Editas Medicine

Editas Medicine specializes in gene editing to develop novel therapeutics for a variety of areas including oncology, ocular diseases, and blood diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 and Cas12a enzymes. 

The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, Editas has seven gene editing programs in its pipeline. 

One of the company’s candidates was in the late clinical trial stages as of March 2024. The Phase I/II clinical trials on Editas’ in vivo gene editing therapy EDIT-101 have been ongoing since 2019 and the company announced that it had achieved proof of concept and identified a responder population in November 2022. For the first time ever (in April 2022), the company delivered its experimental in vivo CRISPR therapy to a pediatric patient to treat Leber congenital amaurosis 10 (LCA10), an inherited early childhood eye disease. Its ex vivo gene editing therapy EDIT-301 holds two rare pediatric disease designations granted by the FDA for transfusion-dependent beta-thalassemia and sickle cell disease. The candidate also received orphan drug designation in May 2022 for beta-thalassemia.

Key customers and partnerships

Founded in September 2013, Editas has secured several collaborations with other pharmaceutical and drug developers including Catalent (for therapy development and manufacturing support), Adverum Biotechnologies (to develop technologies to deliver gene editing medicines to the eye), Asklepios BioPharmaceutical (AskBio) (to research in vivo gene editing therapies for neurological diseases), BlueRock Therapeutics (to develop medicines by combining cell therapy and gene editing technologies), and big pharma such as Bristol-Myers Squibb (to develop cell therapies for oncology). 

The company expanded its partnership with Azzur Cleanrooms on Demand (COD) in July 2023 with regard to continuing to manufacture Editas' candidate EDIT-301.

Funding and financials

The company last raised funds in June 2023 when the company raised USD 125 million in a common stock offering. Editas Medicine did not disclose what the raised funds would be used for. The company has been listed on the Nasdaq since February 2016 and was the first CRISPR gene editing company to go public.