Editas Medicine to Host Virtual Event to Highlight Initial Clinical Data from the RUBY Trial of EDIT-301 for Severe Sickle Cell Disease

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The clinical data will include safety data from the first two patients and efficacy data from the first patient treated with EDIT-301.

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GlobeNewswire, Inc.GlobeNewswire, Inc.
Retrieved on: 
Thursday, December 1, 2022
Information
Tags: 
TDT, Polymerization, Anemia, HBF, CD34, Event, Mutation, HBG1, Fetal hemoglobin, Patient, SCD, Broad Institute, Harvard University, Sickle cell disease, Patent, HBS, Editas Medicine, NCT, CRISPR, Safety, Manifest and latent functions and dysfunctions, Pharmaceutical industry, Vaccine, HBG2
Geotags: 
CAMBRIDGE, MA, US, UNITED STATES
⚠ partially generated by AI ↴
Organisation: 
EDITAS MEDICINE, EDITAS MEDICINE, INC., SICKLE CELL DISEASE, EDIT-301, RUBY
Summary
Key Points: 
  • The clinical data will include safety data from the first two patients and efficacy data from the first patient treated with EDIT-301.
  • In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape.
  • EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease.



CAMBRIDGE, Mass., Dec. 01, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that it will host a live webinar on, Tuesday, December 6, at 8:00 a.m. ET to present initial clinical data from the Phase 1/2 RUBY trial of EDIT-301, under development for the treatment of severe sickle cell disease. The clinical data will include safety data from the first two patients and efficacy data from the first patient treated with EDIT-301.

The live and archived webcast of the presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website. The presentation will also be available for download shortly after the webinar.

A replay of the webinar and webinar materials will be available upon conclusion of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/.

About Sickle Cell Disease
Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.

About EDIT-301
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.

About RUBY
The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576).

About Editas Medicine
As a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard University’s Cas9 patent estates and Broad Institute’s Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.


Contacts:
Media
Cristi Barnett
(617) 401-0113
[email protected]

Investors
Ron Moldaver
(617) 401-9052
[email protected]

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