Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced that Gilmore O’Neill, M.B.,
M.M.Sc., President and Chief Executive Officer, will discuss the
Company’s 2024 strategic priorities and anticipated milestones at
the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday,
January 9, at 4:30 p.m. PST.
In his remarks, Dr. O’Neill will discuss several components of
the Company’s strategic priorities and progress, including an
unchanged focus on developing renizgamglogene autogedtemcel
(reni-cel) towards biologics licensing application (BLA) and
commercialization, building an in vivo pipeline, and increasing
business development activities, including continuing to sublicense
the Company’s foundational Cas9 and Cas12a gene editing
technology.
Dr. O’Neill will also discuss the Company’s anticipated 2024
milestones:
- Continue enrollment and dosing in the RUBY and EdiTHAL clinical
trials of reni-cel,
- Initiate the adolescent cohort in the RUBY trial,
- Present a substantive clinical data set of sickle cell patients
with considerable clinical follow-up in the RUBY trial in mid-2024
and by year-end 2024,
- Establish in vivo preclinical proof-of-concept for an
undisclosed indication, and
- Derive revenue from the Company’s foundational IP, building on
the recently announced license agreements with Vertex
Pharmaceuticals and Vor Bio.
“2023 was a pivotal year for Editas as we launched and executed
our focused strategy, strengthened our leadership team, and hit
multiple clinical milestones to drive Editas’ transformation
towards a commercial-stage company. We expect 2024 to be even more
eventful as we continue to develop our potentially transformative
experimental medicines,” said Dr. O’Neill.
He continued, “As a pioneer in the genome editing field, we
remain focused on driving solutions for people living with serious,
previously untreatable diseases by leveraging our world-class gene
editing platform – in the form of developing our own clinically
differentiated medicines and in the form of licenses and
sublicenses to other pharmaceutical and biotechnology companies
developing medicines. To be a part of this chapter of Editas’
journey is invigorating, and I look forward to what’s next.”
J.P. Morgan Healthcare Conference WebcastDr.
O’Neill will discuss the Company’s strategic priorities and 2024
anticipated key milestones for its gene editing medicines and
platform technology at the 42nd Annual J.P. Morgan Healthcare
Conference on Tuesday, January 9, 2024, at 4:30 p.m. PT / 7:30 p.m.
ET in San Francisco, CA. A live webcast of the presentation will be
available on the “Investors” section of the Editas Medicine website
at www.editasmedicine.com. An archived replay will be available on
the website for approximately 30 days following the
presentation.
About renizgamglogene autogedtemcel
(reni-cel)Reni-cel, formerly known as EDIT-301, is an
experimental gene editing medicine under investigation for the
treatment of severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT). Reni-cel 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 AsCas12a, a novel, proprietary, highly efficient, and
specific gene editing nuclease. Red blood cells derived from
reni-cel 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 reni-cel in patients with severe
sickle cell disease. Enrolled patients will receive a single
administration of reni-cel. The RUBY trial marks the first time
AsCas12a was used to successfully edit human cells in a clinical
trial. Additional details are available
on www.clinicaltrials.gov (NCT04853576).
About the EdiTHAL TrialThe EdiTHAL trial is a
single-arm, open label, multi-center Phase 1/2 study designed to
assess the safety and efficacy of reni-cel in patients with
transfusion-dependent beta thalassemia. Patients will receive a
single administration of reni-cel. Additional details are available
on www.clinicaltrials.gov (NCT05444894).
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
initiation, timing, progress and results of the Company’s
preclinical and clinical studies and its research and development
programs, including initiating the adolescent cohort in the RUBY
trial in 2024 and establishing in vivo proof of concept for an
undisclosed indication in 2024, the timing for the Company’s
receipt and presentation of data from its clinical trials and
preclinical studies, including RUBY clinical updates in mid-2024
and by year-end 2024, the potential of, and expectations for, the
Company’s product candidates, the timing or likelihood of
regulatory filings and approvals, the Company’s expectations
regarding commercial readiness, and the Company’s expectations
regarding cash runway. 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 and EdiTHAL trials, and clinical
development of the Company’s product candidates, including
reni-cel; 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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