Gene Therapy

There are long-term concerns about gene therapy for a number of reasons.

Applied Genetic Technologies Corporation announced an update on the proposed design of an upcoming phase 2/3 trial for its X-linked retinitis pigmentosa clinical program, according to a press release.

The first patient has been dosed in a phase 2 trial evaluating suprachoroidal delivery of RGX-314 to treat wet age-related macular degeneration, according to a Regenxbio press release.

The FDA cleared an investigational new drug application for AGT103-T, an autologous, genetically modified cell therapy for the treatment of HIV, according to the agent’s manufacturer.

The FDA has granted a fourth orphan drug designation for OCU400 to treat PDE6B gene mutation-associated retinal diseases, according to an Ocugen press release.

Retinal sensitivity improved in patients with RPGR-associated X-linked retinitis pigmentosa treated with an AAV-RPGR gene therapy, a speaker said at the virtual American Society of Retina Specialists meeting.

In this video from the virtual American Society of Retina Specialists meeting, Arshad Khanani, MD, discusses results of a phase 1/2a study of RGX-314 gene therapy (Regenxbio) for the treatment of wet age-related macular degeneration.

Applied Genetic Technologies Corporation has received written feedback from the FDA about its potential X-linked retinitis pigmentosa treatment and updated its development plan accordingly, according to a press release.

Massachusetts Eye and Ear and Biogen plan to partner to develop a gene therapy for treatment of inherited retinal degeneration due to mutations in the PRPF31 gene.

A combination of optogenetic gene therapy and use of a light stimulation medical device for the treatment of retinitis pigmentosa was well tolerated among the first cohort of patients enrolled in the PIONEER study, according to data reported at the Association for Research in Vision and Ophthalmology annual meeting.