Ocular Gene Therapy
Gene therapy improves diabetic retinopathy severity at 1 year in phase 2 trial
SAN FRANCISCO — A one-time in-office injection of a gene therapy helped improve disease severity in patients with diabetic retinopathy.
VIDEO: Interim results promising for RGX-314 in diabetic retinopathy
SEATTLE — In this Healio Video Perspective from the ASRS meeting, Dilsher Dhoot, MD, discusses the interim results of the phase 2 ALTITUDE trial investigating three doses of RGX-314 gene therapy in diabetic retinopathy.
Novartis discontinues development of GT005 for geographic atrophy
Novartis is discontinuing the development of GT005 for the treatment of geographic atrophy secondary to dry age-related macular degeneration, according to a press release from Syncona.
VIDEO: Phase 2 data for RGX-314 promising for neovascular AMD treatment
SEATTLE — In this Healio Video Perspective from the ASRS meeting, Ashkan M. Abbey, MD, FASRS, FAAO, overviews data evaluating subretinal delivery of RGX-314 gene therapy for the treatment of neovascular age-related macular degeneration.
Phase 2 results encouraging for ABBV-RGX-314 gene therapy in neovascular AMD
SEATTLE — The phase 2 AAVIATE study showed safety and efficacy of ABBV-RGX-314 gene therapy in the treatment of neovascular age-related macular degeneration, with potential for sustained outcomes with a one-time in-office injection.
Ray Therapeutics closes $100 million in oversubscribed Series A financing
Ray Therapeutics has closed an oversubscribed $100 million Series A financing round to advance the company’s optogenetic gene therapy programs targeting blinding retinal diseases, according to a press release.
VIDEO: Intravitreal gene therapy for wet AMD demonstrates positive results
NEW ORLEANS — In this Healio Video Perspective from the ARVO meeting, Arshad M. Khanani, MD, MA, discusses the PRISM trial evaluating 4D-150 intravitreal gene therapy for the treatment of wet age-related macular degeneration.
Good results seen in early-stage intravitreal gene therapy trial for wet AMD
Intravitreal 4D-150 demonstrated positive interim results for the treatment of wet age-related macular degeneration in a phase 1/2 trial, according to a press release from 4D Molecular Therapeutics.
VIDEO: Leber congenital amaurosis gene therapy promising in phase 1/2 trial
NEW ORLEANS — In this Healio Video Perspective from the ARVO meeting, Christine Kay, MD, discusses 6-month results investigating ATSN-101 for the treatment of Leber congenital amaurosis caused by mutations in the GUCY2D gene.
Gene therapy for inherited retinal diseases receives orphan drug designation
The FDA granted orphan drug designation for HG004, a gene replacement therapy for the treatment of inherited retinal diseases caused by RPE65 mutations, HuidaGene Therapeutics announced in a press release.