Editas Medicine to Host Virtual Event to Discuss EDIT-301 Clinical Data from the RUBY Trial for Severe Sickle Cell Disease and the EDITHAL Trial for Transfusion-dependent Beta Thalassemia
06 Junho 2023 - 9:00AM
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced that it will host a live webinar
on Monday, June 12, at 8:00 a.m. ET to present clinical data
from the RUBY trial of EDIT-301 for the treatment of severe sickle
cell disease. The Company will also present initial clinical data
from the EDITHAL trial of EDIT-301 for the treatment of
transfusion-dependent beta thalassemia.
The webinar follows the oral presentation of the RUBY clinical
trial update at the European Hematology Association (EHA) Hybrid
Congress in Frankfurt, Germany, and via live stream. The EHA oral
presentation is scheduled for Saturday, June 10, from 5:30-5:45
p.m. CEST/11:30-11:45 a.m. EDT.
Key data from four patients treated in the RUBY trial and one
patient treated in the EDITHAL trial will be shared in the
Company’s webinar, including:
- Initial safety and efficacy data from four patients treated in
the RUBY trial with 2-10 months follow-up, covering total
hemoglobin, fetal hemoglobin, and vaso-occlusive events (VOEs)
post-EDIT-301 infusion.
- Initial safety data from one patient treated in the EDITHAL
trial with 1.5 months follow-up, including neutrophil and platelet
engraftment, and initial efficacy data covering total hemoglobin
and fetal hemoglobin post-EDIT-301 infusion.
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/.
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 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 (RBCs) 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 clinical manifestation of RBCs sickling.
About Beta Thalassemia Beta thalassemia is a
common autosomal recessive disorder with an estimated annual
incidence rate of 1 in 100,000 worldwide for symptomatic
individuals. Beta thalassemia mutations reduce or abrogate beta
globin expression. Insufficient beta globin production leads to
ineffective red blood cell production, chronic hemolytic anemia due
to the destruction of red blood cells, and compensatory
extramedullary hematopoiesis (creation of blood cells). Based on
clinical severity and transfusion requirements, beta thalassemia
can be classified into non-transfusion-dependent (NTDT) and
transfusion-dependent beta thalassemia (TDT). TDT is the most
severe form of beta thalassemia, and patients require lifelong
regular red blood cell transfusions to prevent organ failure and
death. Chronic red blood cell transfusions are complicated by iron
overload leading to organ dysfunction and failure. Left untreated,
the mortality rate among TDT patients is high, with a survival rate
of only 15 percent at age five due to severe anemia. It is
estimated that there are approximately 1,000 people in the United
States currently living with transfusion-dependent beta
thalassemia. Higher levels of fetal hemoglobin (HbF) ameliorate
anemia thereby reducing the need for regular red blood cell
transfusions.
About EDIT-301EDIT-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 RUBYThe 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. Patients will receive a single administration of EDIT-301.
Additional details are available
on www.clinicaltrials.gov (NCT# 04853576).
About EDITHALThe 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
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.
Media and Investor Contact:
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
ir@editasmed.com
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