Clinical Trial Pipeline

The Foundation Fighting Blindness is the world’s leading private funding source for research to treat, prevent, and cure retinal degenerative diseases. Many of the trials listed below were made possible by Foundation support.

GENE THERAPIES (GENE TARGET)

PROGRESS

Achromatopsia (CNGA3) – Tubingen Hosp

Phase 1/2

AMD-dry, GA (CD59) – Johnson & Johnson

Phase 2

AMD-dry, GA (RORA) – Ocugen

Phase 1/2

AMD-dry, GA (CFH) – Perceive Bio

Phase 1/2

Batten disease (CLN5) – Neurogene

Phase 1/2

Choroideremia (REP1) – 4DMT

Phase 1/2

LCA (GUCY2D) – Atsena

Phase 1/2

LCA (LCA5, lebercillin) – Opus Genetics

Phase 1/2

RP (CNGA1) – ViGeneron

Phase 1b

RP (PDE6B) – Coave

Phase 1/2

RP (RLBP1) – Novartis

Phase 1/2

RP & LCA (NR2E3) – Ocugen

Phase 3

RP (RdCVF) – SparingVision

Phase 1/2

RP (PDE6A) – Tubingen Hosp

Phase 1/2

Retinoschisis (RS1) – Atsena

Phase 1/2

Retinoschisis (RS1) – NEI

Phase 1/2

Stargardt disease (RORA) – Ocugen

Phase 1/2

Usher syndrome (USH1B) – Aavantgarde

Phase 1/2

X-linked RP (RPGR) – Beacon

Phase 3

X-linked RP (RPGR) – Johnson & Johnson

Phase 3

X-linked RP (RPGR) – 4DMT

Phase 1/2

 

 

RNA/OTHER (MECHANISM)

PROGRESS

LCA10 (AON targeting IVS26) – Sepul Bio 

Phase 2/3

RP-PRPF31 (CNOT3) – PYC

Phase 1/2

RP, Usher, others (optogenetic) – Bionic Sight

Phase 1/2

RP, Usher, others (optogenetic) – GenSight

Phase 1/2

RP, Usher, others (optogenetic) – Nanoscope

Phase 2

Usher syndrome 2A (AON targeting exon 13) – Sepul Bio 

Phase 2

Stargardt disease (RNA editing) – Ascidian

Phase 1/2

Stargardt disease (optogenetic) – Nanoscope

Phase 2

 

 

CELL-BASED THERAPIES (CELL TYPE)

PROGRESS

AMD-dry, GA (RPE) – Astellas

Phase 1/2

AMD-dry, GA (RPE) – Lineage

Phase 1/2

AMD-dry, GA (RPE) – Luxa

Phase 1/2

AMD-dry, GA (RPE from iPSC) – NEI

Phase 1/2

AMD-dry, GA (RPE on scaffold) – Regen Patch

Phase 1/2

RP, Usher (retinal progenitors) – jCyte

Phase 2b

RP (CD34+ stem cells) – UC Davis

Phase 1

 

 

SMALL MOLECULES (MECHANISM)

PROGRESS

AMD-dry, GA (deuterated vit. A) – Alkeus

Phase 3

AMD-dry, GA ((anti-C1q) – Annexon

Phase 3

AMD-dry, GA (RBP4 inhibitor) – Belite Bio

Phase 3

AMD-dry, GA (elamipretide) – Stealth Bio

Phase 3

RP (NAC-anti-oxidant) – Johns Hopkins

Phase 3

RP (small molecule) – Endogena

Phase 1/2

RP (small molecule, photoswtich) – Kiora

Phase 1/2

RP (disulfiram) – University of Washington

Phase 1/2

Stargardt disease (deuterated vit A) – Alkeus

Phase 2

Stargardt disease (C5 inhibitor) – Astellas

Phase 2

Stargardt disease (anti-RBP4) – Belite Bio

Phase 3

Stargardt disease (metformin) – NEI

Phase 2

Usher syndrome (NACA-anti-oxidant) – Nacuity

Phase 2

 

Information updated quarterly. Current as of October 2024.

Some trials listed may have been paused and/or the sponsors are seeking partners to continue their trials. To learn more about all the clinical trials underway for inherited retinal diseases, visit www.ClinicalTrials.gov. The site lists comprehensive information about relevant trials including contacts.