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")
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") is a PHASE1 clinical asset sponsored by Editas Medicine, Inc. in Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital. SEO and diligence focus: EDIT-101, endpoint relevance, enrollment feasibility, competitive positioning, readout timing and IP durability.
Indication: Leber Congenital Amaurosis 10
Modality: small molecule
Target: EDIT-101
Sponsor: Editas Medicine, Inc.
Source URL: ClinicalTrials.gov
Source updated: Detailed source ingestion pending
Ingested: Jul 25, 2026
Model: trialsignal-ai-v1
Validation: validated
Matched by target_normalized: EDIT-101
View original source fields
Condition raw: Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Condition normalized: Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Modality raw: small molecule
Modality normalized: small molecule
Target raw: EDIT-101
Target normalized: EDIT-101