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 ...
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, ...
Humanity is gradually conquering incurable genetic diseases that were once considered untreatable, thanks to a series of ...
Morning Overview on MSN
Gene therapy restored hearing in 90% with OTOF deafness, study says
A single injection into the inner ear restored functional hearing in 90% of people born deaf because of mutations in the OTOF ...
— Finding Hope for FRRS1L plans to initiate clinical trial using TfR1 CapX gene therapy in the second half of 2026, pending regulatory review — FORT COLLINS, Colo. and NEW YORK, Feb. 18, 2026 ...
SpliceBio, a clinical-stage genetic medicines company pioneering protein splicing to address diseases caused by mutations in large genes, today announced that the first patient has been dosed in the ...
Oral and poster presentations highlight progress across cardiac genetic medicine pipeline and optimized, Sf9-baculovirus AAV ...
Paris Margaritis, DPhil, is a professional with over 28 years of experience in AAV gene therapy for hemophilia. A key focus of his work has been treating hemophilia patients with inhibitors. He earned ...
More than four years after the last of four boys died in a troubled Phase I/II trial of a gene therapy candidate for X-linked Myotubular Myopathy (XLMTM), Astellas Gene Therapies says it remains ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results