Editas Medicine Reports on Recent Progress at J.P. Morgan Healthcare Conference
January 07 2019 - 9:00AM
In a presentation to investors on Wednesday, January 9,
2019, at 9:00 a.m. PST at the 37th Annual J.P.
Morgan Healthcare Conference, Editas Medicine,
Inc. (Nasdaq: EDIT) President and CEO Katrine
Bosley will discuss the Company’s plans to initiate patient
screening and patient dosing for EDIT-101. Additionally, she will
outline progress in the Company’s program for the treatment of
sickle cell disease, including data that support opportunities to
develop best-in-class, durable medicines for hemoglobinopathies.
Ms. Bosley will also detail the Company’s progress on “EM22,” the
Company’s long-range goals through the year 2022 and vision for
advancing Editas Medicine as a leading genome editing company,
including sharing details on advancements in its ocular and
engineered cell medicine programs.
EDIT-101 is an experimental CRISPR genome editing medicine being
investigated for the treatment of Leber congenital amaurosis 10
(LCA10). It is set to be the first in vivo, or editing inside the
body, CRISPR-based medicine administered to people anywhere in the
world. In the Phase 1/2 clinical trial, Editas Medicine and
Allergan plan to initiate patient screening mid-year and begin
patient dosing in the second half of 2019, enrolling 10-20 patients
in the U.S. and Europe.
“At Editas Medicine, we are pioneering the possible by
harnessing the power of genome editing, engineered cell therapy and
AAV gene delivery to develop a pipeline of genomic medicines for
people living with serious diseases,” said Bosley. “With our recent
successes, including the FDA’s acceptance of our IND for EDIT-101,
we are entering 2019 with strong momentum towards achieving our
EM22 goals. We look forward to entering the clinic later this year,
and we hope to transform the lives of people living with
LCA10.”
Ms. Bosley will also provide an update on the Company’s progress
on EM22. By year-end 2022, Editas Medicine is driving to deliver
medicines for people with serious diseases around the world by
advancing at least three experimental medicines in early-stage
clinical trials, at least two experimental medicines in or ready
for late-stage clinical trials, a best-in-class platform and
pipeline for developing genomic medicines, and building the company
for the long term with a unique, “Inspiritas” culture. Recent
achievements include:
Continued Commitment to Ocular Disorders
- The LCA10 program is on track to be the first in vivo
CRISPR-based genome editing medicine with patient dosing expected
in the second half of 2019.
- The Company now has ocular programs in early research to treat
Usher syndrome 2A (USH2A) and retinitis pigmentosa.
Important progress in engineered cell medicines
- The Company made recent advances toward a durable medicine for
sickle cell and beta-thalassemia. Editing at the HBG1/2 site is a
differentiated approach for development of a human therapeutic for
the treatment of sickle cell disease and beta-thalassemia as
compared to other medicines currently under development that edit
at the BCL11A erythroid enhancer (BCL11Ae) site. Notably, editing
HBG1/2 promoters upregulated fetal hemoglobin with superior
repopulation of red blood cell precursors as compared to editing
the BCL11Ae site. The red blood cell precursors from bone marrow
edited at the BCL11Ae site had lower productive editing rates
compared to other lineages and showed increased level of apoptosis,
or programmed cell death, in erythroid culture compared to HBG1/2
promoter-edited cells.
- In the Company’s collaboration with Juno Therapeutics, Inc., a
Celgene company, CRISPR-edited product candidates are advancing in
both solid and liquid tumors.
Advancing Organizational Excellence
- The Company added key talent across hematology, oncology,
ophthalmology, manufacturing, and ex vivo (editing outside the
human body) research in 2018. This expertise is critical to the
continued advancement of Editas Medicine’s pipeline and
platform.
About EDIT-101EDIT-101 is a CRISPR-based
experimental medicine under investigation for the treatment of
Leber congenital amaurosis 10 (LCA10). EDIT-101 is
administered via a subretinal injection to reach and deliver the
gene editing machinery directly to photoreceptor cells.
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
years of life, resulting in significant vision loss and potentially
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 The Editas Medicine-Allergan AllianceIn
March 2017, Editas Medicine and Allergan Pharmaceuticals
International Limited (Allergan) entered a strategic alliance and
option agreement under which Allergan received exclusive access and
the option to license up to five of Editas Medicine’s genome
editing programs for ocular diseases, including
EDIT-101. Under the terms of the agreement, Allergan is
responsible for development and commercialization of optioned
products, subject to Editas Medicine’s option to co-develop and
share equally in the profits and losses of two optioned products in
the United States. In August 2018, Allergan exercised its
option to develop and commercialize EDIT-101 globally for the
treatment of LCA10. Additionally, Editas Medicine exercised its
option to co-develop and share equally in the profits and losses
from EDIT-101 in the United States. Editas Medicine is also
eligible to receive development and commercial milestones, as well
as royalty payments on a per-program basis. 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 (also known as Cas12a).
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
clinical trial timeline of EDIT-101 and the Company’s EM22
goals. 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 because 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
Editas Medicine (NASDAQ:EDIT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Editas Medicine (NASDAQ:EDIT)
Historical Stock Chart
From Sep 2023 to Sep 2024