The Foundation Fighting Blindness is the world’s leading private funding source for research to prevent, treat, and cure retinal degenerative diseases. Many of the trials listed below were made possible by Foundation support.
Genetic Technologies
GENE THERAPIES (GENE TARGET) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| Achromatopsia (CNGA3) | Tubingen Hosp | Phase 1/2 |
| AMD-dry, GA (CD59) | Janssen | 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 |
| Best disease (BEST1) | Opus Genetics | Phase 1/2 |
| LCA (GUCY2D) | Atsena | Phase 1/2 |
| LCA (LCA5) | Opus Genetics | Phase 1/2 |
| RP (CNGA1) | VeonGen | Phase 1 |
| RP (RLBP1) | Novartis | Phase 1/2 |
| RP (NR2E3, gene agnostic) | Ocugen | Phase 3 |
| RP (RdCVF, gene agnostic) | SparingVision | Phase 1/2 |
| RP (PDE6A) | Tubingen Hosp | Phase 1/2 |
| Stargardt disease (RORA) | Ocugen | Phase 2/3 |
| Stargardt disease (ABCA4) | Splice Bio | Phase 1/2 |
| Stargardt disease (ABCA4) | VeonGen | Phase 1/2 |
| Usher syndrome (MYO7A) | AAVantgarde | Phase 1/2 |
| X-linked Retinoschisis (RS1) | Atsena | Phase 1/2/3 |
| X-linked RP (RPGR) | Beacon | Phase 2/3 |
| X-linked RP (RPGR) | Janssen | Phase 3 |
RNA THERAPIES (MECHANISM) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| LCA10 (AON targeting CEP290 IVS26) | Sepul Bio | Phase 3 |
| RP11 (AON targeting CNOT3) | PYC | Phase 1/2 |
| RP, Usher (AON targeting USH2A exon 13) | Sepul Bio | Phase 2 |
| Stargardt disease (ABCA4 RNA editing) | Ascidian | Phase 1/2 |
Novel Medical Therapies
SMALL MOLECULES, PROTEINS (INVESTIGATIONAL PRODUCT) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| AMD-dry, GA (Gildeuretinol) | Alkeus | Phase 2/3 |
| AMD-dry, GA (ANX007) | Annexon | Phase 3 |
| AMD-dry, GA (Tinlarebant) | Belite Bio | Phase 3 |
| AMD-dry, GA (BRX001) | Biojiva | Phase 1/2 |
| AMD-dry, GA (BI 1584862) | Boehringer Ingelheim | Phase 2 |
| AMD-dry, GA (ONL1204) | ONL Therapeutics | Phase 2 |
| AMD-dry, GA (Elamipretide) | Stealth Bio | Phase 3 |
| AMD-dry, GA (Pozelimab, Cemdisiran) | Regeneron | Phase 3 |
| RP, CHM (KIO-301) | Kiora | Phase 2 |
| RP (Disulfiram) | University of Washington | Phase 1/2 |
| RP (NAC) | Johns Hopkins University | Phase 3 |
| Stargardt disease (Gildeuretinol) | Alkeus | Phase 2 |
| Stargardt disease (Zimura) | Astellas | Phase 2 |
| Stargardt disease (Tinlarebant) | Belite Bio | Phase 3 |
| Stargardt disease (Metformin) | NEI | Phase 1/2 |
| Usher syndrome (NACA) | Nacuity | Phase 1/2 |
| Usher syndrome 3 (BF844) | USH III Initiative | Phase 1 |
Restorative Therapies
CELL-BASED THERAPIES (CELL TYPE) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| AMD-dry, GA (RPE) | Astellas | Phase 1/2 |
| AMD-dry, GA (RPE) | Eyestem | Phase 1/2 |
| AMD-dry, GA (RPE) | Lineage/Genentech | Phase 2 |
| AMD-dry, GA (RPE) | Luxa Bio | Phase 1/2 |
| AMD-dry, GA (RPE) | NEI | Phase 1/2 |
| AMD-dry, GA (RPE) | Regenerative Patch | Phase 2 |
| RP, Usher, CRD (retinal precursors) | BlueRock | Phase 1/2 |
| RP, Usher (retinal progenitors) | jCyte | Phase 2b |
| RP (iPSC retinal sheets) | Sumitomo Pharma | Phase 1/2 |
| RP (RPE) | Centre D'Etude Des Cellules Souches | Phase 1/2 |
OPTOGENTIC THERAPIES (CELL TARGET) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| RP (Cones) | SparingVision | Phase 1 |
| RP (RGCs) | Bionic Sight | Phase 1/2 |
| RP (RGCs) | GenSight | Phase 1/2 |
| RP (BCs) | Nanoscope | Phase 2 |
| RP, CHM (RGCs) | Ray Therapeutics | Phase 1 |
| Stargardt disease (BCs) | Nanoscope | Phase 2 |
RETINAL PROSTHETICS (DEVICE LOCATION) PROGRESS
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| AMD-dry, GA (Subretinal) | Science Corporation Pilot | Phase |
Approved Therapies
| Disease/Condition | Company | Clinical Trial Phase |
|---|---|---|
| AMD-dry, GA (SYFOVRE, C3 inhibitor) | Apellis | Approved |
| AMD-dry, GA (IZERVAY, C5 inhibitor) | Astellas | Approved |
| LCA (LUXTURNA, RPE65) | Spark/Roche | Approved |
Note: This is not intended to be a comprehensive list, but to highlight the diversity of targets and therapeutic modalities. Some trials listed may have been paused and/or the sponsors may be seeking partners to continue their trials. Additional trials and their details can be found at ClinicalTrials.gov and the International Clinical Trials Registry.
