Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

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 ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

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 ...

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 ...

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 ...

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 ...

It will also make preclinical gene therapy studies in mice more efficient and reproducible, given that a lot of the homework on the best AAV for a given cell type has already been done." This work was ...

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 ...