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 (gene-agnostic, gene modifier) – Ocugen |
Phase 1/2 |
|
AMD-dry, GA (CFH) – Perceive Bio |
Phase 1/2 |
|
Batten disease (CLN5) – Neurogene |
Phase 1/2 |
|
LCA (GUCY2D) – Atsena |
Phase 1/2 |
|
LCA (AIPL1) – MeiraGTx |
Phase 1/2 |
|
LCA (LCA5) – Opus Genetics |
Phase 1/2 |
|
LCA (RPE65, LUXTURNA) |
Approved |
|
RP (CNGA1) – VeonGen |
Phase 1b |
|
RP (PDE6B) – Coave |
Phase 1/2 |
|
RP (gene-agnostic, gene modifier) – Ocugen |
Phase 3 |
|
RP (gene-agnostic, cone preservation) – SparingVision |
Phase 1/2 |
|
RP (PDE6A) – Tubingen Hosp |
Phase 1/2 |
|
Retinoschisis (RS1) – Atsena |
Phase 1/2 |
|
Stargardt disease (gene-agnostic, gene modifier) – 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 |
|
|
|
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 |
|
RP, Usher, others (optogenetic) – Ray Therapeutics |
Phase 1/2 |
|
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 stem cells) – 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 (SYFOVRE, C3 inhibitor) – Apellis |
Approved |
|
AMD-dry, GA (IZERVAY), C5 inhibitor) – Astellas |
Approved |
|
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 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 (small molecule) – Belite Bio |
Phase 3 |
|
Stargardt disease (metformin) – NEI |
Phase 2 |
|
Usher syndrome (NACA-anti-oxidant) – Nacuity |
Phase 2 |
|
Usher syndrome 3 (small molecule) – USH III Initiative |
Phase 1 |
Information updated quarterly. Current as of July 2025.
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.
