Editas Medicine Receives Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Sickle Cell Disease
August 24 2020 - 8:30AM
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing
company, today announced that the U.S. Food and Drug Administration
(FDA) has granted Rare Pediatric Disease (RPD) designation for
EDIT-301, an experimental, autologous cell medicine, being
developed as a potentially best-in-class, durable medicine for
sickle cell disease. The Company plans to file an investigational
new drug application (IND) for EDIT-301 by the end of 2020.
“The Editas team has a bold vision to unlock the potential of
CRISPR to design and develop game-changing medicines. We are making
tremendous progress towards this vision with the continued
development of EDIT-301, a potentially transformative medicine for
the treatment of sickle cell disease, and we are pleased to receive
Rare Pediatric Disease designation from the FDA for this program,”
said Cynthia Collins, Chief Executive Officer, Editas Medicine.
“We know patients are counting on us, and this designation is
a significant milestone for the program that highlights the
serious, life-threatening manifestations of sickle cell
disease.”
The FDA defines a rare pediatric disease as a serious or
life-threating disease in which the serious or life-threatening
disease manifestations primarily affect individuals aged from birth
to 18 years. Pediatric diseases recognized as “rare” affect under
200,000 people in the United States. Under the FDA’s Rare Pediatric
Disease Designation and Voucher Programs, a sponsor who receives an
approval for a drug or biologic for a "rare pediatric disease" may
be eligible for a voucher that can be redeemed to receive priority
review of a subsequent marketing application for a different
product.
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 the disc shape. The abnormal shape
causes the cells 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. Fetal hemoglobin (HbF) protects against sickle cell
disease by inhibiting HbS polymerization.
About EDIT-301EDIT-301 is an experimental,
autologous cell therapy medicine under investigation for the
treatment of sickle cell disease. EDIT-301 is comprised of sickle
patient CD34+ cells genetically modified using a highly specific
and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein
(RNP) to edit the HBG1/2 promoter region in the beta-globin locus.
Red blood cells derived from EDIT-301 CD34+ cells demonstrate a
sustained increase in fetal hemoglobin (HbF) production, which has
the potential to provide a durable treatment benefit for people
living with sickle cell disease.
About Editas MedicineAs a
leading genome editing company, Editas Medicine is focused on
translating the power and potential of the CRISPR/Cas9 and
CRISPR/Cpf1 (also known as 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 plans with respect to timing of filing an IND for
EDIT-301 by the end of 2020. 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 Quarterly Report on Form 10-Q, which is on file 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:MediaCristi
Barnett(617) 401-0113 cristi.barnett@editasmed.com
InvestorsMark Mullikin(617)
401-9083mark.mullikin@editasmed.com
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