Gene Therapy

The gene therapy etranacogene dezaparvovec conferred a substantial decrease in bleeding events among patients with moderate or severe hemophilia B, according to preliminary data presented at the virtual ASH Annual Meeting and Exposition.

Twelve-month results from a phase 1/2 trial of sepofarsen for Leber congenital amaurosis type 10 showed a manageable safety profile in adults and children, according to a poster presented at the virtual American Academy of Ophthalmology meeting.

Janssen Pharmaceuticals has acquired the rights to HMR59, an investigational one-time intravitreal injection gene therapy being developed for the treatment of geographic atrophy, from Hemera Biosciences, according to a press release.

Patients with neovascular age-related macular degeneration treated with ADVM-022 in the OPTIC phase 1 study have experienced a substantial reduction in annualized anti-VEGF injection frequency, according to a speaker.

At 12 months, data from the phase 1/2 study of AAV5-RPGR gene therapy for RPGR-associated X-linked retinitis pigmentosa showed significant improvement in retinal sensitivity and functional mobility with the low and intermediate doses.

Patients with advanced retinitis pigmentosa who received intravitreal optogenetics gene therapy in a phase 1/2a study experienced improved visual acuity from baseline at 16 weeks, according to a speaker.

The first surgery using the Orbit subretinal delivery system to deliver GT005 to a patient with geographic atrophy secondary to dry age-related macular degeneration has been conducted in the ongoing phase 1/2 FOCUS trial.

Huntington’s disease is a fatal genetic disorder that results in the deterioration a person’s physical and mental abilities. It is known as the “quintessential family disease” because every child of a parent with Huntington’s disease has a 50-50 chance of inheriting the HD gene, according to the Huntington’s Disease Society of America (HDSA). There are approximately 41,000 Americans with Huntington’s disease (HD) and more than 200,000 at risk for inheriting the disease.

The first patient has been dosed in a phase 3 clinical trial of debcoemagene autoficel for the treatment of recessive dystrophic epidermolysis bullosa, Castle Creek Biosciences announced in a press release.

Novartis has acquired Vedere Bio and added a platform for adeno-associated virus-based gene therapy delivery, according to a press release.