- Tags: gene therapy
Pipeline
4DMT advances DME gene therapy to phase 3 evaluations
Phase 3 4SIGHT trial has begun enrolling patients and 2-year phase 2 SPECTRA results show over 60% treatment burden reduction.
Pipeline
Beacon Therapeutics releases positive topline phase 2 data on laru-zova for XLRP
VISTA is reportedly the first pivotal trial in XLRP to achieve its FDA-endorsed primary endpoint; company plans to submit a rolling BLA later this year.
Pipeline
FDA accepts Nanoscope's optogenetic gene therapy BLA for RP
The newly-renamed MOGENRY could become the first gene-agnostic treatment for patients with RP having severe vision loss.
Pipeline
Ocugen doses first patient in phase 3 GA gene therapy trial
OCU410 becomes third gene therapy from Ocugen to reach late-stage clinical trials; BLA filing is anticipated in 2028.
Pipeline
Opus Genetics completes phase 3 enrollment for OPGx-LCA5 gene therapy
Topline 6-month efficacy data is expected by the end of 2027, with the goal of supporting a BLA.
Research
Adverum reports 5-year extension data on wet AMD gene therapy
At ASRS 2026, the OPTIC extension finds a single Ixo-vec injection led to a 87% cumulative reduction in annualized anti-VEGF injections.
Pipeline
RegenxBio doses first patient in phase 2b/3 trial for DR
One-time gene therapy continues to move down the pipeline, with presentations on efficacy and safety data expected at ASRS.
Pipeline
Atsena doses first patient in phase 3 XLRS LIGHTHOUSE trial
Enrollment is expected to finish in Q1 2027, with topline results anticipated in H1 2028.
Pipeline
Atsena releases positive XLRS interim data from LIGHTHOUSE study
Enrollment for Part C of the study is underway, with the company targeting a 2028 BLA submission for ATSN-201, an investigational gene therapy.
Pipeline
FDA grants RMAT designation to Ray Therapeutics' RP optogenetic therapy
RTx-015 is a single, IVT-administered therapeutic designed to restore vision by converting surviving neurons into photo-sensing cells.
Pipeline
Complement Therapeutics begins patient dosing in GA gene therapy trial
Opti-GAIN trial is evaluating a single subretinal injection of CTx-001, an AAV-based gene therapy modulating multiple pathways of the complement system.
Pipeline
4DMT completes enrollment for phase 3 wet AMD gene therapy trial
4FRONT-1 study is evaluating 4D-150 as a single, low-dose IVT injection among treatment-naïve wet AMD patients; topline data expected in H1 2027.
Pipeline
Complement Therapeutics secures Fast Track Designation for lead GA therapy
AAV-based CTx001 modulates multiple pathways of the complement system when delivered as a one-time, subretinal injection.
Pipeline
FDA clears Zhongmou Therapeutics' IND for RP optogenetic gene therapy
Clearance of ZM-02 marks the first optogenetic gene therapy originating from China to receive U.S. IND authorization.
Pipeline
Coave Therapeutics debuts lead SCS gene therapy for retinal vascular diseases
In-office, suprachoroidal delivery of CoTx-101 offers potential for durable and long-lasting vision gains in treating wet AMD and DME.
Pipeline
Nanoscope reports 3-year vision improvements for RP optogenetic therapy
Long-term extension from phase 2b/3 RESTORE trial notes potential of MCO-010 as a one-time, gene-agnostic optogenetic therapy.
Business
Lilly to purchase Adverum Biotechnologies
With a Q4 closing date in mind, acquisition supports ongoing clinical trials and development activities for Ixo-vec, a one-time IVT gene therapy injection to treat wet AMD.Pipeline
Complement Therapeutics' GA gene therapy IND receives FDA clearance
Clearance gives green light to begin Q1 2026 patient enrollment for the Opti-GAIN study evaluating CTx001 as a one-time, durable treatment.
Pipeline
FDA grants Orphan Drug designation to AAVantgarde's Stargardt gene therapy
Regulatory milestone is second designation for AAVB-039; includes tax credits for qualified clinical testing and, if approved, seven years of U.S. market exclusivity.
Pipeline