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 (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.