MeiraGTx’s AAV-AIPL1 has already led to vision gains in blind children, as regulatory submissions for the gene therapy close ...

The MarketWatch News Department was not involved in the creation of this content.-- Expanded Dyno Frontiers Program connects therapeutic developers to a marketplace of partners re ...

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 ...

HOUSTON, Sept. 8, 2025 /PRNewswire/ -- AAVivo, Inc., a pioneering biotechnology developing novel, precision targeted biotherapeutics to enable in vivo generation of CAR-T cells, today announced that ...

Voyager faces a challenging outlook, with a negative enterprise value and declining stock amid industry-wide setbacks in AAV gene therapy. The company's lead asset, VY7523, is early in clinical ...

Adeno-associated virus (AAV)–mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had been ...

AstraZeneca’s rare disease unit Alexion has penned a $825 million deal for adeno-associated virus (AAV) capsids from existing partner JCR Pharmaceuticals to develop genomic medicines. Under the deal, ...

Complement activation in patients with AAV who are in remission may be reflective of ongoing renal scarring. Persistent complement activation during disease remission in antineutrophil cytoplasmic ...