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 (CNGB3) – AGTC |
Phase 1/2 |
|
Achromatopsia (CNGB3) – MeiraGTx / Janssen |
Phase 1/2 |
|
Achromatopsia (CNGA3) – MeiraGTx / Janssen |
Phase 1/2 |
|
Achromatopsia (CNGA3) – Tubingen Hosp |
Phase 1/2 |
|
AMD-dry (CFI) – Novartis (Gyroscope) |
Phase 2 |
|
Batten disease (CLN5) – Neurogene |
Phase 1/2 |
|
Choroideremia (REP1) – 4DMT |
Phase 1/2 |
|
Choroideremia (REP1) – Tubingen Hosp |
Phase 2 |
|
LCA (GUCY2D) – Atsena |
Phase 1/2 |
|
LCA and RP (RPE65) – MeiraGTx / Janssen |
Phase 1/2 |
|
RP (PDE6B) – Coave |
Phase 1/2 |
|
RP (RLBP1) – Novartis |
Phase 1/2 |
|
RP (NR2E3, RHO) – Ocugen |
Phase 1/2 |
|
RP (PDE6A) – Tubingen Hosp |
Phase 1/2 |
|
Retinoschisis (RS1) – NEI |
Phase 1/2 |
|
X-linked RP (RPGR) – AGTC |
Phase 2 |
|
X-linked RP (RPGR) – MeiraGTx / Janssen |
Phase 1/2 |
|
X-linked RP (RPGR) – 4DMT |
Phase 1/2 |
|
|
|
RNA/OTHER (MECHANISM) |
PROGRESS |
|
AMD-dry (CB inhibitor) – Ionis |
Phase 2 |
|
LCA (CEP290, AON) – ProQR |
Phase 2/3 |
|
RP, Usher, others (optogenetic) – Bionic Sight |
Phase 1/2 |
|
RP, Usher, others (optogenetic) – GenSight |
Phase 1/2 |
|
RP, Usher, others (optogenetic) – Nanoscope |
Phase 2 |
|
Stargardt disease (optogenetic) – Nanoscope |
Phase 2 |
|
Usher syndrome 2A (AON) – ProQR |
Phase 2/3 |
|
|
|
CELL-BASED THERAPIES (CELL TYPE) |
PROGRESS |
|
AMD-dry (RPE) – Lineage |
Phase 1/2 |
|
AMD-dry (RPE) – Luxa |
Phase 1/2 |
|
AMD-dry (RPE from iPSC) – NEI |
Phase 1/2 |
|
AMD-dry (RPE on scaffold) – Regen Patch |
Phase 1/2 |
|
RP, Usher (retinal progenitors) – jCyte |
Phase 2b |
|
Stargardt disease (RPE) – Astellas |
Phase 1/2 |
|
|
|
SMALL MOLECULES (MECHANISM) |
PROGRESS |
|
AMD-dry (C3 inhibitor) – Apellis |
Phase 3 |
|
AMD-dry (C5 inhibitor) – Iveric bio |
Phase 3 |
|
RP (NAC-anti-oxidant) – Johns Hopkins |
Phase 3 |
|
RP (methotrexate) – Aldeyra |
Phase 2 |
|
RP (small molecule) – Endogena |
Phase 1/2 |
|
PR (small molecule, photoswtich) – Kiora |
Phase 1/2 |
|
Stargardt disease (deuterated vit A) – Alkeus |
Phase 2 |
|
Stargardt disease (C5 inhibitor) – Iveric bio |
Phase 2 |
|
Stargardt disease (anti-RBP4) – Belite Bio |
Phase 3 |
|
Stargardt disease (metformin) – NEI |
Phase 1/2 |
|
Usher syndrome (NACA-anti-oxidant) – Nacuity |
Phase 1/2 |
Information updated quarterly. Current as of January 2023.
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.
