Some promising gene therapy updates

Advernum Biotechnologies have announced their promising long-term data from the OPTIC clinical trial of ADVM-022 single – an intravitreal injection for gene therapy in patients with wet age-related macular degeneration (AMD). The data is reflective of patients who have been followed for up to 2 years in order to determine the efficacy of the treatment. “We are very pleased to report that both the promising safety profile and durability of treatment effect continue to be maintained in this patient population,” Laurent Fischer, MD, president and chief executive officer at Adverum Biotechnologies.

Gyroscope Therapeutics have presented their positive interim Phase 1/2 data for investigational gene therapy. The clinical trial focuses on people with geographic atrophy and age-related macular degeneration (AMD). The safety data from 28 patients showed it to be well tolerated with no adverse events. Biomarker data from 13 patients, ranging from 7 months to 2 years, demonstrated sustained increases in vitreous complement factor I levels. “It’s an exciting time in complement research, and we believe a one-time gene therapy has the potential to be a long-term solution for people with geographic atrophy” Nadia Waheed, MD, MPH, Chief Medical Officer, Gyroscope Therapeutics.

REF: https://eyewire.news/news/adverum-data-shows-gene-therapy-maintained-efficacy-durability-safety-in-wet-amd-patients https://eyewire.news/news/adverum-data-shows-gene-therapy-maintained-efficacy-durability-safety-in-wet-amd-patients