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 |
PROGRESS |
|
Achromatopsia (CNGB3) – AGTC |
Phase 1/2 |
|
Achromatopsia (CNGB3) – MeiraGTx / Janssen |
Phase 1/2 |
|
Achromatopsia (CNGA3) – AGTC |
Phase 1/2 |
|
Achromatopsia (CNGA3) – Tubingen Hosp |
Phase 1/2 |
|
AMD (Dry) – Gyroscope |
Phase 2 |
|
Choroideremia (REP1) – 4DMT |
Phase 1/2 |
|
Choroideremia (REP1) – Biogen (Nightstar) |
Phase 3 |
|
Choroideremia (REP1) – Spark |
Phase 1/2 |
|
Choroideremia (REP1) – Tubingen Hosp |
Phase 2 |
|
LCA (GUCY2D) – Atsena |
Phase 1/2 |
|
LCA and RP (RPE65) – MeiraGTx / Janssen |
Phase 1/2 |
|
LCA and RP (RPE65) – Spark |
FDA Approved |
|
RP (PDE6B) – Horama |
Phase 1/2 |
|
RP, Usher, others (optogenetic) – Allergan |
Phase 1/2 |
|
RP, Usher, others (optogenetic) – Bionic Sight |
Phase 1/2 |
|
RP, Usher, others (optogenetic) – GenSight |
Phase 1/2 |
|
RP (RLBP1) – Novartis |
Phase 1/2 |
|
RP (PDE6A) – Tubingen Hosp |
Phase 1/2 |
|
Retinoschisis (RS1) – NEI |
Phase 1/2 |
|
X-linked RP (RPGR) – AGTC |
Phase 1/2 |
|
X-linked RP (RPGR) – MeiraGTx / Janssen |
Phase 1/2 |
|
X-linked RP (RPGR) – 4DMT |
Phase 1/2 |
|
X-linked RP (RPGR) – Biogen (Nightstar) |
Phase 2/3 |
|
|
|
CELL-BASED THERAPIES |
PROGRESS |
|
AMD-dry (RPE) – Astellas |
Phase 1/2 |
|
AMD-dry (RPE) – Cell Cure |
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 |
|
RP, Usher (retinal progenitors) – ReNeuron |
Phase 2 |
|
Stargardt (RPE) – Astellas |
Phase 1/2 |
|
|
|
MOLECULES, PROTEINS, AONS |
PROGRESS |
|
AMD-dry (C3 inhibitor) – Apellis |
Phase 3 |
|
AMD-dry (CB inhibitor) – Ionis |
Phase 2 |
|
AMD-dry (C5 inhibitor) – Iveric bio |
Phase 2 |
|
LCA (CEP290, AON) – ProQR |
Phase 2/3 |
|
LCA (CEP290, CRISPR) – Editas |
Phase 1/2 |
|
RP (RHO, AON) – ProQR |
Phase 1/2 |
|
Stargardt disease (emixustat) – Acucela |
Phase 3 |
|
Stargardt disease (deuterated vit A) – Alkeus |
Phase 2 |
|
Stargardt disease (C5 inhibitor) – IVERIC bio |
Phase 2 |
|
Stargardt disease (anti-RBP4) – Belite Bio |
Phase 1 |
|
Stargardt disease (anti-RBP4) – Stargazer |
Phase 2 |
|
Usher syndrome (NACA-anti-oxidant) – Nacuity |
Phase 1/2 |
|
Usher syndrome 2A (AON) – ProQR |
Phase 1/2 |
Information current as of March 2021.
