Editas Medicine is at the forefront of gene editing technology, primarily utilizing CRISPR (Clustered, Regularly Interspaced, Short Palindromic Repeats) to develop innovative genomic medicines aimed at treating serious diseases. The company employs two distinct CRISPR nucleases, Cas9 and Cas12a (also known as Cpf1), each with unique capabilities for gene editing. Cas9 is a programmable protein that targets specific DNA sequences, creating double-stranded breaks that the cell then repairs, allowing for precise edits. In contrast, Cas12a offers advantages in targeting unique DNA sequences and making staggered cuts, enhancing the efficiency and accuracy of gene repair. This dual approach enables Editas Medicine to address a wide range of genetic mutations and develop both in vivo (inside the body) and ex vivo (outside the body) gene editing therapies.

The company's research focuses on creating transformative medicines for conditions with limited treatment options, such as sickle cell disease and beta-thalassemia. One of their leading programs, renizgamglogene autogedtemcel (reni-cel), utilizes CRISPR/Cas12a to edit patient-derived hematopoietic stem cells, aiming to increase fetal hemoglobin production and provide durable treatment benefits. Additionally, Editas Medicine is collaborating with Bristol Myers Squibb to enhance the efficacy of cancer immunotherapies by editing T cells to improve their ability to target and destroy cancer cells. Through these innovative approaches, Editas Medicine is committed to translating the potential of gene editing into effective therapies that can significantly improve patient outcomes.

United States

At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.

Cancer

Genetic Diseases

Hematologic Diseases

Editas Medicine

Genetic disorder

Sickle cell disease

EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.
Gene editing

Reni-cel (renizgamglogene autogedtemcel) (EDIT-301)

Eye disease

Hepatitis C

Rare diseases

Rare genetic disease

EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
Gene editing

Editas Medicine

Company Details