EDIT-301 is under clinical development by Editas Medicine and currently in Phase II for Sickle Cell Disease. According to GlobalData, Phase II drugs for Sickle Cell Disease have a 59% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how EDIT-301’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

EDIT-301 overview

EDIT-301 is under development for the treatment of beta-thalassemia and sickle cell disease. It is developed based on CRISPR/Cas12a genome editing technology. The therapeutic candidate constitutes ex vivo treated stem cells for the B-cell CLL/lymphoma 11 A (BCL11A) gene. The cells are modified for the expression of BCL11A gene using CRISPR/Cas12a ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus and guide RNA molecules to eliminate the mutation in the BCL11A gene. The gene is delivered using adeno-associated virus (AAV) vector to introduce normal copies of gene into the cells. The drug candidate is administered through intravenous route.

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, BlueRock Therapeutics, Bayer AG, 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.

For a complete picture of EDIT-301’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 2 September 2023

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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.