Gene Therapy

Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research.

Nine-month data from a phase 1/2 clinical trial of AAV-RPGR gene therapy for X-linked retinitis pigmentosa showed clinically meaningful improvement in retinal sensitivity and vision-guided mobility.

Gene therapy represents a new potential avenue for the treatment of retinal diseases, including age-related macular degeneration, according to a presenter at the virtual OSN New York Retina meeting.

Gene therapies for inherited retinal degenerations may offer new treatments for the estimated one in 2,000 patients affected by this heterogenous group of diseases, according to a speaker.

The first adult patient has been dosed in a phase 1/2 clinical trial of FCX-013 for the treatment of scleroderma, Castle Creek Biosciences announced in a press release.

Injection in the subretinal space, currently performed or investigated for several cell-based and gene therapy procedures, could soon become more precise and safer with the help of robotics.

Gene therapy for corneal disease holds promise but still has limitations, according to one speaker at the virtual EuCornea congress.

September is National Sickle Cell Awareness Month.

Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release.

The FDA has granted fast track designation to GT005 for the treatment of geographic atrophy secondary to dry age-related macular degeneration, according to a press release from Gyroscope Therapeutics.