Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced that a scientific abstract
detailing safety and efficacy clinical data from the RUBY trial of
EDIT-301 in patients with severe sickle cell disease and from the
EdiTHAL trial of EDIT-301 in patients with transfusion-dependent
beta thalassemia has been accepted for a poster presentation at the
65th American Society of Hematology (ASH) Annual Meeting and
Exposition being held December 9-12, 2023, in San Diego, CA, and
online.
Editas Medicine will also host a Company-sponsored webinar on
Monday, December 11, at 1:00 p.m. ET to discuss the RUBY and
EdiTHAL clinical data.
Key data from patients treated in the RUBY trial and in the
EdiTHAL trial will be shared, including:
- RUBY
- Clinical data on 11 patients, including two patients with at
least 12 months follow-up and an additional four patients with at
least five months follow-up.
- Efficacy data, including total hemoglobin, fetal hemoglobin,
and vaso-occlusive events, or VOEs.
- Safety data, including neutrophil and platelet
engraftment.
- EdiTHAL
- Clinical data on six patients, including at least five months
data from the first two patients treated.
- Efficacy data, including total hemoglobin and fetal
hemoglobin.
- Safety data, including neutrophil and platelet
engraftment.
“We are making significant progress with EDIT-301, and we look
forward to sharing clinical data, including additional data
collected since the submission of the abstract, from the RUBY and
EdiTHAL trials next month at the American Society of Hematology
Annual Meeting and in a Company-sponsored webinar. This data
further supports our belief that EDIT-301 has the potential to be a
clinically differentiated, one-time, durable medicine that can
provide life-changing clinical benefits to patients,” Baisong Mei,
M.D., Ph.D., Senior Vice President and Chief Medical Officer,
Editas Medicine. “I would like to thank the participants, their
families and caregivers, clinicians, and colleagues at
collaborating institutions that contribute to the RUBY and EdiTHAL
trials.”
Webinar Presentation Details: The live and
archived webcast of the Company’s webinar presentation will be
accessible through this webcast link, or through the Events &
Presentations page of the “Investors” section of the Company’s
website.
A replay of the webinar will be available upon conclusion of the
webinar in the Investors section of the Editas Medicine website
at https://www.editasmedicine.com/.
ASH Presentation Details:
Title: AsCas12a Gene Editing of HBG1/2 Promoters
with EDIT-301 Results in Rapid and Sustained Normalization of
Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe
Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia
Presenting Author: Rabi Hanna, M.D., Department of
Pediatric Hematology Oncology and Blood and Marrow Transplantation,
Cleveland Clinic Children’s, Cleveland, OH, United
StatesDate/Time: Monday, December 11, 2023, 6:00 –
8:00 p.m. PT/9:00 – 11:00 p.m. ETLocation: San
Diego Convention Center, Halls G-HSession: 801.
Gene Therapies: Poster IIIPublication Number:
4996
The abstract can be accessed on the ASH website.
EDIT-301 is currently being investigated in a clinical study in
patients with severe sickle cell disease (RUBY trial, NCT04853576)
and transfusion-dependent beta thalassemia (EDITHAL trial,
NCT05444894).
About Sickle Cell DiseaseSickle cell disease is
an inherited blood disorder caused by a mutation in the beta-globin
gene that leads to polymerization of the sickle hemoglobin (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-301EDIT-301 is an experimental gene
editing 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 the RUBY TrialThe 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 the EdiTHAL TrialThe EdiTHAL 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
transfusion-dependent beta thalassemia. Patients will receive a
single administration of EDIT-301. Additional details are available
on www.clinicaltrials.gov (NCT# 05444894).
About Editas MedicineAs a
clinical-stage genome editing company, Editas Medicine is focused
on translating the power and potential of the CRISPR/Cas12a and
CRISPR/Cas9 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’s Cas12a patent estate and Broad
Institute and Harvard University’s Cas9 patent estates for human
medicines. For the latest information and scientific presentations,
please visit www.editasmedicine.com.
Forward-Looking Statements This press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995.
The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’
‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’
‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’
‘‘would,’’ and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Actual results or
events could differ materially from the plans, intentions and
expectations disclosed in these forward-looking statements as a
result of various important factors, including: uncertainties
inherent in the initiation and completion of preclinical studies
and clinical trials, including the RUBY and EdiTHAL trials, and
clinical development of the Company’s product candidates, including
EDIT-301; availability and timing of results from preclinical
studies and clinical trials; whether interim results from a
clinical trial will be predictive of the final results of the trial
or the results of future trials; expectations for regulatory
approvals to conduct trials or to market products and availability
of funding sufficient for the Company’s foreseeable and
unforeseeable operating expenses and capital expenditure
requirements. These and other risks are described in greater
detail under the caption “Risk Factors” included in the Company’s
most recent Annual Report on Form 10-K, which is on file with the
Securities and Exchange Commission, as updated by the
Company’s subsequent filings with the Securities and Exchange
Commission, and in other filings that the Company may make with the
Securities and Exchange Commission in the future. Any
forward-looking statements contained in this press release speak
only as of the date hereof, and the Company expressly disclaims any
obligation to update any forward-looking statements, whether
because of new information, future events or otherwise.
Media and Investor Contact:
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
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