• Tags: gene therapy
FDA clears Zhongmou Therapeutics' IND for RP optogenetic gene therapy
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.
Coave Therapeutics debuts lead SCS gene therapy for retinal vascular diseases
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.
Nanoscope reports 3-year vision improvements for RP optogenetic therapy
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.
Lilly to purchase Adverum Biotechnologies
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.
Complement Therapeutics' GA gene therapy IND receives FDA clearance
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.
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
Pipeline

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
Pipeline

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.