EDIT-301 for Beta Thalassemia

Recruiting at 7 trial locations

Overseen By Editas Medicine Clinical Trial Team

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Editas Medicine, Inc.

Disqualifiers: Alpha thalassemia, Hemoglobinopathy, Malignancy, others

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more details.

What makes the treatment EDIT-301 unique for beta thalassemia?

EDIT-301 is unique because it uses genome editing to directly correct genetic mutations causing beta thalassemia, potentially offering a more targeted and long-lasting solution compared to traditional treatments like blood transfusions or iron chelation therapy. ^{1 2 3 4 5}

Eligibility Criteria

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.

Inclusion Criteria

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

I have been diagnosed with Transfusion Dependent B-Thalassemia.

I am mostly able to care for myself.

Exclusion Criteria

My organs are not functioning properly.

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

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

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of EDIT-301 via intravenous infusion

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including HSPC engraftment

24 months

Treatment Details

Interventions

• EDIT-301 (Gene Therapy)

Trial Overview 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.

Participant Groups

Group I: EDIT-301 Experimental Treatment 1 Intervention

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

EDIT-301 is already approved in Canada for the following indications:

Approved in Canada as EDIT-301 for:

• None approved yet; currently in clinical trials for Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Who Is Running the Clinical Trial?

Editas Medicine, Inc.

Lead Sponsor

Trials

5

Recruited

170+

Findings from Research

A novel mutation in the promoter region of the β-globin gene, identified as HBB: c.-127G > C, was discovered in a family with suspected β-globin mutations, indicating its potential role in β-thalassemia.

The study found that this mutation is associated with a mild phenotype of β-thalassemia, as evidenced by hematological and clinical evaluations of the family members carrying the mutation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Novel Mutation in the Promoter Region of the β-Globin Gene: HBB: c.-127G > C. Bilgen, T., Canatan, D., Delibas, S., et al.[2017]

A new β(0) frameshift mutation (HBB: c.44delT) was identified in a family with β-Thalassemia intermedia, highlighting the genetic complexity of the condition.

The study emphasizes the role of secondary genetic modifiers in influencing the severity of β-Thalassemia intermedia, which can help in better identifying and understanding the disease within families.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A new β(0) frameshift mutation, HBB: c.44delT (p.Leu14ArgfsX5), identified in an Argentinean family associated with secondary genetic modifiers of β-thalassemia. Pepe, C., Eberle, SE., Chaves, A., et al.[2014]

A new base editing strategy has been developed that can correct the HbE mutation responsible for severe β-thalassaemia, achieving over 90% editing efficiency in primary human CD34+ cells.

This approach not only targets the mutation effectively but also demonstrates safety by profiling off-target effects and using machine-learning methods to predict potential impacts, indicating a promising avenue for treating or preventing severe thalassaemia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Direct correction of haemoglobin E β-thalassaemia using base editors. Badat, M., Ejaz, A., Hua, P., et al.[2023]

β-thalassemia intermedia (β-TI) is a complex condition characterized by varying levels of anemia, and its management includes traditional treatments like transfusion therapy and splenectomy, as well as newer approaches such as genome editing and novel erythropoiesis-stimulating agents.

Recent advancements in molecular therapies, including CRISPR/Cas9 and epigenetic modulation, show promise in improving treatment outcomes for β-TI by targeting the underlying genetic causes of the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An Expert Overview on Therapies in Non-Transfusion-Dependent Thalassemia: Classical to Cutting Edge in Treatment. Saeidnia, M., Fazeli, P., Farzi, A., et al.[2023]

Genetic polymorphisms and mutations significantly influence the severity and clinical manifestations of β-thalassemia, including the need for blood transfusions, as identified through a review of relevant studies since 1982.

Increased production of hemoglobin F, influenced by specific genetic alterations, can alleviate symptoms of β-thalassemia, suggesting that understanding these genetic factors can help in predicting disease prognosis and guiding early interventions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Influence of Polymorphisms in Disease Severity in β-Thalassemia. Mohammdai-Asl, J., Ramezani, A., Norozi, F., et al.[2015]

References

1. England pubmed.ncbi.nlm.nih.gov

A Novel Mutation in the Promoter Region of the β-Globin Gene: HBB: c.-127G > C. [2017]

2. England pubmed.ncbi.nlm.nih.gov

A new β(0) frameshift mutation, HBB: c.44delT (p.Leu14ArgfsX5), identified in an Argentinean family associated with secondary genetic modifiers of β-thalassemia. [2014]

3. England pubmed.ncbi.nlm.nih.gov

Direct correction of haemoglobin E β-thalassaemia using base editors. [2023]

4. England pubmed.ncbi.nlm.nih.gov

An Expert Overview on Therapies in Non-Transfusion-Dependent Thalassemia: Classical to Cutting Edge in Treatment. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

The Influence of Polymorphisms in Disease Severity in β-Thalassemia. [2015]