Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EDIT-301 for the Treatment of Severe Sickle Cell Disease
16 Octubre 2023 - 7:00AM
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced that the U.S. Food and Drug
Administration (FDA) granted Regenerative Medicine Advanced Therapy
(RMAT) designation to EDIT-301, an investigational, gene editing
medicine, for the treatment of severe sickle cell disease (SCD).
“Sickle cell disease is a devastating disease that leads to
anemia, pain crises, organ failure, and early death. Receiving RMAT
designation for EDIT-301 for severe sickle cell disease highlights
the urgent need for new treatment options for patients and supports
our belief that EDIT-301 can provide life-changing clinical
benefits to patients,” Gilmore O’Neill, M.B., M.M.Sc., President
and Chief Executive Officer, Editas Medicine. “I would like to
thank the participants, their families, clinicians, and colleagues
at collaborating institutions that contribute to the RUBY trial. We
look forward to sharing further clinical updates including
additional data for the trial prior to year-end.”
Established under the 21st Century Cures Act, RMAT designation
is a dedicated program designed to expedite the development and
review processes for promising regenerative medicine therapies. An
investigational cell therapy medicine or gene editing medicine is
eligible for RMAT designation if it is intended to treat, modify,
reverse, or cure a serious or life-threatening disease or
condition, and preliminary clinical evidence indicates that the
experimental medicine has the potential to address unmet medical
needs for the disease or condition. Advantages of the RMAT
designation include all the benefits of the fast track and
breakthrough therapy designation programs, including but not
limited to intensive FDA guidance on efficient and expedited drug
development, possible rolling review, and priority review of the
biologics license application (BLA), and FDA’s organizational
commitment involving senior managers.
The FDA previously granted Orphan Drug Designation and Rare
Pediatric Disease designation to EDIT-301 for the treatment of
sickle cell disease and beta thalassemia.
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 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. Forward-looking
statements in this press release include statements regarding the
Company’s expectation to provide further clinical data updates,
including additional data for the RUBY trial by year-end. 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 important
factors, including: uncertainties inherent in the initiation and
completion of preclinical studies and clinical trials, including
the RUBY trial, 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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