- Tags: 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.
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.
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.
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.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.
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.
Pipeline
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.
Pipeline
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.
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.
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.
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.
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