Celine Halioua
Celine Halioua

List of Ocular Gene Therapies in Active Clinical Trials

As of May 2019. This is conservative in so far that I removed any where I couldn’t find recent confirmation of its development.

Developer - vector name - stage of clinical development - specific mutation targeted if relevant - whether still in development


Achromatopsia

1.MeiraGTx – AAV-CNGA3 – Phase I/II – CNGA3 – still in development

2.Applied Genetic Technologies Corp. – AGTC-402 – I/II – CNGA3 – still in development

3.STZ Eyetrial – rAAV.hCNGA3 – I/II – CNGA3 – still in development

4.Applied Genetic Technologies Corp. – rAAV2tYF-PR1.7-hCNGB3– I/II – CNGB3 – still in development

5.MeiraGTx – AAV-CNGB3 – I/II – CNGB3 – still in development

Wet Age-Related Macular Degeneration

1.Sanofi Genzyme – AAV2-sFLT01 – I – still in development

2.Adverum Biotechnologies – ADVM-022 – I – still in development

3.Ocular Therapeutics – OKX-TKI – I

4.Oxford BioMedica – RetinoStaat – I – still in development

5.Regenxbio Inc – RGX-314 – I - still in development

6.Adverum Biotechnologies – rAAV.sFlt-1 – I/II – still in development

7.Hemera Biosciences – AAVCAGsCD59 (HMR59) – I – still in development, I  - same product as the dry AMD

Choroideremia

1.Nightstar Therapeutics – AAV2-REP1 – III – still in development

2.Spark Therapeutics – AAV2-REP1 – I/II – still in development

Geographic Atrophy

1.Gyroscope Therapeutics – GT005 – I/II – still in development

2.Hemera Biosciences – AAVCAGsCD59 (HMR59) – I  - same product as the wet AMD

Leber Congenital Amaurosis

1.Allergan & Editas – AGN-151587 – I/II – LCA10 – still in development

2.ProQR – QR-110 – I/II – LCA10 – still in development

3.Sanofi - SAR439483 – I/II – GUCY2D – still in development

4.Spark Therapeutics – AAV2-RPE65 – III – RPE65 *market approved

5.Applied Genetic Technologies Corp – AAV2-CB-RPE65 – I/II – RPE65 - still in progress

6.MeiraGTx – AAV2/5-OPTIRPE65 – I/II - RPE65 – still in progress

1.ProQR Therapeutics – QR-110 – I/II – CEP290

Leber Hereditary Optic Neuropathy

1.GenSight – GSO10 – III – G11778A – still in progress

Retinitis Pigmentosa

1.Allergan – RST-001 – I/II – advanced RP – still in progress (uncertain)

2.GenSight – rAAV2.7m8-CAG-ChrimsonR-tdTomato (GS030) – I/II – non-syndromic – still in development

3.Horama SA – AAV2/5-hPDE6B – I/II – PDE6B – still in progress

4.Novartis – CPK850 AAV8 – I/II – RLBP1 – still in progress

5.Applied Genetic Technologies Corp. - rAAV2tYF-GRK1-RPGR – I/II – RPGR – still in development

6.MeiraGTx – AAV-RPGR – I/II – RPGR – still in development

7.Nightstar Therapeutics -  AAV-RPGR – I/II – RPGR – still in development

Ushers

1.ProQR - QR-421a – I/II – 2A – still in development

X-linked retinoschisis 

1.Applied Genetic Technologies Corp. - rAAV2tYF-CB-hRS1 – I/II – RS1 – still in development


Again, credit to Jasleen Jolly for her original ocular gene therapy database