Andelyn Biosciences has been selected by Drake Rayden Foundation to apply the AAV Curator Platform to manufacture clinical grade AAV for the Treatment of Nonketotic Hyperglycinemia (NKH).
Gene therapy (introducing genetic material into living cells to fix, replace, enhance, or block a faulty gene) is rapidly gaining traction as a strategy for the treatment of genetic diseases. The ...
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Unlocking the secrets of gene therapy delivery: New insights into genome ejection from AAV vectors
A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. Subscribe ...
The US Pharmacopeia (USP) has recognized mass photometry as a key orthogonal method for characterizing its new adeno-associated virus (AAV) reference standards, as referenced in the draft general ...
Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their research efforts around the adeno-associated virus (AAV) vectors used to ...
WALTHAM, Mass.--(BUSINESS WIRE)--Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies ...
PHILADELPHIA, PA — Latus Bio, Inc. has unveiled groundbreaking research on a novel adeno-associated virus (AAV) capsid variant, AAV-Ep+, which shows significant promise for advancing gene therapy ...
Adeno-associated viruses (AAVs) have emerged as essential tools for delivering genetic material into tissues and engineering therapeutic cells. As gene and cell therapies advance, researchers optimize ...
Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today ...
SOUTH SAN FRANCISCO, Calif. , May 14, 2025 /PRNewswire/ -- Plasmidsaurus, the company that sequences while you sleep, today announced the launch of a new product update that delivers full-length AAV ...
A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent ...
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