Hearing recovery occurred in 90 percent of gene therapy patients with OTOF-related hearing loss, with particular improvements in children.

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 ...

AviadoBio will combine its proprietary vMiX™ RNAi platform with Apertura’s novel human TfR1 capsid (TfR1 CapX™) to enable one-time gene silencing in the central nervous system AviadoBio will present k ...

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 FDA has approved Otarmeni, the first-ever gene therapy for genetic hearing loss caused by the OTOF gene, for both ...

After a patient died following a high dose of Neurogene’s Rett syndrome gene therapy candidate, the biotech is sharing details about the event and the new safety protocols being incorporated into the ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...

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 ...

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

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 ...