Stem Cell Clinic

Precision for Medicine and Akoya Partner to Develop Advanced Liquid Biopsy and Tissue Biomarker Tests – Yahoo Finance

MENLO PARK, Calif. & HOUSTON--(BUSINESS WIRE)--

Akoya Biosciences, The Spatial Biology Company today announced a strategic alliance with Precision for Medicine (formerly ApoCell), a pioneer in customized biomarker solutions, to develop proprietary liquid biopsy and tissue biomarker tests using Akoyas Vectra Polaris System. By combining the Vectra Polaris system with Precision for Medicines ApoStream technology, the collaboration is designed to advance immuno-oncology candidates in clinical trials using both tumor biopsies and liquid biopsies to generate data required for assessing drug efficacy and validation of companion diagnostics (CDx).

We are very excited to be the first commercial laboratory to launch the Vectra Polaris system for image analysis of circulating tumor cells following ApoStreams isolation and enrichment functionality, said Darren Davis, PhD, Precision for Medicine Senior Vice President. Together, these technologies will enable accelerated drug development through detection of biomarkers in liquid biopsies. These tools have enabled our scientists to better understand the biological correlation of circulating metastatic cancer cells with cancer cells present in the tumor tissue.

Circulating tumor cells (CTCs) have long been known to exist in cancer patients blood. However, the promise of clinical application of CTCs as a liquid biopsy has not born fruit because these cells are difficult to detect using current molecular biology techniques. Precision for Medicines proprietary ApoStream technology captures significant quantities of rare circulating cancer cells from whole blood for characterization using Akoyas Opal technology and Vectra Polaris imaging platform. ApoStream has been used in more than 80 clinical trials, including several ongoing phase III studies for CDx development.

Akoyas Vectra Polaris Automated Quantitative Pathology System, part of the companys Phenoptics 2.0 next-generation biomarker multiplexing platform, enables researchers to gain a deeper level of understanding of disease mechanisms related to new cancer immunotherapy approaches. The Vectra Polaris system integrates high throughput, seven-color multispectral imaging with whole-slide scanning in a simplified digital pathology workflow to support the quantification and analysis of tissue sections discernible with Opal detection kits. A recent meta-analysis of several studies showed that incorporating spatial information using Akoyas multiplexed immunofluorescence technology is important for improving the predictive accuracy of immuno-oncology biomarkers.

The Phenoptics portfolio is capable of interrogating multiple protein markers on any tissue or cytology slide preparation. Precision for Medicine has validated several immune biomarker panels across various therapeutic applications using both types of preparation. These immune panels are currently being used to monitor immune cell infiltration including cancer, psoriasis, lupus and atomic dermatitis.

The Vectra Polaris imaging system offers an innovative approach to assessing immunotherapy candidates in translational and clinical research, said Dr. Cliff Hoyt, Akoya Vice President of Translational and Scientific Affairs. We are pleased to see this technology paired with ApoStream for a comprehensive method of imaging CTCs and improving our understanding of disease mechanisms in cancer.

For more information on ApoCell and the ApoStream technology, please click here.

About Precision for Medicine

Precision for Medicine is the first biomarker-driven clinical research services organization supporting life sciences companies in the use of biomarkers essential to targeting patient treatments more precisely and effectively. Precision applies novel biomarker approaches to clinical research that take advantage of the latest advancements in science and technology, focusing predominantly on genomics, immune-response assays, global specimen logistics, biomarker analytics, companion diagnostics, and clinical trial execution. Precision for Medicine is part of Precision Medicine Group, with more than 1,900 employees in 34 locations in the US, Canada, and Europe. For more information, visit http://www.Precision for Medicine.com.

About Akoya

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Akoya Biosciences, The Spatial Biology Company, offers the most comprehensive, end-to-end solutions for high-parameter tissue analysis from discovery through clinical and translational research, enabling the development of more precise therapies for immuno-oncology and other drug development applications. The company has two industry-leading platforms that empower investigators and researchers to gain a deeper understanding of complex diseases such as cancer, and other immune system or neurological disorders. The CODEX system is the only benchtop platform that can efficiently quantify more than 40 biomarkers and is ideally suited for biomarker discovery. The Phenoptics platform is the only end-to-end multiplexed immunofluorescence solution with the robustness and high throughput necessary for translational research and clinical trials. For more information, please visit https://www.akoyabio.com/.

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Precision for Medicine and Akoya Partner to Develop Advanced Liquid Biopsy and Tissue Biomarker Tests - Yahoo Finance

Tmunity and Oncora Medical Partner to Advance the Use of Real-World Data to Accelerate the Availability of CAR-T Therapies for Cancer Patients -…

PHILADELPHIA--(BUSINESS WIRE)--Tmunity Therapeutics, Inc., a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy, today announced it has entered into a strategic collaboration with Oncora Medical to access Oncoras proprietary real-world data platform to support the design of future clinical programs with cell therapy.

Oncora brings to this collaboration a suite of intuitive analytics software that collects and deploys real-world clinical data that can be used to drive better decision making and treatment options and ultimately improve patient outcomes. Tmunity brings expertise in clinical oncology and clinical trial design, regulatory strategy and cell therapy. Together, this collaboration has the potential to innovate the way clinical trials for cell therapy are executed to bring treatments to patients more efficiently. Tmunity will leverage Oncoras data and analytics capabilities to refine, customize and validate a real-world evidence strategy. Tmunity hopes to validate the strategy in a clinical program selected from its highly innovative pipeline.

Partnering with Oncora Medical to utilize real-world data in clinical development puts Tmunity at the forefront of innovation. This project has the potential to help us more effectively design and execute our clinical trials to advance the next-generation of CAR-T therapies, said Usman Oz Azam, MD, President and Chief Executive Officer of Tmunity. We are excited to begin our relationship with Oncora Medical to revolutionize the way clinical trials are designed with the ultimate goal of reducing the time to deliver targeted T-cell therapies to patients in need.

We believe that use of real-world data and evidence has the potential to change the way cancer therapies are developed and to further personalize outcome predictions that will guide treatment decisions, said Oncora Medical co-founder and CEO, David Lindsay. We hope our partnership with Tmunity will innovate the way clinical trials are designed and thereby, to bring important cancer treatments to patients more efficiently.

About Tmunity Therapeutics

Tmunity is a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy to patients with devastating diseases. Integrating a foundational collaboration with the University of Pennsylvania (Penn) with the groundbreaking scientific, clinical, and manufacturing expertise, and the demonstrated track record of its founders (Carl June, MD; Bruce Blazar, MD; Bruce Levine, PhD; Yangbing Zhao, MD, PhD; Jim Riley, PhD; and Anne Chew, PhD), Tmunity represents a new center of gravity in translational T-cell medicine. Through the University of Pennsylvania, the Parker Institute for Cancer Immunotherapy and collaborations with the University of California San Francisco and Childrens Hospital of Philadelphia, the company is developing a diversified portfolio of novel treatments that exhibit best-in-class control over T-cell activation and direction in the body, with a focus in cancer and three programs currently in clinic development. With headquarters in Philadelphia, Tmunity utilizes laboratories and production facilities at Penn and its own dedicated cGMP manufacturing facility in East Norriton, PA, to pursue process improvement and production scale-up in support of clinical development of its T-cell therapies. For more information, visit http://www.tmunity.com and connect with us on social media at @TmunityTx and LinkedIn.

About Oncora Medical

Oncora is a privately held oncology analytics company in Philadelphia, PA, building next generation software capable of analyzing diverse healthcare data and applying advanced machine learning techniques to produce valuable clinical insights. Oncora leverages its unique data and analytic capabilities to support clinical development for research partners and biopharmaceutical companies. Oncoras platform is currently being used by physicians to achieve more personalized treatment plans for cancer patients. For more information on how Oncora is fighting cancer with data, visit oncoramedical.com and connect with us on social media at @oncoramed and LinkedIn.

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Tmunity and Oncora Medical Partner to Advance the Use of Real-World Data to Accelerate the Availability of CAR-T Therapies for Cancer Patients -...

Notch Therapeutics a new company with a revolutionary allogeneic ("off the shelf") T cell technology – GlobeNewswire

Toronto, ON, Nov. 05, 2019 (GLOBE NEWSWIRE) -- Notch Therapeutics, a company in the emerging field of gene-modified T cell therapy, has been created to commercialize a revolutionary technology that creates allogeneic (donor) gene-edited T cells from stem cells on an industrial scale, efficiently making T cell therapies that are clinically robust and of a consistently high quality. Notch is actively pursuing industry partnerships.

The founders of Notch - Sunnybrook Health Sciences Centre, University of Toronto (UoT), Toronto Innovation Acceleration Partners (TIAP) and CCRM, with Lumira Ventures as an additional investor - created Notch to provide a vehicle through which to further develop and bring to market the combined pioneering research, nearly ten years underway, from the labs of Drs. Juan CarlosZiga-Pflcker,Senior Scientistin Biological Sciences atSunnybrook,and Chair of the Department of Immunology atUoT;andof Peter Zandstra,Professor, Institute of Biomaterials and Biomedical Engineering at UoTandDirector, School of Biomedical engineering and Michael Smith Laboratories at the University of British Columbia.

"This technology is very promising and might be used to create therapiesto treatsome of our greatest medical challenges,like cancer, autoimmune diseases and organ transplant rejection. Its also the first and only method that can reconstitute immune systems," says Dr. Andy Smith, President and CEO of Sunnybrook Health Sciences Centre. "Finally having optionstotarget these high-impact areasfor our patientsis what we mean when we say we are inventing the future of health care."

Jennifer Fraser, Director Innovations at the University of Toronto, comments that "Dr. Ziga-Pflckers allogeneic T cell therapy was one of the first projects I worked on when I joined UoT. Its very gratifying to see the technology move toward the clinic."

Even as the field for these therapies grows rapidly, major challenges have until now prevented wider adoption mainly due to a slow and expensive manufacturing process which yielded variable results. The Notch technology, however, shows promise for surmounting these issues cost-effectively and reliably. Notch, having been incubated at CCRM, will be able to leverage its in-house process development expertise and Good Manufacturing Practices (GMP) facility, located in downtown Toronto. It offersuniversally enhanced T cell therapies against high-impact diseases, using stem cells as a renewable source to expand treatment options and deliver cost-effective immunotherapies to patients. The aim of Notch is to generate T cells from multiple sources of stem cells and provide a platform for research and development, and a better way of manufacturing T cells and their applications for treating cancers or immune deficiencies.

"TIAP is pleased that, after many years of nurturing this research and investing in the risky early-stages with our co-founders, we are now seeing a truly ground-breaking new health science technology make its way through development in a very encouraging way. This is yet another example of what can be done through TIAPs unique ability within the community to bundle technologies across multiple institutions. This is a true collaboration which has resulted in development of a technology that will have significant impact", says Dr. Rafi Hofstein, President & CEO of TIAP.

"Notch Therapeutics is a star pupil in CCRMs incubation program," says Dr. Michael May, President and CEO of CCRM. "By de-risking the technology and designing Proof of Concept studies to appeal to investors, attracting experienced start-up management, and working with our ecosystem partners, we have collectively given Notch every opportunity to succeed."

Dr. Benjamin Rovinski, Managing Director of Lumira Ventures, states: "Early on, our team recognizedNotchs novel and differentiated platform and its potential to produce safer, more effective, and scalable allogeneic T cell therapies. We are pleased to be part of the financing syndicate. The quality and breadth of science coming out of Canadian universities is phenomenal, and important innovators like Notch are able to access capital, knowledge and the engaged support of Lumira and other investors, to enable the development and commercialization of their technology. We are excited to work with the entire Notch team."

About TIAP

TIAP is a leading provider of venture building services, early-stage funding, and deal-brokering with industry and private investors. As a member-based organization made up of 14 member institutions including University of Toronto and affiliated teaching hospitals TIAPs mandate is to drive the commercialization of their most promising research breakthroughs. TIAP has an active portfolio of more than 60 companies in sectors such as therapeutics, medical devices and IT/AI, which have raised in excess of CDN$300M from global investors, and has created more than 1000 direct/indirect jobs.

For more information, please visit http://www.tiap.ca

About University of TorontoFounded in 1827, the University of Toronto is Canadas leading institution of learning, discovery and knowledge creation. U of T is one of the worlds top research-intensive universities, driven to invent and innovate. It is also one of the top five universities in the world for its start-up incubator programs. In the last 10 years, the U of T entrepreneurship community has created over 500 companies and raised over $1.5 billion in investment capital. http://www.utoronto.ca

About Sunnybrook Health Sciences CentreSunnybrook Health Sciences Centre is inventing the future of health care for the 1.3 million patients the hospital cares for each year through the dedication of its more than 10,000 staff and volunteers. An internationally recognized leader in research and education and a full affiliation with the University of Toronto distinguishes Sunnybrook as one of Canadas premier academic health sciences centres. Sunnybrook specializes in caring for high-risk pregnancies, critically-ill newborns and adults, offering specialized rehabilitation and treating and preventing cancer, cardiovascular disease, neurological and psychiatric disorders, orthopaedic and arthritic conditions and traumatic injuries. The Hospital also has a unique and national leading program for the care of Canadas war veterans. For more information about how Sunnybrook is inventing the future of health care. Please visit us online at http://www.sunnybrook.ca

About CCRMCCRM, a Canadian not-for-profit organization funded by the Government of Canada, the Province of Ontario, and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, dedicated funding, and unique infrastructure. CCRM is the commercialization partner of the Ontario Institute for Regenerative Medicine and the University of Torontos Medicine by Design. CCRM is hosted by the University of Toronto. Visit us atccrm.ca.

About Lumira VenturesLumira Ventures isCanadasleading and most active healthcare venture capital firm. Lumira invests in best-in-class North American companies developing innovative therapeutics and medical technologies whose products offer transformative improvements to patient health outcomes and provide meaningful reductions to the overall cost of healthcare delivery. Since inception, Lumiras portfolio companies have brought 50+ new therapies to market, impacting the lives of 1+ billion patients globally, generating $65+ billion in cumulative revenue.

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

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Notch Therapeutics a new company with a revolutionary allogeneic ("off the shelf") T cell technology - GlobeNewswire

BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update – Yahoo Finance

NEW YORK, Nov. 05, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2019, and provide a corporate update, at 8:00 a.m., Eastern Standard Time, on Thursday, November 14, 2019.

BrainStorms President & CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, and Preetam Shah, PhD, Chief Financial Officer.

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., Eastern Standard Time, Tuesday, November 12.

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

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

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (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. 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 U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began 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 (U.S. FDA) and the European Medicines Agency (EMA) in 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. 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 causeBrainStorm Cell Therapeutics Inc.'sactual 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, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms 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 BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms 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 athttp://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.

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BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update - Yahoo Finance

UPDATED: Andy Plump teams with MD Anderson on the race to cell therapy 2.0 and they’re already planning a pivotal – Endpoints News

Usually a short attack takes months to play out. For Aaron Wedlunds latest all-out assault, well likely know pretty much whether hes right or wrong about a looming biotech stock disaster in 4 days.

Thats the deadline for FibroGen and AstraZeneca the big US licensee to get into the details on their pooled safety analysis of roxadustat, an anemia drug that has been a central focus for investors for years now. Positive efficacy data have been rewarded with a $3.5 billion market cap.

Wedlund, who handled Sahm Adrangis short thesis biotech analyses at Kerrisdale for a number of years before going off on his own, is betting that roxadustat will be revealed as a clear loser when researchers unveil the hard numbers on the pivotal safety data on major cardio events, or MACE, compared to placebo and EPO.

Essentially, he tells me, if the MACE is higher (on roxa) then this drug is dead. And up to now he claims the sponsors have only been able to manage concerns about safety by manipulating the results which regulators wont approve of.

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UPDATED: Andy Plump teams with MD Anderson on the race to cell therapy 2.0 and they're already planning a pivotal - Endpoints News

Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to…

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTx), a development-stage biotechnology company leading the field of RNA-editing gene therapy, announces $35.5M in Series A financing, led by New Enterprise Associates (NEA), with additional participation from CureDuchenne Ventures. The new capital will enable the company to extend its growing portfolio of intellectual property, recruit and hire top scientific talent and advance its groundbreaking RNA and protein targeting platforms focused on curing human diseases.

These platforms include the proprietary ShapeTx RNAfix technology that enables direct in vivo targeting and modification of RNA by leveraging proteins such as Adenosine Deaminases Acting on RNA (ADARs), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix platform differentiates from other contemporary genome engineering technologies by engaging natural human cellular machinery to modify RNA.

ShapeTx was founded on the work of Dr. Prashant Mali, Assistant Professor of Bioengineering at UCSD, who during his postdoctoral fellowship in the George Church laboratory at Harvard Medical School pioneered the use of CRISPR in human cells. ShapeTx RNAfix platform is built upon his lab's most recent work demonstrating in vivo use of guide RNAs to recruit native ADARs and to fix mutations in multiple rare genetic disease mouse models.

Our technology can correct mutations or target specific genes in neurodegenerative, oncology, metabolic and rare genetic disorders by hijacking naturally occurring proteins such as ADARs present in our cells using just a short guide RNA. Our proprietary new platform avoids the risk of in vivo immunogenicity and permanent off-target damages commonly associated with CRISPR-based approaches, explained Francois Vigneault, Ph.D., President and CEO, who was previously VP of Research at Juno Therapeutics after a successful co-acquisition of AbVitro, Inc. by Juno and Celgene.

Ed Mathers, Partner at NEA and Board member at ShapeTx, said, One rarely comes across a proprietary technology platform with such transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development. The team has shown us an exciting demonstration of the technology in multiple in vivo models, alongside one of the strongest IP estates we have seen in the field. NEA looks forward to backing the company in future rounds as they move the technology toward the clinic.

While the ShapeTx platform will be enabling for many other genetic diseases, Dr. Malis in vivo proof of concept in Duchenne Muscular Dystrophy was quite exciting and could potentially lead to a cure for families suffering from such a debilitating disorder, said Debra Miller, CEO and Founder of CureDuchenne and CureDuchenne Ventures.

The ShapeTx Series A financing coincides with the formation of a world-class Scientific Advisory Board comprised of foremost global experts in genomics, bioengineering, and gene editing, including George Church Ph.D., James Collins Ph.D., and Don Cleveland Ph.D. The scientific advisory board will serve as strategic advisors and ensure that the research and development of its platforms meet the highest standards of scientific merit.

Prashant and Francois are some of the most innovative and brilliant individuals that have come through my lab over the years, and it will be impressive to see these two disrupt the field of gene therapy with this paradigm-shifting technology, said Dr. George Church, Professor in Genetics at Harvard Medical School and member of the ShapeTx Scientific Advisory board.

Shape Therapeutics Scientific Advisory Board Members:

George Church, Ph.D.

George Church Ph.D., world-famous geneticist, molecular engineer, and chemist. He developed the methods used for the first genome sequence & million-fold cost reductions since, as well as pioneered many of the CRISPR advances in genome editing. He is currently a Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT). He is Director of the U.S. Department of Energy Technology Center and Director of the National Institutes of Health Center of Excellence in Genomic Science. He has received numerous awards, including the 2011 Bower Award and Prize for Achievement in Science from the Franklin Institute and election to the National Academy of Sciences and Engineering.

James Collins, Ph.D.

James Collins Ph.D., is one of the pioneers of the field of synthetic biology and has made multiple synthetic biology and bioengineering breakthroughs in biotechnology and biomedicine. He serves as the Termeer Professor of Medical Engineering & Science and Professor of Biological Engineering at MIT, as well as a member of the Harvard-MIT Health Sciences & Technology Faculty, and core member of the Wyss Institute. His many awards include a Rhodes Scholarship, a MacArthur Genius Award, a National Institutes of Health Directors Pioneer Award. Jim is also an elected member of the National Academy of Sciences, the National Academy of Engineering, the National Academy of Medicine, the American Academy of Arts & Sciences, as well as a charter fellow of the National Academy of Inventors.

Don Cleveland Ph.D.

Don Cleveland Ph.D. is an award-winning inventor and pioneer in the field of Antisense Oligonucleotide (ASO) and their uses in gene therapy. He was recently awarded the Breakthrough Prize in Life Sciences for his work on the pathogenesis of disease and ASO-mediated treatment approaches in ALS and Huntingtons disease. Don is currently Professor of Medicine and Department Chair of Cellular and Molecular Medicine and Neurosciences at the University of California at San Diego, and Head, Laboratory for Cell Biology at the San Diego branch of Ludwig Cancer Research. He has made pioneering discoveries on the mechanisms of chromosome movement and cell-cycle control during normal cellular division, as well as the principles of neuronal cell development and the relationship to defects that contribute to inherited neurodegenerative disease.

About Shape Therapeutics, Inc.

Shape Therapeutics, Inc. is creating the worlds leading RNA and protein targeting platforms focused on the cure of human diseases. These include developing precision RNA editing through proteins such as ADAR (Adenosine Deaminase Acting on RNA), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix technology allows for the editing of RNA using natural human cellular machinery, limiting the risk associated with immunogenicity, cellular toxicity, or off-target DNA editing. The teams founders include Prashant Mali, Ph.D., Francois Vigneault, Ph.D., and John Suliman. ShapeTx is headquartered in Seattle, Washington, with a satellite site opening in Cambridge, Massachusetts. For additional information, visit http://www.ShapeTx.com.

About NEA

New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With more than $20 billion in cumulative committed capital since the firm's founding in 1978, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 225 portfolio company IPOs and more than 375 acquisitions. For additional information, visit http://www.nea.com.

About CureDuchenne Ventures

CureDuchenne Ventures supports Duchenne research by using philanthropic donations to encourage the development of new Duchenne drugs. Through an impact financing model, we can provide equity or royalty financing to biotech and pharmaceutical companies. CureDuchennes portfolio includes 16 wide-ranging projects with several successful exits. Investments from CureDuchenne Ventures have successfully de-risked and leveraged more than $2.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne. For additional information, visit https://www.cureduchenne.org/ventures/.

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Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to...

Prediction of off-target specificity and cell-specific fitness of CRISPR-Cas System using attention boosted deep learning and network-based gene…

CRISPR-Cas is a powerful genome editing technology and has a great potential forin vivo gene therapy. Successful translational application of CRISPR-Cas to biomedicine still faces many safety concerns, including off-target side effect, cell fitness problem after CRISPR-Cas treatment, and on-target genome editing side effect in undesired tissues. To solve these issues, it is needed to design sgRNA with high cell-specific efficacy and specificity. Existing single-guide RNA (sgRNA) design tools mainly depend on a sgRNA sequence and the local information of the targeted genome, thus are not sufficient to account for the difference in the cellular response of the same gene in different cell types. To incorporate cell-specific information into the sgRNA design, we develop novel interpretable machine learning models, which integrate features learned from advanced transformer-based deep neural network with cell-specific gene property derived from biological network and gene expression profile, for the prediction of CRISPR-Cas9 and CRISPR-Cas12a efficacy and specificity. In benchmark studies, our models significantly outperform state-of-the-art algorithms. Furthermore, we find that the network-based gene property is critical for the prediction of cell-specific post-treatment cellular response. Our results suggest that the design of efficient and safe CRISPR-Cas needs to consider cell-specific information of genes. Our findings may bolster developing more accurate predictive models of CRISPR-Cas across a broad spectrum of biological conditions as well as provide new insight into developing efficient and safe CRISPR-based gene therapy.

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Prediction of off-target specificity and cell-specific fitness of CRISPR-Cas System using attention boosted deep learning and network-based gene...