Genethon Using AI to Improve Effectiveness of Gene Therapies and Patient Access CEO Frederic Revah discusses how Artificial Intelligence is creating next-generation gene therapy vectors and reducing bioproduction costs in the latest issue of Genethon’s Newsletter Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy…..
Broken String Biosciences Closes $15M Series A Funding Round Broken String Biosciences, a UK-based genomics company building a technology platform to drive the development of cell and gene therapies that are safer by design, has closed a $15 million Series A investment round, co-led by Illumina Ventures and Mérieux Equity…..
Efficacy of gene therapy demonstrated in patients suffering from Crigler-Najjar Syndrome Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the liver enzyme UGT1A1, can cause significant neurological damage and become fatal. At the current time, the only…..
Novartis expands Zolgensma® manufacturing capacity with approval of multi-product North Carolina facility The FDA has granted commercial licensure approval for Novartis' Durham, N.C. site, a multi-product gene therapy manufacturing facility. This approval allows the state-of-the-art, 170,000 square-foot facility to make, test and release commercial Zolgensma®, as well as produce…..
Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology Collaboration combines Intellia’s proprietary genome editing technology platform with SparingVision’s significant ophthalmology expertise Intellia will grant SparingVision exclusive rights to its leading in vivo CRISPR/Cas9 technology for the development of ocular therapies directed to three…..
License Option Agreement with Pfizer for Voyager Therapeutic's Next-Generation TRACERᵀᴹ AAV Capsids Voyager Therapeutics, a gene therapy company developing adeno-associated virus (AAV) platform technologies, announced an agreement through which Pfizer may exercise options to license novel capsids generated from Voyager’s RNA-driven TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of…..
New study expands evaluation of gene therapy for spinal muscular atrophy German researchers have just published results of a retrospective analysis of routine data collected from all patients with spinal muscular atrophy (SMA) treated with Zolgensma® (onasemnogene abeparvovec - adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy…..
Biogen plans to build a new gene therapy manufacturing facility in Research Triangle Park Biogen, a pioneer in neurosciences, plans to build a new gene therapy manufacturing facility at its Research Triangle Park (RTP) manufacturing campuses in North Carolina to support its growing gene therapy pipeline across multiple therapeutic areas.…..
Vector-Based Gene Therapy and Gene-Editing Products Ongoing Clinical Trials Gene therapies are now a reality. They open new perspectives for the treatment and management of a wide range of rare and common diseases. We have listed ongoing clinical trials using viral vector and gene editing products to get a more…..
A new gene therapy could pave the way to the treatment of mitochondrial dysfunctions Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating dominant optic atrophy (DOA). This eye disease is characterized by degeneration of the optic nerves and primarily…..
