• Tags: gene therapy
FDA clears Ocugen for phase 2/3 trial on Stargardt gene therapy
Pipeline

FDA clears Ocugen for phase 2/3 trial on Stargardt gene therapy

IND approval enables continued clinical evaluation of OCU410ST, a modifier gene therapy with a favorable safety and tolerability profile; plans include a 2027 BLA submission.
ViGeneron rebrands as VeonGen Therapeutics with new FDA designation
Pipeline

ViGeneron rebrands as VeonGen Therapeutics with new FDA designation

German-based genetic medicine company also receives a Rare Pediatric Disease Designation for its Sargardt disease gene therapy candidate.
Opus Genetics reports positive phase 3 data for phentolamine 0.75%
Pipeline

Opus Genetics reports positive phase 3 data for phentolamine 0.75%

Topline findings indicate the gene therapy achieved a ≥15-letter (≥3-line) gain in mesopic low contrast distance visual acuity versus placebo.
FDA grants Rare Pediatric Disease designation to Stargardt gene therapy
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FDA grants Rare Pediatric Disease designation to Stargardt gene therapy

OCU410ST could receive a priority review voucher if approved; company plans to initiate a phase 2/3 trial in coming weeks—along with a BLA filing in 2027.
Atsena releases favorable LIGHTHOUSE trial data on XLRS gene therapy
Pipeline

Atsena releases favorable LIGHTHOUSE trial data on XLRS gene therapy

Positive findings from 1-year post-treatment show meaningful improvements on visual function measurements, with foveal schisis closure observed in 67% of patients.
FDA grants RMAT designation to 4DMT for DME gene therapy
Pipeline

FDA grants RMAT designation to 4DMT for DME gene therapy

4D-150 provides a multi-year sustained delivery of anti-VEGF from the retina via a single, low-dose IVT delivery—potentially reducing the need for frequent bolus injections.
FDA grants RMAT designation to Atsena's XLRS gene therapy
Pipeline

FDA grants RMAT designation to Atsena's XLRS gene therapy

This marks ATSN-201’s fourth designation in the FDA’s expedited drug development program, propelling it one step closer to becoming the first approved treatment for this IRD.
Opus Genetics reports first-in-human trial data on LCA gene therapy
Pipeline

Opus Genetics reports first-in-human trial data on LCA gene therapy

One-month findings from the first of three pediatric dosings indicate a clinically meaningful improvement in vision following just one dosing.
FDA approves Neurotech's ENCELTO cell therapy for MacTel
Products

FDA approves Neurotech's ENCELTO cell therapy for MacTel

Approval of revakinagene taroretcel-lwey is the first-ever for the treatment of this rare, neurodegenerative retinal disease. 
Positive 52-week data supports 4DMT's wet AMD gene therapy
Pipeline

Positive 52-week data supports 4DMT's wet AMD gene therapy

PRISM trial extension cohort finds 4D-150 continues to be well tolerated after 3 years of follow-up; phase 3 program initiation to begin this year.
FDA grants RMAT designation to Beacon Therapeutics XLRP gene therapy
Pipeline

FDA grants RMAT designation to Beacon Therapeutics XLRP gene therapy

Laru-zova (AGTC-501) targets the full expression of the RPGR protein, with clinical data reporting its safety, tolerance, and robust visual function improvement effects.
FDA grants Rare Pediatric Disease Designation for ViGeneron's RP gene therapy
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FDA grants Rare Pediatric Disease Designation for ViGeneron's RP gene therapy

Designation qualifies VG901 to receive a priority review voucher if approved; company also receives dose escalation approval for phase 1 trial.
Opus Genetics and FDA agree on SPA for phase 3 NPDR gene therapy trial
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Opus Genetics and FDA agree on SPA for phase 3 NPDR gene therapy trial

Study’s newly-established primary endpoint will support a potential new drug application submission of APX3300 for market approval.
FDA clears SpliceBio's IND of gene therapy for Stargardt trial
Pipeline

FDA clears SpliceBio's IND of gene therapy for Stargardt trial

Clearance represents a significant milestone as the first-ever IND for a protein-splicing gene therapy designed to target the genetic root cause of this disease.
Adverum reports extended 4-year findings on wet AMD gene therapy
Pipeline

Adverum reports extended 4-year findings on wet AMD gene therapy

Promising results from the OPTIC extension program support long-term benefits of Ixo-vec in reducing treatment burden of anti-VEGF injections.
Adverum releases positive phase 2 data on wet AMD gene therapy
Pipeline

Adverum releases positive phase 2 data on wet AMD gene therapy

Investigators of the LUNA study select promising dosage of Ixo-vec determined to have a predictable long-term favorable safety profile for a planned phase 3 program.
Atsena and Nippon Shinyaku to collaborate on LCA1 gene therapy in US
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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. 
Ocuphire Pharma just purchased Opus Genetics
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.
Beacon Therapeutics reports positive 24-month phase 2 XLRP gene therapy data
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. 
Nanoscope to submit optogenetic therapy BLA for RP
Pipeline

Nanoscope to submit optogenetic therapy BLA for RP

Company reports productive FDA meeting regarding MCO-010, plans for a Q1 2025 submission.