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 ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks 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 ...

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 ...

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 ...

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 ...

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 ...

At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...

Neovascular age-related macular degeneration (nAMD) is a progressive eye disease characterized by choroidal neovascularization and subretinal hemorrhage and exudation, leading to vision impairment.

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 ...