• Category: Pipeline
  • Tags: retina
FDA grants Orphan Drug Designation to SignaBlok's ROP therapy
Pipeline

FDA grants Orphan Drug Designation to SignaBlok's ROP therapy

TREM-1 inhibitor is designed to selectively target inflammation via a noninvasive delivery; company plans to report preclinical data at ARVO annual meeting.
EyePoint begins dosing in phase 3 DME trials for DURAVYU
Pipeline

EyePoint begins dosing in phase 3 DME trials for DURAVYU

COMO and CAPRI pivotal studies are evaluating vorolanib insert when intravitreally-injected every 6 months; topline data expected H2 2027.
FDA grants Orphan Drug designation to BlueRock's RP cell therapy
Pipeline

FDA grants Orphan Drug designation to BlueRock's RP cell therapy

Marks second designation for OpCT-001; includes tax credits for qualified clinical testing and, if approved, 7 years of U.S. market exclusivity.
BioAge Labs expands into DME clinical program for oral inhibitor asset
Pipeline

BioAge Labs expands into DME clinical program for oral inhibitor asset

Preclinical evidence on NLRP3 inhibitor BGE-102 shows potential for therapeutic retinal exposure, with an initial POC trial to kick off later this year.
Complement Therapeutics secures Fast Track Designation for lead GA therapy
Pipeline

Complement Therapeutics secures Fast Track Designation for lead GA therapy

AAV-based CTx001 modulates multiple pathways of the complement system when delivered as a one-time, subretinal injection.
Aviceda releases phase 2b topline data on AVD-104 for GA
Pipeline

Aviceda releases phase 2b topline data on AVD-104 for GA

Latest SIGLEC trial results show both dosing arms with 31% to 38% less lesion growth than prior trials; phase 3 plans are underway.
FDA rejects Outlook Therapeutics' third BLA submission of bevacizumab for wet AMD
Pipeline

FDA rejects Outlook Therapeutics' third BLA submission of bevacizumab for wet AMD

Agency cites need for confirmatory evidence of efficacy as reason for latest CRL; ONS-5010 is already approved in the EU and UK as LYTENAVA.
Ocular Therapeutix plans for NDA submission of AXPAXLI for wet AMD
Pipeline

Ocular Therapeutix plans for NDA submission of AXPAXLI for wet AMD

Company expects to submit package in Q1 2026, following the anticipated release of positive 1-year data from the phase 3 SOL-1 trial.
SeaBeLife and Unither to formulate and develop retinal degenerative disorder formulation
Pipeline

SeaBeLife and Unither to formulate and develop retinal degenerative disorder formulation

Collaboration focuses on accelerating development of SBL03, formulated for topical delivery to target retinal cell necrosis in GA and dry AMD.
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.
Belite Bio reports phase 3 topline data on oral tinlarebant for Stargardt disease
Pipeline

Belite Bio reports phase 3 topline data on oral tinlarebant for Stargardt disease

DRAGON study is evaluating the once-daily tablet for reducing lesion growth among adolescent patients over a 24-month period.
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.
PolyActiva and RareSight to advance long-acting therapies for pediatric IRDs
Pipeline

PolyActiva and RareSight to advance long-acting therapies for pediatric IRDs

Collaboration leverages PolyActiva’s PREZIA drug delivery platform to develop NCE-enabling pharmacologic drugs for early-onset vision loss.
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.
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.
Nanoscope's optogenetic therapy demonstrates visual improvements for Stargardt
Pipeline

Nanoscope's optogenetic therapy demonstrates visual improvements for Stargardt

Findings from phase 2 STARLIGHT trial support the safety and efficacy of MCO-010 in Stargardt disease.
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 Breakthrough Therapy Designation to Nacuity's RP tablets
Pipeline

FDA grants Breakthrough Therapy Designation to Nacuity's RP tablets

Third designation for NPI-001 follows recent positive 24-month data showing a 50% photoreceptor reduction after 2 years of daily dosing.
FDA grants priority review for Chiesi's LHON therapeutic
Pipeline

FDA grants priority review for Chiesi's LHON therapeutic

Approved by the EMA as Raxone, idebenone could become the first and only clinically proven treatment to address the underlying cause of LHON.
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.