Gene Therapy
New molecule may help adults who do not respond to traditional sickle cell disease therapy
Researchers at Boston Medical Center identified a new small molecule that could benefit people with sickle cell disease who do not respond to hydroxyurea.
Gene therapy superior to routine prophylaxis in hemophilia A
Giroctocogene fitelparvovec reduced bleeding for men with moderately severe to severe hemophilia A, according to data released by the agent’s manufacturer.
Disease progression slowed by 80% at 24 months in phase 1/2 studies of HD gene therapy
Interim results of phase 1/2 clinical trials of an investigational gene therapy for Huntington’s disease showed that disease progression slowed by 80% at 24 months, according to the manufacturer.
VIDEO: Gene therapy candidate may reduce treatment burden in neovascular AMD
PARK CITY, Utah — In this Healio Video Perspective from Clinical Trials at the Summit, Adam Turpcu, PhD, of Adverum Biotechnologies discusses Ixo-vec for neovascular age-related macular degeneration.
World Sickle Cell Awareness Day: Gene therapy ‘transformative’
Today is World Sickle Cell Awareness Day.
Gene editing therapy shows potential for CEP290-associated inherited retinal degeneration
A gene editing therapy showed safety and improvement in cone photoreceptor function in patients with CEP290-associated inherited retinal degeneration due to an IVS26 variant, according to a phase 1/2 study.
FDA selects Rett syndrome gene therapy for rare disease pilot drug program
The FDA has selected an investigational AAV9 gene therapy for Rett syndrome to participate in a pilot program created to provide a boost to novel drug development for rare diseases, according to a release from the manufacturer.
FDA grants regenerative medicine advanced therapy designation for Huntington’s treatment
The FDA has granted regenerative medicine advanced therapy designation for an investigational gene therapy to treat Huntington’s disease, according to the manufacturer.
Q&A: ‘Astounding’ success for gene therapy in otoferlin-related deafness
A gene therapy for otoferlin-related deafness was able to improve the hearing in two children, according to results from an ongoing early-stage trial reported at the American Society of Cell and Gene Therapy Meeting.
Racing modified cells against each other in humans may be the next step in gene therapies
The next evolution of gene therapies may involve pitting T cells with different modifications against one another in a “race” inside a human to determine which edits produce the best results against a tumor, an expert panel predicted.