academia (University of London), industry (GlaxoSmithKline Pharmaceuticals) and the largest applied research organisation in Europe (Fraunhofer Institute). He is also an adjunct lecturer at NUI-Galway, Ireland and scientific co-founder of Transcriptogen Ltd. He has co-ordinated work packages in drug discovery projects, which have attracted more than 7 million funding and has organised 42 drug discovery workshops since 2011 across the globe and trained 780 scientists

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COVID-19 therapies in the pipeline - Drug Target Review

The Jammu-based Indian Institute of Integrative Medicine (IIIM) is undertaking clinical trials of 3-4 formulations for developing a COVID-19 drug, a senior official had said. The IIIM is also in the final stages of validating a new machine-less coronavirus diagnostics kit, as already reported by the PTI, which can help the country scale up COVID-19 testing.

"For Covid-19, we are undergoing clinical trials. In collaboration with Ayush ministry and industry, we are involved in it. Three to four clinical trials are going in different plant species with regard to COVID-19 drugs on 3 to 4 formulations, Director CSIR-Indian Institute of Integrative Medicine (IIIM), Dr D Srinivasa Reddy, told PTI. If they (all the requisite trials) are successful, we can make medicines soon available, Reddy said.

"We are definitely getting closer. So many research groups from across the world are giving their best to find treatment for COVID-19. Discovering new medicines is a very long and costly process, he said. The director said that repurposing already known drugs to treat COVID-19 patients is the best option under the present circumstances.

"Several academic and industry groups across the globe are continuously working. In India, in particular the CSIR (Council of Scientific and Industrial Research) is a frontrunner in this direction, he added.

Dr Reddy, who recently took over as the director of IIIM for the next six years, said the first activity that IIIM undertook under him was testing COVID-19 samples.

"We started testing in the first week of April, in collaboration with Government Medical College (GMC), Jammu. We have completed over 40,000 samples till date, he said. We are in the process of increasing the number of samples tested, he said.

The IIIM is also in the process of developing a new formulation based on Zinc Gluconate and natural Vitamin C coming from Acerola Cherry for boosting immunity, he said. It is in collaborations with a company.

He said that the development processes for Active pharmaceutical ingredients (API)as part of repurposing of drugs is underway and our scientists have made significant progress on this activity and one of the processes has been demonstrated to an industry partner in Jammu.

"We continue to work along these lines and start some new initiatives to address COVID-19 related problems. Our scientists and students rose to the occasion and contributed significantly in a short time, he added.

Dr Reddy said that the IIIM laboratory is a unique place for discovering medicines based on natural products everything is under one roof for plant-based or new chemical entity (NCE)-based drugs. It has got rich biodiversity in the region which is known for medicinal and aromatic plants. It has a diverse scientist pool with expertise and experience from various functions. I see a lot of opportunities here, he added. He said that IIIM can lead programmes of national importance in addition to existing assets and expand compound or natural product extracts library and open it to others research purposes.

The IIIM can develop agricultural technologies and commercial cultivation in the Western Himalayas Kashmir Valley and Ladakh regions, he said. There are high-value medicinal and aromatic plants, (but) they seem to be facing problems in the supply chain, in particular, for the international markets. The IIIM can put more efforts in that direction, he added.

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Institute of Integrative Medicine Conducting Clinical Trials of 3-4 Covid-19 Drug Formulations: Director - Yahoo India News

Western medicine, also known as traditional medicine, is the familiar system in which healthcare providers treat symptoms and diseases using drugs, radiation and/or surgery. Complementary and alternative medicine (CAM) is a term used for medical products and practices that are typically not part of traditional medicine or included in standard medical care. The terms complementary and alternative both refer to treatments like herbal remedies or acupuncture. However, complementary medicine is when these therapies are used along with traditional western medicine practices. Alternative medicine refers to using alternative approaches instead of using traditional western medical approaches. Some practices of CAM include massage therapy, acupuncture, acupressure, Tai Chi, aromatherapy, herbal medicine and chiropractic.

When it comes to CAM, there are four major alternative medical systems that were developed by the National Center for Complementary and Alternative Medicine (NCCAM) in 2000. These four alternative medical systems include:

1. Mind-body interventions: involve using specific techniques to boost the minds capacity to influence bodily function and enhance health (i.e. meditation and yoga)

2. Biologically-based treatment: the use of substances found in nature (i.e. herbs, foods and vitamins)

3. Manipulative and body-based methods: focuses on applying specific treatments to address health issues (i.e. reflexology and chiropractic)

4. Energy therapies: based on the idea that energy fields surround and penetrate the human body (i.e. therapeutic touch and Reiki)

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As you can see from the four major alternative medical systems, CAM is used for physical, mental and spiritual health. In addition, CAM is widely used today and is increasing in popularity. In the United States, complementary and alternative medicine is used by about 38% of adults and 12% of children, according to John Hopkins Medical.

If you are interested in integrating or learning more about CAM, ask your primary care provider if integrated therapy is right for you. You can also ask your primary care provider if they can provide you with recommendations and/or contact your local hospital or medical school as they often keep lists of integrative medicine practitioners in the area.

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In recent years, the popularity of aromatherapy has significantly increased, especially after celebrities like "Grey's Anatomy" star Ellen Pompeo and "Sister, Sister" actress Tamera Mowry admitted that essential oils were included in their routines.

As Elizabeth Ko, medical director of the UCLA Health Integrative Medicine Collaborative, explained, essential oils carry the "essence" of the plant, and our smell receptors quickly absorb it.

AROMATHERAPY AND ESSENTIAL OILS

Depending on the plant you're using, the effect is different. Most commonly, they range from having anxiolytic or anti-inflammatory properties to boosting your energy and preventing acne.

It is worth noting that the best way to use essential oils is with a diffuser, which is a device that you have to fill with water and add a couple of drops of your favorite essential oil.

Celebrities like Kerry Washington ("Scandal"), Gwyneth Paltrow ("Shakespeare in Love"), and Jenna Dewan ("Soundtrack") have been open about their use of aromatherapy, so after doing our research, we found some of the best essential oils on the market.

CHAMOMILE

This herb has been consumed for hundreds of years by different cultures as a natural remedy for a variety of health conditions, so it was only natural to have it as an essential oil.

Chamomile oil is recommended for people who want to go to sleep without struggling so much, but it is also a good option for sore muscles and to reduce swelling on the skin.

ORANGE

Since orange oils are extracted from sweet oranges, it is one of the most affordable essential oils on the market. If that wasn't enough, it has an anxiolytic effect on people due to its sweet aroma, making it one of the best essential oils for anxiety.

Apart from that, it can also reduce some symptoms of PTSD. It is one of Ellen Pompeo's favorites ("Grey's Anatomy"), so that should give you a good idea of its benefits.

CINNAMON

While cinnamon is commonly used as a spice to give your food that extra touch of quality, it can also be breathed in as an essential oil to boost your focus, as it can impact the area of your brain that governs alertness.

"Soundtrack" star Jenna Dewan confessed that she boosts her immune system by putting Thieves oil, which combines clove, lemon, cinnamon, eucalyptus, and rosemary essential oils, under her tongue.

LAVENDER

Lavender is one of the best essential oils for sleep on the market as investigations have shown that people wake up more refreshed. Apart from that, it is used in dermatology to heal wounds quickly, ease the consequences of insect bites, and reduce redness.

"Sister, Sister" star Tamera Mowry recommends lavender oils to get rid of "a pesky migraine" due to its anxiolytic effect, so this one is a no-brainer.

YLANG YLANG

Due to its sweet floral scent, ylang ylang essential oil is mostly recommended as a soothing option, especially after investigators found that it helped lower people's heart rate and blood pressure.

If that wasn't good enough, this oil is reported to lift people's moodsand their self-esteem while also helping with inflammation.

TEA TREE

Also known as melaleuca, tea tree oil has antibacterial and antifungal properties, which is probably why Australian aboriginal people used it for wound healing in the past.

Nowadays, it is mostly recommended to prevent and treat acne and to help with your energy levels, but it can also be used to repel bugs.

ROSE

One of the best smelling essential oils out there is rose oil, which is reported to relieve pain, including the unsettling menstrual cramps. Additionally, it has antibacterial and antifungal properties, which makes it an excellent option to prevent fungal and staph infections.

If you are like Ellen Pompeo and don't like too-flowery scents, you can combine rose oils with "a dash" of vetiver or sandalwood to give it "a little earthiness."

The information in this article is not intended or implied to be a substitute for professional medical advice, diagnosis or treatment. All content, including text, and images contained on, or available through this NEWS.AMOMAMA.COM is for general information purposes only. NEWS.AMOMAMA.COM does not take responsibility for any action taken as a result of reading this article. Before undertaking any course of treatment please consult with your healthcare provider.

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Best Essential Oils: How to Choose the Proper One and Use the Power of Nature - AmoMama