• Category: Pipeline
jCyte doses first patients in RP allogeneic cell therapy trial
Pipeline

jCyte doses first patients in RP allogeneic cell therapy trial

Phase 2b JC02-88 trial to evaluate a single IVT injection of jCell (famzeretcel), an agnostic cell therapy with the potential to restore vision in all RP genetic subtypes.
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 green lights Myra Vision's IDE study for glaucoma
Pipeline

FDA green lights Myra Vision's IDE study for glaucoma

IDE approval enables company to initiate the open-label ADAPT study on its proprietary Calibreye TGT Surgical System.
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 agrees to SPA for Ocular Therapeutix NPDR registrational trial
Pipeline

FDA agrees to SPA for Ocular Therapeutix NPDR registrational trial

Agreement includes planned design for study evaluating AXPAXLI (formerly OTX-TIKI), an IVT implant with the potential for long-term durability and fewer injections.
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.
Kiora's KIO-104 receives US patent for expanded protection of inflammatory retinal diseases
Pipeline

Kiora's KIO-104 receives US patent for expanded protection of inflammatory retinal diseases

The investigational small molecule’s intellectual property and potential market exclusivity are now protected through 2043.
Boehringer partners with Re-Vana on extended-release ophthalmic therapeutics
Pipeline

Boehringer partners with Re-Vana on extended-release ophthalmic therapeutics

Collaboration involves photo-crosslinked, biodegradable implants designed to enable continuous, 3-to-12-month drug delivery to treat retinal diseases.
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 accepts Aldeyra's resubmitted reproxalap NDA
Pipeline

FDA accepts Aldeyra's resubmitted reproxalap NDA

Company hopes third submission of the DED application is the charm for achieving marketing approval by a Dec. 26, 2025 PDUFA date.
Belite Bio concludes enrollment for phase 3 GA trial on oral tinlarebant
Pipeline

Belite Bio concludes enrollment for phase 3 GA trial on oral tinlarebant

Also under clinical development for Stargardt disease, the once-daily tablet is under evaluation for its efficacy in slowing atrophic lesion growth.
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.
LENZ and Théa partner on Canadian commercialization of presbyopia eye drop
Pipeline

LENZ and Théa partner on Canadian commercialization of presbyopia eye drop

Licensing and commercialization agreement gives LENZ $70 million in upfront and milestone payments; news comes as company awaits FDA approval.
Opus Genetics' VEGA-3 presbyopia trial meets endpoints for phentolamine 0.75%
Pipeline

Opus Genetics' VEGA-3 presbyopia trial meets endpoints for phentolamine 0.75%

Topline data found a statistically significant number of patients achieved a ≥15-letter improvement in binocular DCNVA; H2 2025 NDA submission expected.
Stuart Therapeutics reports initial phase 3 DED eye drop data
Pipeline

Stuart Therapeutics reports initial phase 3 DED eye drop data

Results on ST-100 (vezocolmitide), a collagen mimetic peptide, support its role as a fast-acting resolution for dry eye signs and symptoms.
Aldeyra resubmits reproxalap NDA for dry eye (again)
Pipeline

Aldeyra resubmits reproxalap NDA for dry eye (again)

Company includes new phase 3 dry eye crossover data in updated submission package; an FDA acknowledgement response is expected within 30 days.
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.