Clinical Trial Pipeline

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) – Nightstar

Phase 3

Choroideremia (REP1) – Spark

Phase 1/2

Choroideremia (REP1) – Tubingen Hosp

Phase 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) – GenSight

Phase 1/2

RP (RLBP1) – Novartis

Phase 1/2

Retinoschisis (RS1) – AGTC

Phase 1/2

Retinoschisis (RS1) – NEI

Phase 1/2

Stargardt disease (ABCA4) – Sanofi

Phase 1/2

Usher syndrome 1B (MYO7A) – Sanofi

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 3 Pen.

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

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 (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 2A (AON) – ProQR

Phase 1/2