Editas Medicine Demonstrates Dose-Dependent, In Vivo Editing with EDIT-101 in CEP290 Transgenic Mice
October 19 2017 - 12:45PM
Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing
company, today announced results from a pre-clinical study in
transgenic mice demonstrating dose-dependent, in vivo editing using
EDIT-101, Editas Medicine’s pre-clinical product
candidate for the treatment of Leber Congenital Amaurosis type 10
(LCA10). The study was conducted in mice that have a human CEP290
common intron 26 knock-in (HuCEP290 IVS26 KI mice), an animal model
for most common genetic change that causes LCA10. The results of
this study further reinforce Editas Medicine’s belief in the
transformative potential of EDIT-101 as a genome editing medicine
to help patients with LCA10. LCA10 is an inherited retinal
degenerative disease caused by mutations in the CEP290 gene that
appears in childhood and leads to blindness. The Company reported
these data today in a poster presentation at the 25th Anniversary
Congress of the European Society of Gene and Cell Therapy (ESGCT)
in Berlin.
In this study, HuCEP290 IVS26 KI mice were treated with EDIT-101
by subretinal injection, resulting in efficient transduction and
gene editing in the retinal photoreceptor cells of the mice, which
is the cell type affected in LCA10 patients. The onset of
CEP290 gene editing was rapid and was detectable as early as three
days post-delivery with a further significant increase in editing
observed by one week. The components were measured, and both Cas9
mRNA and guide RNA (gRNA) levels correlated with editing. At the
administered dose of 1E+12 vg/mL vector concentration, editing
levels exceeded levels predicted to be therapeutically relevant in
90 percent (27/30) of EDIT-101 treated eyes with a median of 31
percent of photoreceptors harboring productively edited CEP290 in
the transduced neuroretina. The productive CEP290 gene editing
rates were stable through six months of observation, and the
expression of Cas9 mRNA and gRNA decreased over time.
“To date, we have made significant progress in our LCA10
program. We have achieved predictive therapeutic levels of
productive CEP290 gene editing in several pre-clinical settings,
including in neural retinas in human cells, in transgenic mice, and
in non-human primates. Collectively, these data demonstrate that we
can deliver EDIT-101 to photoreceptors and can edit in the eye at
levels well above the anticipated minimum therapeutic threshold,
supporting the clinical development of EDIT-101 for the treatment
of patients suffering from LCA10,” said Charles Albright, Ph.D.,
Chief Scientific Officer, Editas Medicine.
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 (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. To learn more about Editas
Medicine, please visit www.editasmedicine.com.
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 goal of submitting of an IND for the LCA10 program by
mid-2018 and translating the Company’s CRISPR technology into
medicines to help patients suffering from LCA10. 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.
ContactsMedia:Cristi
Barnett(617) 401-0113cristi.barnett@editasmed.com
Investors:Mark Mullikin(617)
401-9083mark.mullikin@editasmed.com
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