• Tags: gene therapy
FDA grants Orphan Drug designation to AAVantgarde's Stargardt gene therapy
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.
FDA grants RMAT designation to Nanoscope for Stargardt gene therapy
Pipeline

FDA grants RMAT designation to Nanoscope for Stargardt gene therapy

MCO-010 optogenetic therapeutic has potential to become first-ever treatment for Stargardt; phase 3 trial expected to kick off in Q4 2025.
Avista and Forge Biologics partner to advance XLRS gene therapy
Pipeline

Avista and Forge Biologics partner to advance XLRS gene therapy

Collaboration leverages Forge’s in-house AAV development and manufacturing services for AVST-101, an AAV-based IVT gene therapy.
FDA clears Opus Genetics' IND for BEST1 IRD gene therapy
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FDA clears Opus Genetics' IND for BEST1 IRD gene therapy

Company plans to initiate phase 1/2 clinical trial on OPGx-BEST1, a potential first-ever treatment for this patient base.
FDA grants Fast Track designation to AAVantgarde's Stargardt gene therapy
Pipeline

FDA grants Fast Track designation to AAVantgarde's Stargardt gene therapy

Status benefits clinical study of AAVB-039, an AAV8 intended to address the disease’s root cause by delivering the full-length ABCA4 gene via a single subretinal injection.
4DMT releases 60-week phase 2 analysis on wet AMD gene therapy
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4DMT releases 60-week phase 2 analysis on wet AMD gene therapy

Reported at ASRS, data supports sustained vision gains and a 78% reduction in supplemental injections for 4D-150’s highest dose as company preps for phase 3 trial.
FDA grants Sanofi Fast Track designation for GA gene therapy
Pipeline

FDA grants Sanofi Fast Track designation for GA gene therapy

Status gives expedited pathway toward regulatory approval for SAR446597, designed as a one-time injection that blocks two of the three complement system pathways.
FDA OKs AAVantgarde's IND for Stargardt gene therapy trial
Pipeline

FDA OKs AAVantgarde's IND for Stargardt gene therapy trial

Clearance enables initiation of FIH study on AAVB-039, an AAV8 that delivers the full-length ABCA4 gene to address the disease’s root cause—enabling treatment for all types.
Nanoscope begins rolling BLA submission for RP gene-agnostic therapy
Pipeline

Nanoscope begins rolling BLA submission for RP gene-agnostic therapy

With a full submission planned for early 2026, MC0-010 has the potential to become the first optogenetic therapy to restore vision in legally blind RP patients.
FDA agrees to Atsena's regulatory pathway for XLRS gene therapy
Pipeline

FDA agrees to Atsena's regulatory pathway for XLRS gene therapy

Expansion of LIGHTHOUSE study will support potential BLA submission in early 2028 to become the first FDA-approved treatment for the inherited retinal disease.
4DMT cuts 25% of workforce and fast-tracks wet AMD gene therapy program
Business

4DMT cuts 25% of workforce and fast-tracks wet AMD gene therapy program

Company reports streamlined planning to advance phase 3 clinical trials of 4D-150, under evaluation to deliver a single, low-dose, and sustained multi-year intravitreal injection.
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 Stargardt 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
Pipeline

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.