Retinal Pigmentosa, Leber congenital amaurosis (LCA) - Healing Genes

Retinal Pigmentosa, Leber congenital amaurosis (LCA)

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Single Ascending Dose Study in Participants With LCA10

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of AGN-151587 (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”)

Phase 1/2


Doctors at the Massachusetts Eye and Ear Infirmary are seeking adult and pediatric patients with Leber Congenital Amaurosis Type 10 (LCA10) with specific genetic mutations causing retinal degeneration. The drug uses a genetic editing tool, delivered only within the retina, designed to eliminate the harmful mutation in the CEP290 gene. It uses a novel gene editing tool that works like a find-and-replace system, cutting out the mutated DNA and restoring normal protein expression and function of remaining retinal cells.

Treatment will involve a single injection surgery to the retina at the back of the eye, then follow up for 1 year.


  • Be 3 years of age and older
  • Meet genetic screening requirements specific to the study aim
  • Not have had prior gene therapy or oligonucleotide treatment


  1. Screening before the treatment
  2. A single injection of the genetic therapy to the retina at the back of the eye
  3. Follow up for 1 year


The study location is at the Massachusetts Eye and Ear Infirmary. Map.

Contact: Clinical Trials Registry Team  |  1-877-277-8566  |  [email protected]




Editas Medicine, Inc.


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