Editas Medicine
Editas Medicine works on 3 rare diseases tracked on Trial Friend, including Leber Congenital Amaurosis, Sickle Cell Disease, Thalassemia, with 2 recruiting clinical trials.
Editas Medicine develops CRISPR/Cas9 gene-editing therapies for rare genetic diseases. EDIT-101, an in vivo CRISPR therapy for Leber congenital amaurosis type 10 (LCA10), was the first in vivo CRISPR medicine dosed in patients but enrollment was paused due to the small eligible population. In late 2024 the company undertook a major restructuring, discontinuing its ex vivo reni-cel program and pivoting to in vivo gene editing approaches.
Focus areas at Editas Medicine
As a rare disease specialist, Editas Medicine has active clinical trial programs and drug development efforts across 3 rare diseases, including Leber Congenital Amaurosis, Sickle Cell Disease, Thalassemia. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Editas Medicine, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Editas Medicine is headquartered in Cambridge, United States, founded in 2013, publicly traded under the ticker symbol EDIT. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Editas Medicine Drug Pipeline
Editas Medicine has 2 active clinical trials across 1 development stage, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Editas Medicine's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Editas Medicine Clinical Trials (2)
Active and recruiting clinical trials sponsored by Editas Medicine, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Editas Medicine Trial Locations
Editas Medicine clinical trials are running at 32 sites in 2 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (3)
Diseases targeted by Editas Medicine's clinical trial and drug development programs
Leber congenital amaurosis is a group of rare inherited retinal dystrophies that cause severe vision loss or blindness from birth or early infancy. The condition results from mutations affecting retin...
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes severe anemia, organ damage from iron overload, and bone abnormalities. Severe form...
Patient Resources
Organizations and resources related to Editas Medicine's rare disease focus areas
Frequently Asked Questions About Editas Medicine
Common questions about Editas Medicine's rare disease programs, clinical trials, and treatments.