Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Beta Thalassemia
May 12 2022 - 4:30PM
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing
company, today announced that the U.S. Food and Drug Administration
(FDA) granted Orphan Drug Designation to EDIT-301, an
investigational, gene editing medicine, for the treatment of beta
thalassemia. The FDA previously granted Rare Pediatric Disease
designation to EDIT-301 for the treatment of beta thalassemia and
sickle cell disease.
“Beta thalassemia is a devastating disease that
leads to severe anemia, organ failure, and premature death.
Receiving Orphan Drug Designation for EDIT-301 for beta thalassemia
highlights the urgent need for new treatment options for patients,”
said James C. Mullen, Chairman, President, and Chief Executive
Officer, Editas Medicine. “Preparations to initiate the Phase 1/2
clinical trial of EDIT-301, a potentially transformative medicine
for people living with beta thalassemia, are underway, and we look
forward to dosing the first patient in the clinical trial this
year.”
The FDA's Orphan Drug Designation program provides
orphan status to drugs or biologics intended for the prevention,
diagnosis, or treatment of diseases that affect fewer than 200,000
people in the United States. Sponsors of medicines that are granted
Orphan Drug Designation are entitled to certain incentives,
including tax credits for qualified clinical trials, prescription
drug user-fee exemptions, and potential seven-year marketing
exclusivity upon FDA approval.
EDIT-301 is currently being investigated in a
clinical study in patients with severe sickle cell disease (RUBY
trial, NCT04853576). Editas expects to initiate a Phase 1/2 study
of EDIT-301 in patients with transfusion-dependent beta thalassemia
in 2022.
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.
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 sickle cell disease
and transfusion-dependent beta thalassemia.
About Editas
MedicineAs a leading 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. 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. Forward-looking
statements in this press release include statements regarding the
Company’s expectation to initiate a Phase 1/2 study of EDIT-301 in
patients with TDT in 2022 and dosing the first patient in the TDT
clinical trial this year. The Company may not actually achieve
the plans, intentions, or expectations disclosed in these
forward-looking statements, and you should not place undue reliance
on these forward-looking statements. Actual results or events
could differ materially from the plans, intentions and expectations
disclosed in these forward-looking statements as a result of
various factors, including: uncertainties inherent in the
initiation and completion of pre-clinical studies and clinical
trials and clinical development of the Company’s product
candidates; availability and timing of results from pre-clinical
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
represent the Company’s views only as of the date hereof
and should not be relied upon as representing its views as of any
subsequent date. Except as required by law, the Company
explicitly disclaims any obligation to update any forward-looking
statements.
Contacts:
Media
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
Investors
Ron Moldaver
(617) 401-9052
ir@editasmed.com
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