- tags:gene therapy
Pipeline
Atsena and Nippon Shinyaku to collaborate on LCA1 gene therapy in US
Nippon Shinyaku will receive exclusive U.S. commercial rights to ATSN-101, a subretinally-injected AAV5 capsid and human RHO promoter.
Business
Ocuphire Pharma just purchased Opus Genetics
Operating under the name “Opus Genetics,” the combined company features a pipeline of IRD-targeted gene therapy candidates.
Pipeline
Beacon Therapeutics reports positive 24-month phase 2 XLRP gene therapy data
Latest findings demonstrate AGTC-501 to be generally safe and well-tolerated, with a 57% response rate noted among high-dose-treated eyes.
Pipeline
Nanoscope to submit optogenetic therapy BLA for RP
Company reports productive FDA meeting regarding MCO-010, plans for a Q1 2025 submission.
Pipeline
FDA grants Orphan Drug designation for Atsena's XLRS gene therapy
ATSN-201 is currently being studied in the phase 1/2 LIGHTHOUSE trial among patients diagnosed with XLRS caused by mutations in the R1 gene.
Pipeline
Nanoscope plans for phase 3 trial on Stargardt macular degeneration
Company reports productive end-of-phase 2 meeting with FDA regarding its gene therapy vector.
Business
Newly-launched Tern Therapeutics acquires ex-REGENXBIO gene therapies
Biotech company receives $15 million in funding to advance clinical development of TTX-381 and TTX-181 for treating ocular and CNS manifestations of CNL2 disease.
Pipeline
FDA grants Opus Genetics Rare Pediatric Disease Designation for LCA gene therapy
Designation qualifies OPGx-LCA5 to receive a priority review voucher, if approved.
Pipeline
FDA grants Atsena Rare Pediatric Disease designation for XLRS gene therapy
New designation of subretinal-injection therapeutic comes amidst the ongoing patient enrollment and dosing of the phase 1/2 LIGHTHOUSE study.
Pipeline
Adverum receives FDA RMAT designation for wet AMD gene therapy
Ixoberogene-soroparvovec is being clinically evaluated as a durable, well-tolerated, and cost-effective, in-office treatment option with extended efficacy following a single injection.
Pipeline
Beacon Therapeutics to evaluate patented AAV gene therapy for retinal diseases
Licensing agreement involves potential development and commercialization of Abeona Therapeutics’ patented capsid, a pan-retinal-administered vector.
Pipeline
Beacon Therapeutic raises $170M for XLRP gene therapy development
Funding supports clinical trials on AGTC-501, lead asset designed to address all photoreceptor damage caused by XLRP.
Pipeline
Ocugen doses first patient in phase 3 RP gene therapy trial
Prior clinical data supports OCU400 in improving baseline vision among patients with RP associated RHO and N2ER3 mutations.
Pipeline
Beacon Therapeutics doses first patient in VISTA XLRP gene therapy trial
Randomized study is investigating AGTC-501 for the treatment of patients diagnosed with x-linked retinitis pigmentosa.
Research
CRISPR gene editing may prevent early-onset vision loss
BRILLIANCE trial examines safety and efficacy of a single injection genome editing medication in 14 LCA patients.
Pipeline
SalioGen Therapeutics's gene coding therapy targets Stargardt disease
One-time, non-viral subretinal injection uses novel Gene Coding technology for gene integration.
Pipeline
Ocugen's IND amendment gets FDA OK for phase 3 RP gene therapy trial
Investigational candidate OCU400 is the first potential treatment for RP associated with rhodopsin mutations.
Pipeline
Nanoscope reports positive data from RESTORE trial on optogenetic therapy for RP
Statistical significance noted for MCO-010 in demonstrating clinically meaningful vision improvement for legally blind, advanced RP patients.
Research
Oculogenex's AMD gene therapy to undergo testing in space
Investigational therapy will launch with the SpaceX CRS mission as part of the ISS National Laboratory-sponsored investigation.
Pipeline