What side effects are associated with this type of intervention?

Common treatments for Beta Thalassemia include regular blood transfusions and iron chelation therapy. Blood transfusions can lead to iron overload, requiring chelation therapy, which may cause gastrointestinal issues, kidney and liver dysfunction, and allergic reactions. Emerging gene editing therapies, like those studied in EDIT-301, are still being evaluated, but potential side effects could include off-target genetic changes, immune responses, and long-term unknown effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Beta Thalassemia, including gene therapy approaches. One notable approval is Zynteglo (betibeglogene autotemcel), a gene therapy that adds functional copies of the beta-globin gene into a patient's hematopoietic stem cells. This therapy aims to reduce or eliminate the need for blood transfusions in patients with transfusion-dependent Beta Thalassemia. While EDIT-301 is a gene editing therapy under investigation, it represents a similar advanced approach to correcting the genetic defect in Beta Thalassemia by directly editing the patient's DNA to produce functional hemoglobin.

What other types of research exist?

Promising alternatives to EDIT-301 for beta thalassemia patients include CRISPR/Cas9-mediated gene editing, which aims to correct the genetic defect at the HBB locus. However, improvements in the fidelity and specificity of this platform are necessary before clinical application. Additionally, RNA trans-splicing targeting endogenous β-globin pre-messenger RNA in human erythroid cells is another novel approach that has shown potential in preclinical studies. These methods provide a basis for future gene therapy advancements.

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Gene Therapy

EDIT-301 for Beta Thalassemia

Phase 1 & 2

Recruiting

Research Sponsored by Editas Medicine, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of Transfusion Dependent B-Thalassemia as defined by documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records

History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent

Must not have

Inadequate organ function

Available 10/10 human leukocyte antigen (HLA)-matched related donor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up edit-301 infusion (day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/l for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post edit-301 infusion.

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new treatment called EDIT-301, which modifies a patient's own stem cells to treat severe beta Thalassemia. It targets adults who need regular blood transfusions. The goal is to fix their cells so they can produce healthy blood cells and reduce the need for transfusions.

Who is the study for?

This trial is for adults with Transfusion-Dependent Beta Thalassemia, who have needed regular blood transfusions and are in stable condition to undergo a stem cell transplant. They should not have had previous transplants or gene therapy, no significant organ issues, infections, other hemoglobin disorders, cancer or immunodeficiency.

What is being tested?

The study tests EDIT-301's safety and effectiveness in treating Beta Thalassemia. Participants will receive this treatment before undergoing an autologous Hematopoietic Stem Cell Transplant (HSCT), where they use their own stem cells.

What are the potential side effects?

While specific side effects of EDIT-301 aren't listed here, similar treatments may cause immune reactions, infection risk increase due to bone marrow involvement, fatigue from the procedure and potential organ inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with Transfusion Dependent B-Thalassemia.

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I've had significant blood transfusions in the last 2 years.

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I am mostly able to care for myself.

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I am healthy enough for a stem cell transplant using my own cells.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My organs are not functioning properly.

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I have a donor who is a perfect match for my bone marrow transplant.

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I have had a stem cell transplant or cannot have one due to health reasons.

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I have a documented history of inherited blood disorders like sickle cell or thalassemia.

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A close family member has or might have a hereditary cancer syndrome.

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I have previously received gene therapy.

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I have had cancer or an immune system disorder before.

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My liver disease is in an advanced stage.

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My white blood cell count is low, or my platelet count is below the normal range.

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I do not have any active serious infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ edit-301 infusion (day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/l for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post edit-301 infusion.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~edit-301 infusion (day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/l for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post edit-301 infusion.

This trial's timeline: 3 weeks for screening, Varies for treatment, and edit-301 infusion (day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/l for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post edit-301 infusion. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Kinetics of HSPC engraftment

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: EDIT-301Experimental Treatment1 Intervention

EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Beta Thalassemia include regular blood transfusions, iron chelation therapy, and hematopoietic stem cell transplantation (HSCT). Blood transfusions provide patients with healthy red blood cells, but they can lead to iron overload, which is managed by iron chelation therapy. HSCT offers a potential cure by replacing the defective hematopoietic stem cells with healthy ones. Gene editing therapies, like those being studied in the EDIT-301 trial, aim to correct the genetic defect causing Beta Thalassemia at the DNA level. This approach has the potential to provide a long-term cure by enabling the patient's own cells to produce functional hemoglobin, reducing or eliminating the need for transfusions and chelation therapy. This is particularly important for Beta Thalassemia patients as it addresses the root cause of the disease and can significantly improve their quality of life.