Editas Medicine Initiates Clinical Natural History Study to Evaluate Patients with Leber Congenital Amaurosis Type 10 (LCA10)...
September 12 2017 - 8:00AM
Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing
company, today announced that the Company initiated a clinical
natural history study of Leber Congenital Amaurosis type 10
(LCA10). LCA10 is caused by mutations in the CEP290 gene. The study
will prospectively evaluate patients to assess the course of the
disease and to pilot potential clinical trial endpoints and
designs. This knowledge will inform the interventional
clinical trial design for EDIT-101, Editas
Medicine’s pre-clinical product candidate to treat LCA10.
“We are very excited with the progress we are making in our
LCA10 program. The initiation of this natural history study brings
us one step closer to our goal of making a CRISPR-based medicine
available to people with significant vision loss caused by LCA10,”
said Gerry Cox, M.D., Ph.D., Chief Medical Officer, Editas
Medicine. “The data generated in this study will increase our
knowledge of the disease and its impact on vision-related
activities. It is a key step towards interventional clinical
trials, and we are pleased to be working with Massachusetts Eye and
Ear.”
Editas Medicine plans to enroll approximately 40 patients, ages
three and above, at multiple sites in the U.S. and Europe in this
study. The study will evaluate and follow patients for at least one
year. Massachusetts Eye and Ear, an international center for
treatment and research and a teaching hospital of Harvard Medical
School, is the first site enrolling patients for this study.
“We are delighted to be working at the forefront of this
research with Editas Medicine to better understand the course of
disease for patients with CEP290-associated retinal degeneration,
and to be working towards the interventional study,” said Eric A.
Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and
Solman and Libe Friedman Professor of Ophthalmology at
Massachusetts Eye and Ear and Harvard Medical School, and Principal
Investigator for the study.
Editas Medicine plans to submit an Investigational New Drug
(IND) application for EDIT-101 in mid-2018. In March, Editas
Medicine and Allergan Pharmaceuticals International Limited
(“Allergan”) entered into a strategic research and development
alliance under which Allergan received an exclusive option to
license up to five of Editas Medicine’s genome editing ocular
programs, including Editas Medicine’s lead program for LCA10. The
agreement covers a range of first-in-class ocular programs
targeting serious, vision-threatening diseases based on Editas
Medicine’s unparalleled CRISPR genome editing platform, including
CRISPR/Cas9 and CRISPR/Cpf1.
About Leber Congenital
AmaurosisLeber Congenital Amaurosis, or LCA, is a group of
inherited retinal degenerative disorders caused by mutations in at
least 18 different genes. It is the most common cause of inherited
childhood blindness, with an incidence of two to three per 100,000
live births worldwide. Symptoms of LCA appear within the first year
of life, resulting in significant vision loss and blindness. The
most common form of the disease, LCA10, is a monogenic disorder
caused by mutations in the CEP290 gene and is the cause of disease
in approximately 20‑30 percent of all LCA patients.
About Editas MedicineEditas Medicine is a
leading genome editing company dedicated to treating patients with
genetically-defined diseases by correcting their disease-causing
genes. The Company was founded by world leaders in genome editing,
and its mission is to translate the promise of genome editing
science into a broad class of transformative genomic medicines to
benefit the greatest number of patients.
Forward-Looking StatementsThis 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 goals for the Clinical Natural History
study and goal of submitting of an IND for the LCA10 program by
mid-2018. 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 preclinical studies and clinical
trials and clinical development of the Company’s product
candidates; 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 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 speak only as of the date hereof, and the Company
expressly disclaims any obligation to update any forward-looking
statements, whether as a result of new information, future events
or otherwise.
Contacts:MediaCristi
Barnett(617) 401-0113 cristi.barnett@editasmed.com
InvestorsMark Mullikin(617)
401-9083mark.mullikin@editasmed.com
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