Clinical Trial

Disease: Leber Congenital Amaurosis, LCA, (NCT03872479)

Disease info:

Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy.

Leber congenital amaurosis occurs in 2 to 3 per 100,000 newborns. It is one of the most common causes of blindness in children.
Official title:
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")

United States, Florida

Bascom Palmer Eye Institute, Miami, Florida, United States, 33136


United States, Massachusetts

Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, United States, 02114


United States, Michigan

W.K. Kellogg Eye Center - University of Michigan, Ann Arbor, Michigan, United States, 48105


United States, Oregon

Casey Eye Institute - OSHU, Portland, Oregon, United States, 97239


United States, Pennsylvania

University of Pennsylvania, Philadelphia, Pennsylvania, United States, 19104

Study start:
Sep. 26, 2019
34 participants
Gene editing method:
Type of edit:
Gene correction
Centrosomal Protein 290 (CEP290)
Delivery method:
Adeno-associated virus (AAV5) - In-vivo
EDIT-101 uses an AAV5 vector to deliver the Staphylococcus aureus Cas9 and CEP290-specific guide RNAs (gRNAs) to photoreceptor cells by subretinal injection
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of AGN-151587 (EDIT-101) administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26"). AGN-151587 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.

Last updated: May. 29, 2024
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