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The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.

NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.

The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Among 12 patients evaluable for efficacy, 10 patients experienced stabilization or improvement from baseline in BCVA in their treated eyes.

Opus Genetics plans to add a pediatric cohort to the phase 1/2 trial once safety is established in adults.

Review top news and interview highlights from the week ending September 15, 2023.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

It was the first time the Eye Center treated a patient with a gene therapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

ATSN-201 utilizes Atsena’s novel spreading capsid AAV.SPR, which is expected to spread laterally and allow transduction of the gene therapy in the central retina.

The single-arm, open-label clinical trial will seek to enroll approximately 18 patients aged 18 to 75 years.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending August 18, 2023.

The senior partner at Retina Vitreous Associates Medical Group discussed new findings from the AAVIATE study of RGX-314.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed unmet needs with the current treatment landscape for DR.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Magdalena Cichewicz, PhD, a scientist II at Senti Biosciences, discussed preclinical research she presented at ASGCT’s 2023 conference.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The scientist II at Senti Biosciences discussed the potential of synthetic promoters to improve target specificity in AAV vector-based gene therapies in retinal diseases and beyond.

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.