Latest news with #AAV-based
Yahoo
21-05-2025
- Business
- Yahoo
Coave Therapeutics Establishes New Scientific Advisory Board to Accelerate Innovation in Genetic Medicines for Eye and CNS Diseases
Paris, France – May 21, 2025 – Coave Therapeutics ('Coave'), a company pioneering the future of genetic medicines, today announces the evolution of its Scientific Advisory Board (SAB). This renewed SAB reflects Coave's strategic shift from a platform-focused approach to the development of a therapeutic pipeline, as the Company advances multiple gene therapy programs. The SAB brings together internationally recognized leaders in adeno-associated viral (AAV) vector biology, gene therapy development for ophthalmology and central nervous system (CNS) diseases, and innovative approaches to extra-hepatic gene delivery. The SAB members include: Olivier Danos, PhD (Chair) – Chief Scientific Officer at REGENXBIO (Rockville, MD, USA) Dr. Danos is a pioneer in gene therapy with over 30 years of academic and industrial leadership, including roles at Biogen, Genethon and University College London, and now leads REGENXBIO's scientific strategy Robin Ali, PhD – Professor of Human Molecular Genetics at King's College London (London, UK) and Founder of MeiraGTx Dr. Ali is a world-leading expert in gene therapy for retinal diseases and a pioneer of translational research advancing vision restoration therapies Aravind Asokan, PhD – Professor at Duke University School of Medicine and Director of the Danaher Beacon for Gene Therapy Innovation (Durham, NC, USA) Dr. Asokan specializes in AAV vector biology and synthetic virology, developing engineered AAVs to enable next-generation gene therapies Juliette Hordeaux, DVM, PhD – Chief Scientific Officer at GEMMA Biotherapeutics (Philadelphia, PA, USA) and formerly Executive Director of Translational Research at the Gene Therapy Program, University of Pennsylvania (Philadelphia, PA, USA) Dr. Hordeaux is a recognized leader in the development of AAV-based CNS-targeted gene therapies, focused on translating preclinical innovations to clinical-stage programs Federico Mingozzi, PhD – Chief Executive Officer of Nava Therapeutics (Philadelphia, PA, USA) Dr. Mingozzi brings extensive expertise in gene therapy R&D and translation, having previously served as Chief Scientific Officer at Spark Therapeutics and earlier led programs at Genethon, INSERM and the Children's Hospital of Philadelphia Amy Pooler, PhD – Senior Vice President, Research and Development at Life Edit Therapeutics (Durham, NC, USA), previously Vice President, Head of Research at Sangamo Therapeutics (Brisbane, CA, USA) Dr. Pooler has extensive experience in translational neuroscience and genetic medicine, with leadership roles in developing CNS-targeted therapies at Sangamo and Life Edit The SAB members will provide strategic scientific and clinical guidance to support Coave's R&D initiatives, leveraging its ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform to unlock improved precision and safety in genetic medicine. Their combined expertise strengthens Coave's leadership in developing AAV-based therapeutics targeting diseases beyond the liver, including the eye and CNS. Lolita Petit, CSO of Coave Therapeutics commented, 'We are absolutely delighted to welcome this exceptional group of world-leading experts to our Scientific Advisory Board. Their collective expertise across AAV biology, gene therapy development and translational research will be invaluable as we continue to enhance our ALIGATER™ platform and advance our therapeutic pipeline. Their support reinforces our commitment to delivering next-generation genetic medicines with improved precision and safety and accelerates our mission to transform the lives of patients with serious unmet medical needs.' *** Photos of SAB members available on request About ALIGATER™Coave's proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform is a breakthrough technology addressing key limitations in the delivery of genetic payloads to extra-hepatic tissues, including limited tissue specificity, delivery efficiency and safety. ALIGATER™ enables conjugation of targeting ligands, such as small molecules, peptides, or antibody fragments, on AAV or non-viral vectors, offering superior delivery efficiency, tissue specificity and safety profile for a broad range of diseases. Importantly, the platform streamlines the manufacturing process by avoiding prior AAV capsid modifications. These capabilities will enable Coave to develop best-in-class gene therapies designed for specific indications. About Coave Therapeutics Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases. Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit and follow us on LinkedIn. CONTACTS Coave Therapeutics Rodolphe Clerval, CEO contact@ MEDiSTRAVA Sylvie Berrebi, Mark Swallow coavetx@ in to access your portfolio
Business Wire
16-05-2025
- Business
- Business Wire
JCR Pharmaceuticals Presents Preclinical Gene Therapy Data that Demonstrate Promising CNS Uptake at American Society of Gene and Cell Therapy
HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; 'JCR') announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs at the American Society of Gene and Cell Therapy (ASGCT) 28 th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. In an oral presentation, the JCR researcher reported that the Company's proprietary J-Brain Cargo ® (JBC) technology enables the efficient delivery of an adeno-associated virus (AAV) gene therapy across the blood-brain barrier (BBB) and into the central nervous system (CNS) in mice, monkeys, and several animal models of CNS diseases. We have successfully developed JUST-AAV, a novel AAV vector platform technology designed to enhance targeted delivery and reduce liver tropism, thereby improving safety and efficacy of AAV-based gene delivery technologies to the CNS. JUST-AAV encompasses a range of vector types optimized for various target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. This proprietary technology holds significant promise for advancing the field of AAV-based gene therapy. 'These findings represent a significant advancement toward new treatments for previously challenging CNS diseases,' said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR Pharmaceuticals. 'Our preclinical data demonstrate that our brain-targeting JUST-AAV technology delivers therapeutic agents to the central nervous system far more efficiently than conventional AAV9, while significantly reducing liver accumulation. This enhanced safety profile greatly increases the potential for clinical translation. This research is a crucial step in our ongoing commitment to developing innovative solutions for unmet medical needs.' JCR showcased the following presentation: Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism Presenter: Yuhei Ashida (JCR Pharmaceuticals) Researchers successfully created a brain-targeting AAV vector (brain-targeting JUST-AAV) by incorporating a miniaturized antibody that binds to the transferrin receptor, into the AAV capsid. Furthermore, unique modifications to the AAV capsid sequence significantly reduced AAV vector accumulation in the liver, a known source of adverse effects. In mouse studies, the JUST-AAV vector achieved 77-fold higher expression of green fluorescent protein (GFP) in the brain compared to AAV9, while reducing the tropism to the liver by 99%. To further enhance brain targeting and BBB permeability, the researchers incorporated additional molecules that bind to receptors other than the transferrin receptor into the AAV capsid. In monkey studies, this bispecific vector demonstrated an improved gene delivery efficiency to the brain by several orders of magnitude compared with AAV9, while reducing infection in potentially problematic tissues such as the liver and dorsal root ganglia by more than 90%. Application of JUST-AAV to a mouse model of neuronal ceroid lipofuscinosis resulted in the disappearance of symptoms such as seizures and prolonged lifespan to an extent of a functional cure. These results suggest that the newly developed JUST-AAV technology offers the potential for safer and more efficient gene therapy than conventional gene delivery vectors. About the American Society of Gene and Cell Therapy (ASGCT) The American Society of Gene and Cell Therapy (ASGCT) is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. The mission of the ASGCT is to advance knowledge, awareness, and education, leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. For more information, please visit www. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. JUST-AAV JUST-AAV is a proprietary platform technology that utilizes modified adeno-associated virus (AAV) vectors. The technology entails insertion of miniaturized antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery to those tissues and organs. Further capsid modifications minimize off-target effects and improve safety. The name is derived from ' J CR' ' U ltimate destination of organ' ' S afeguarding against off-target delivery' and ' T ransformative technology' reflecting its potential for broad application across various diseases. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as 'believe,' 'estimate,' 'anticipate,' 'intend,' 'plan,' 'will,' 'would,' 'target' and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
Yahoo
12-05-2025
- Business
- Yahoo
Siren Biotechnology to Present Universal AAV Immuno-Gene Therapy Data at the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
SAN FRANCISCO, May 12, 2025 (GLOBE NEWSWIRE) -- Siren Biotechnology Inc., pioneers of Universal AAV Immuno-Gene Therapy for Cancer, today announced that it will present new scientific data at the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place May 13–17 in New Orleans, LA. The company's presentations include one oral session and two poster presentations that highlight recent scientific advances and underscore the therapeutic potential of Siren's AAV-based immuno-gene therapy platform across solid tumor indications. Presentation details: Oral Presentation: SRN-101 Universal AAV Gene Therapy for Solid Tumors. Dr. Nicole K. Paulk, CEO, Founder, President, Siren Biotechnology May 17, 2025 | 8:00am CST Poster Presentations: Vectorized Interferons Provide a Robust and Sustained Transcriptional Response Over Traditional Non-Vectorized Recombinant Cytokine Therapies in Human High-Grade Glioma Cells. Justin Lee, BS, Preclinical Associate Scientist May 14, 2025 | 5:30 – 7:00pm CST Identification of Pharmacologically Relevant Species for IND-Enabling Toxicology Studies by Deep Comparative Transcriptomic Profiling of PBMCs Treated with Recombinant Human Payloads. Christopher Lovejoy, MS, Preclinical Associate Scientist May 14, 2025 |5:30 – 7:00pm CST 'We're excited to share our latest data with the scientific community at ASGCT, which remains the most prestigious venue for showcasing gene therapy innovations,' said Dr. Nicole K. Paulk, PhD, Siren Biotechnology's CEO and Founder. 'These presentations reflect our continued progress advancing our Universal AAV Immuno-Gene Therapy Platform and our commitment to developing transformative therapies to treat solid tumor cancers.' About Siren Biotechnology Headquartered in San Francisco, CA, Siren Biotechnology is sounding the alarm against cancer. We are the pioneers of Universal AAV Immuno-Gene Therapy, which combines the promise of two transformative therapeutic technologies, AAV gene therapy and cytokine immunotherapy, into a single modality which we believe will redefine how we destroy tumor cells and elicit anti-tumor immunity. Our vision is for Universal AAV Immuno-Gene Therapy to become the standard of care for any solid tumor cancer. To learn more, visit and follow us on LinkedIn, Twitter, Facebook and Instagram. Universal AAV Immuno-Gene Therapy for Cancer. It's Here. CONTACT: Contact Akela Kuwahara press@
Yahoo
12-05-2025
- Business
- Yahoo
Siren Biotechnology to Present Universal AAV Immuno-Gene Therapy Data at the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
SAN FRANCISCO, May 12, 2025 (GLOBE NEWSWIRE) -- Siren Biotechnology Inc., pioneers of Universal AAV Immuno-Gene Therapy for Cancer, today announced that it will present new scientific data at the 2025 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place May 13–17 in New Orleans, LA. The company's presentations include one oral session and two poster presentations that highlight recent scientific advances and underscore the therapeutic potential of Siren's AAV-based immuno-gene therapy platform across solid tumor indications. Presentation details: Oral Presentation: SRN-101 Universal AAV Gene Therapy for Solid Tumors. Dr. Nicole K. Paulk, CEO, Founder, President, Siren Biotechnology May 17, 2025 | 8:00am CST Poster Presentations: Vectorized Interferons Provide a Robust and Sustained Transcriptional Response Over Traditional Non-Vectorized Recombinant Cytokine Therapies in Human High-Grade Glioma Cells. Justin Lee, BS, Preclinical Associate Scientist May 14, 2025 | 5:30 – 7:00pm CST Identification of Pharmacologically Relevant Species for IND-Enabling Toxicology Studies by Deep Comparative Transcriptomic Profiling of PBMCs Treated with Recombinant Human Payloads. Christopher Lovejoy, MS, Preclinical Associate Scientist May 14, 2025 |5:30 – 7:00pm CST 'We're excited to share our latest data with the scientific community at ASGCT, which remains the most prestigious venue for showcasing gene therapy innovations,' said Dr. Nicole K. Paulk, PhD, Siren Biotechnology's CEO and Founder. 'These presentations reflect our continued progress advancing our Universal AAV Immuno-Gene Therapy Platform and our commitment to developing transformative therapies to treat solid tumor cancers.' About Siren Biotechnology Headquartered in San Francisco, CA, Siren Biotechnology is sounding the alarm against cancer. We are the pioneers of Universal AAV Immuno-Gene Therapy, which combines the promise of two transformative therapeutic technologies, AAV gene therapy and cytokine immunotherapy, into a single modality which we believe will redefine how we destroy tumor cells and elicit anti-tumor immunity. Our vision is for Universal AAV Immuno-Gene Therapy to become the standard of care for any solid tumor cancer. To learn more, visit and follow us on LinkedIn, Twitter, Facebook and Instagram. Universal AAV Immuno-Gene Therapy for Cancer. It's Here. CONTACT: Contact Akela Kuwahara press@
Yahoo
17-03-2025
- Business
- Yahoo
ChromaTan and Landmark Bio Awarded National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) Grant to Advance Intensified Recombinant Adeno-Associated Virus (AAV) Production Process
Grant allows ChromaTan and Landmark Bio to create a scalable, cost-effective, and accessible platform for the manufacturing and characterization of AAV-based gene therapy vectors Project developed with award from NIIMBL and financial assistance from the National Institute of Standards and Technology SPRING HOUSE, Pa. and WATERTOWN, Mass., March 17, 2025 /PRNewswire/ -- ChromaTan Inc., and Landmark Bio PBLLC, today announced they have been awarded a grant from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) to support the development of an intensified recombinant adeno-associated virus (AAV) production process as part of NIIMBL's Viral Vector Manufacturing and Analytics Program. The NIIMBL award will enable ChromaTan and Landmark Bio to create a scalable, cost-effective, and accessible platform for the manufacturing and characterization of AAV-based gene therapy vectors. The collaboration will leverage ChromaTan's BioRMB™ technology and Kascade instrumentation along with Landmark Bio's expertise in viral vector process development, chemistry, manufacturing and controls, and GMP manufacturing to improve purification efficiency and process intensification. "We are excited to collaborate with Landmark Bio, showcasing their capabilities together with ChromaTan's BioRMB as a next-generation purification platform for gene therapy products," said Oleg Shinkazh, chief executive officer of ChromaTan. "This grant will enable development of our advanced manufacturing technology for multiple AAV serotypes, evaluation of their key process variables as well as manufacturability. Being able to share our findings not just with our partner but also with the broader NIIMBL community is incredibly gratifying." Gregg Nyberg, Ph.D., chief technology officer of Landmark Bio said, "We believe this technology has the potential to reduce resin costs and improve step recoveries, increasing capacity while lowering the cost of goods and making these groundbreaking treatments more accessible to patients. Landmark is eager to integrate continuous chromatography into our Postmark™ AAV manufacturing platform." "This project exemplifies NIIMBL's commitment to identifying, investing in, and advancing high-potential member technologies through collaboration and partnership. By fostering innovation, we are strengthening manufacturing capabilities for emerging biopharmaceutical products. We look forward to the continued advancements in AAV vector production and their impact on the field," said Sandeep Kedia, senior technology fellow at NIIMBL. NIIMBL is a public-private partnership whose mission is to accelerate biopharmaceutical innovation, support the development of standards that enable more efficient and rapid manufacturing capabilities, and educate and train a world-leading biopharmaceutical manufacturing workforce, fundamentally advancing U.S. competitiveness in this industry. NIIMBL is part of Manufacturing USA®, a network of federally sponsored manufacturing innovation institutes, and is funded through a cooperative agreement with the National Institute of Standards and Technology (NIST) in the U.S. Department of Commerce with significant additional support from its members. This project was developed with an award from NIIMBL and financial assistance from the U.S. Department of Commerce, NIST (70NANB21H086). About ChromaTan, in Spring House, PA, ChromaTan is a trusted partner to the biotechnology industry, specializing in biomanufacturing process intensification through its proprietary Biologic Real Moving Bed (BioRMB™) technology. BioRMB™ is a column-free, continuously cycling resin slurry that enables continuous purification of biologic feeds, including viral vectors (AAV, Lentivirus), mRNA, plasmids, recombinant proteins, and antibodies. For more information, visit or follow ChromaTan on LinkedIn. About Landmark BioLandmark Bio PBLLC, a statutory public benefit limited liability company, or PBLLC, is a collective endeavor launched by leaders from academia, the life sciences industry, and world-renowned research hospitals to accelerate the development and industrialization of novel therapeutics. Inspired by recent advancements in cell and gene therapy, Landmark Bio was established to remove barriers in drug development, create accessible capability, expertise, and solutions, and offer a collaboration platform to advance manufacturing technologies for the new generation of medicines to come. Founding partners include Harvard University, Massachusetts Institute of Technology (MIT), Cytiva, FUJIFILM Diosynth Biotechnologies (FDB), and Alexandria Real Estate Equities, Inc. Other collaborating institutions include Beth Israel Deaconess Medical Center, Boston Children's Hospital, Mass General Brigham, and the Dana- Farber Cancer Institute. For more information, visit View original content to download multimedia: SOURCE Landmark Bio Sign in to access your portfolio
