What rare diseases does Editas Medicine focus on?

Editas Medicine has active programs across 3 rare diseases: Leber Congenital Amaurosis, Sickle Cell Disease, Thalassemia. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is Editas Medicine focused exclusively on rare diseases?

Yes. Editas Medicine is a rare disease specialist headquartered in Cambridge, United States, founded in 2013. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Leber Congenital Amaurosis, Sickle Cell Disease, Thalassemia.

How can I find Editas Medicine clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Editas Medicine, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in an Editas Medicine clinical trial?

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Editas Medicine have?

None that we track as of November 2026. No Editas Medicine label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions. In development: the trials list on this page shows what Editas Medicine is recruiting for now, and the FDA calendar lists any application once a decision date is public.

When was Editas Medicine founded and where is it based?

Editas Medicine was founded in 2013 and is headquartered in Cambridge, United States. The company has been active for 13 years. It trades publicly under the ticker symbol EDIT. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.

Editas Medicine

Rare disease drugmaker · based in Cambridge, United States · founded 2013 · ticker EDIT · editas.com

Editas Medicine works on 3 rare diseases we track, including Leber Congenital Amaurosis, Sickle Cell Disease and 1 more. It has 2 active clinical trials, though none are looking for new volunteers right now.

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.

Editas Medicine pipeline

Each row lists the diseases Editas Medicine is studying at that stage, from the ones closest to approval down to the earliest work.

Phase 2

Mid-size studies that check whether a drug works and at what dose.

Thalassemia · Sickle Cell Disease

Editas Medicine clinical trials

These are Editas Medicine trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

2 trials underway, not taking new volunteers

Underway, not taking new volunteersPhase 1/2Hasn't posted an update in over a yearNCT05444894

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Tests EDIT-301

For Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major and Thalassemia Intermedia

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Ages 18 Years - 35 Years8 locations

View full study

Underway, not taking new volunteersPhase 1/2Hasn't posted an update in over a yearNCT04853576

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

Tests EDIT-301

For Sickle Cell Disease and Hemoglobinopathies

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).

Ages 12 Years - 50 Years24 locations

View full study

Ask about Editas Medicine's trials

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Trial Alerts, when a new trial opens or a trial changes
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More about 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 active and recruiting rare disease 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.

Where Editas Medicine trials take place

The trials above run at 32 study sites in 2 countries. Tap a country to see its states, cities and hospitals.

United States

28▼

Canada

4▼

Rare diseases Editas Medicine works on

Each disease page lists every open trial, not only Editas Medicine's, plus approved treatments.

Leber Congenital Amaurosis

Leber congenital amaurosis is a group of rare inherited retinal dystrophies that cause severe vision loss or blindness from birth or early infancy.

How common: 2 to 3 in every 100,000 newborns

Sickle Cell Disease

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.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Thalassemia

Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes anemia, from mild or none in carriers to severe in the major forms, where it can...

How common: About 1 in 100,000 people have symptoms of beta thalassemia, according to NORD, most often people with Mediterranean, Middle Eastern, African or Asian roots

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Editas Medicine's own site is editas.com, and our help paying for treatment page lists drugmaker programs and charity funds.

NIH GARD - Leber Congenital Amaurosis
Leber Congenital Amaurosis and Sickle Cell Disease
Foundation Fighting Blindness - LCA
Leber Congenital Amaurosis
Sickle Cell Disease Association of America
Sickle Cell Disease
Cooley's Anemia Foundation
Thalassemia
Thalassaemia International Federation
Thalassemia