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

PROGRESS

Achromatopsia (CNGB3) – AGTC

Phase 1/2

Achromatopsia (CNGB3) – MeiraGTx

Phase 1/2

Achromatopsia (CNGA3) – AGTC

Phase 1/2

Achromatopsia (CNGA3) – Tubingen Hosp

Phase 1/2

AMD (Dry) – Gyroscope

Phase 1/2

Choroideremia (REP1) – 4DMT

Phase 1/2

Choroideremia (REP1) – 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

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

Phase 1/2

X-linked RP (RPGR) – 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

Usher syndrome (NACA-anti-oxidant) – Nacuity

Phase 1/2

Usher syndrome 2A (AON) – ProQR

Phase 1/2

Information current as of September 2020.