Clinical Trials in the Pre-Start Phase:

1. A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene. ClinicalTrials.gov ID NCT06627179
2. Global Patient Registry of Inherited Retinal Diseases. ClinicalTrials.gov ID NCT05957276

Clinical Trials in the Recruitment Phase:

1. A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene  (ASTRA).
2. Rate of Progression in USH2A Related Retinal Degeneration (RUSH2A). ClinicalTrials.gov ID NCT03146078.
3. Ascidian Therapeutics, Inc. / “Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants with ABCA4-related Retinopathy” (Stellar). ClinicalTrials.gov ID NCT06467344
4. A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Participants with X-linked Retinitis Pigmentosa (Dawn). ClinicalTrials.gov ID NCT06275620
5. A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects with RS1-Associated X-linked Retinoschisis (LIGHTHOUSE). ClinicalTrials.gov ID NCT05878860
6. An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (POLARIS). ClinicalTrials.gov ID NCT06435000
7. Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants (UniRare)
8. A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants with X-linked Retinitis Pigmentosa (VISTA). ClinicalTrials.gov ID NCT04850118.
9.  A phase i/ii study to assess the safety and tolerability of a single subretinal administration of spvn06 gene therapy in subjects with rod-cone dystrophy (rcd) due to a mutation in the rho, pde6a, or pde6b gene (prodygy). ClinicalTrials.gov ID NCT05748873

Clinical Trials Closed to Enrollment:

1. A Natural History and Outcome Measure Discovery Study of PRPF31 Mutation–Associated Retinal Dystrophy (Quokka). ClinicalTrials.gov ID NCT05573984
2. A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy (Platypus). ClinicalTrials.gov ID NCT05902962
3. A Phase 1/2 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Repeatedly Administered Intravitreally VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy (Wallaby). ClinicalTrials.gov ID NCT06455826
4. A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa (RP) associated with NR2E3 and RHO mutations and Leber Congenital Amaurosis (LCA) with mutation(s) in CEP290 gene. ClinicalTrials.gov ID NCT05203939
5. The Natural History of LCHAD Retinopathy
6. A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (SOLSTICE). ClinicalTrials.gov ID NCT03584165
7. A Phase 1/2 Dose Escalation Study of Subretinally Injected ATSN-101 Administered in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D. ClinicalTrials.gov ID NCT03920007
8. An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males with X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene. ClinicalTrials.gov ID NCT04517149
9. Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS). ClinicalTrials.gov ID NCT04127006
10. An Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) in Subjects with X-linked Retinitis Pigmentosa caused by RPGR-ORF15 mutations (Horizon). ClinicalTrials.gov ID NCT03316560
11. A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects with X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene (Skyline). ClinicalTrials.gov ID NCT06333249
12. A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC-401, a Recombinant Adeno-associated Virus Vector Expressing CNGB3, in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene. ClinicalTrials.gov ID NCT02599922
13. A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC-402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients with Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene. ClinicalTrials.gov ID NCT02935517
14. An open label study to determine the longterm safety, tolerability and biological activity of SAR422459 in patients with Stargardt’s macular degeneration. ClinicalTrials.gov ID NCT01736592
15. An open-label study to determine the long-term safety, tolerability and biological activity of SAR421869 in patients with Usher syndrome Type 1B. ClinicalTrials.gov ID NCT02065011
16. Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene (“LCA10-IVS26”). ClinicalTrials.gov ID NCT03872479
17. A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated with Voretigene Neparvovec in US.