New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: AGTC, Dyne, Genenta, Luye, Metagenomi, Otonomy, Regenxbio, Tessa, Ultragenyx.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: 4D, Affinia, Arcellx, Generation, Homology, Rocket, Sio Gene, Uniqure.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: Adaptimmune, Akouos, Beam, Catamaran, Codiak, Dyno, Freeline, M6P, Neurogene, Passage, Phio, Poseida, Precision, Senti, Verve.
Capsida Biotherapeutics Inc., a gene therapy startup focused on advanced capsid engineering to generate tissue-selective vectors, emerged from stealth with $50 million in series A funding and another $90 million in cash from a strategic collaboration and option agreement in neurodegenerative disease with Abbvie Inc.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $581 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
Researchers at the University of California at San Diego have used a gene therapy approach to treat pain by specifically suppressing the Nav 1.7 ion channel in the spinal cord, both preventing and reversing pain in several animal models with distinct underlying reasons for pain.
LONDON – The National Health Service (NHS) in the U.K. has agreed to reimburse Zolgensma, the one-off gene therapy treatment for spinal muscular atrophy billed as the most expensive drug in the world, after a confidential deal was struck with Novartis Gene Therapies.
Gene therapy developer Beam Therapeutics Inc. has acquired drug delivery specialist Guide Therapeutics Inc. in a deal valued at up to $440 million, adding yet another piece of the integrated platform CEO John Evans told BioWorld the company is striving to build. Guide's lipid nanoparticle (LNP) screening technology could help medicines developed by Beam and its partners reach new targets beyond the liver, an increasingly important endeavor for gene therapy developers like Beam, seeking to maximize the breadth of their growing portfolios. Terms of the deal included $120 million up front, plus up to $320 million in stock-based technology and product success milestone payments. Shares in Beam (NASDAQ:BEAM) fell 12.6% to $96.48 on Feb. 23.