EDIT-101 is under clinical development by Editas Medicine and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect EDIT-101’s likelihood of approval (LoA) and phase transition for Leber Congenital Amaurosis (LCA) took place on 04 Oct 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their EDIT-101 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

EDIT-101 overview

AGN-151587 is under development for the treatment of Leber congenital amaurosis 10. It is developed based on CRISPR/Cas9 and CRISPR/Cpf1 genome editing technology. It is administered through the subretinal route. The therapeutic candidate constitutes a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene-editing constructs. It is a new molecular entity. It acts by targeting CEP290.

Editas Medicine overview

Editas Medicine formerly Gengine, is a clinical stage biotechnology company focused on the development of genome treatments for rare diseases. The company is developing a proprietary genome editing platform based on its in-licensed clustered regularly interspaced short palindromic repeats (CRISPR) technology. The company’s pre-clinical pipeline spans investigational candidates for the treatment of ocular and blood diseases, cancer, and diseases of other organs and tissues. The company works in collaboration with Allergan/ AbbVie Inc, AskBio, Bayer AG, BlueRock Therapeutics, The Broad Institute Inc, Massachusetts Institute of Technology, Harvard Institute, The Rockefeller University, Celgene Corp and Bristol Myers Squibb to discover and develop its pipeline products. Editas Medicine is headquartered in Cambridge, Massachusetts, the US.

Quick View EDIT-101 LOA Data

Report Segments
  • Innovator
Drug Name
  • EDIT-101
Administration Pathway
  • Intraocular
Therapeutic Areas
  • Ophthalmology
Key Developers
Highest Development Stage
  • Phase II


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.