LCA Therapy Development Pipeline
Understanding the LCA Therapy Development Pipeline
Leber congenital amaurosis (LCA) is caused by mutations in more than 27 different genes, and researchers around the world are working to develop therapies for many of them. This page is designed to provide a snapshot of the current LCA treatment landscape, including therapies in clinical trials and those being studied in pre-clinical development. While not every gene currently has a therapy in development, research is continuing to grow and evolve. We hope this resource helps individuals and families better understand where progress is being made and what the path toward future treatments may look like.
What’s the difference between pre-clinical and clinical development?
Pre-clinical development includes therapies being studied in the lab or in animal models before they can move into human testing.
Clinical development includes therapies that have advanced into human clinical trials to evaluate safety and effectiveness.
Also, there are additional gene-agnostic therapies (e.g., optogenetics, cells) not listed below that may become relevant to LCA. For those who would like to learn more about the terms and concepts mentioned on this page, please visit our Glossary for additional information and definitions. If you have questions about emerging therapies, send an email to info@hopeinfocus.org.
Last updated: April 2026
PRE-CLINICAL PIPELINE FOR EMERGING LCA THERAPIES (SELECT PROJECTS)
| TYPE-GENE | MODALITY | SPONSOR | STATUS |
|---|---|---|---|
| LCA3 (SPATA7) | Gene therapy | Baylor | Efficacy in mouse model |
| LCA6 (RPGRIP1) | Gene therapy | Odylia Therapeutics | Safety in toxicology study in preparation for potential clinical trial |
| LCA7 (CRX) and potentially LCA10 (CEP290), NPHP1, and other retinal ciliopathies | Gene therapy | Variant | Approaching clinical trial. Efficacy in CRX, CEP290, and NPHP1 models |
| LCA8 (CRB1) | Gene therapies | Duke, Leiden, and others | Efficacy in mouse and organoid models. Developing pig model. Additional disease mechanism studies ongoing |
| LCA9 (NMNAT1) | Gene therapy | Opus Genetics | Efficacy in LCA9 mouse model |
| LCA9 (NMNAT1) | NAC (oral small molecule) | Mass Eye and Ear | Efficacy in LCA9 mouse model |
| LCA13 (RDH12) | Gene therapy | Opus Genetics | Partnership with Global RDH12 Alliance. Working toward clinical trial authorization |
| LCA16 (KCNJ13) | Gene therapy | Hubble Therapeutics | Efficacy in mouse and cell models |
| LCA16 (KCNJ13) | CRISPR gene editing | University of Wisconsin-Madison | Efficacy in mouse and cell models |
| NPHP5 (IQCB1) | Gene therapy | NIH – Bespoke Gene Therapy Consortium | Efficacy in canine model. Approaching clinical trial |
LCA CLINICAL DEVELOPMENT PIPELINE CHART
| TYPE-GENE | MODALITY | SPONSOR | STAGE | COMMENTS |
|---|---|---|---|---|
| LCA (GUCY2D) | AAV gene therapy (GUCY2D) | Atsena Therapeutics | Planning Phase 3 | Vision improvements in Phase 1/2 |
| LCA2 (RPE65) | AAV gene therapy (RPE65) | Genentech | LUXTURNA®: Approved by FDA, EMA | Hundreds dosed after approval, most with vision improvements |
| LCA2 (RPE65) | AAV gene therapy (RPE65) | HuidaGene Therapeutics | Phase 1/2 | Vision improvements reported |
| LCA4 (AIPL1) | AAV gene therapy (AIPL1) | MeiraGTx Eli Lilly | MHRA Specials License | Vision improvement for 11 young children |
| LCA5 | AAV gene therapy (LCA5) | Opus Genetics | Recruiting Phase 3 | Vision improvements in Phase 1/2 |
| LCA10 (CEP290) (IVS26 mutation) | RNA therapy targeting IVS26 mutation in CEP290 | Sepul Bio | Recruiting Phase 3 | Vision improvements in the previous Phase 1/2 and 2/3 |