Prime Medicine to Highlight New Preclinical Data, Including In Vivo Data in Wilson’s Disease, at Upcoming Scientific Meetings
15 Oktober 2024 - 2:00PM
Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company
committed to delivering a new class of differentiated one-time
curative genetic therapies, today announced the Company will
highlight advances from across its Prime Editing pipeline and
platform at the upcoming European Society of Gene and Cell Therapy
(ESGCT) 31st Annual Congress, being held October 22-25, 2024 in
Rome, and the American Association for the Study of Liver Diseases
(AASLD), being held November 15-19, 2024 in San Diego.
“At the ESGCT and AASLD meetings, we will present
new preclinical data from across our pipeline, including from our
efforts in liver disease,” said Jeremy Duffield, M.D., Ph.D., Chief
Scientific Officer of Prime Medicine. “We are particularly excited
to share in vivo data from our Wilson’s Disease program, as well as
from our universal liver-directed lipid nanoparticle (LNP)
platform. Together, these data reinforce our strategy of
prioritizing Wilson’s Disease – a large, genetically defined
condition for which there are no currently approved
disease-modifying treatments, and which we believe is uniquely
suited to a Prime Editing-based approach – and reinforce the
potential for our modular universal LNP to be used broadly across
current and future programs. We look forward to advancing our
Wilson’s Disease program into IND-enabling studies later this year
and, longer term, to potentially leveraging the modularity of our
platform to expand into numerous follow-on indications.”
Details of the presentations are as follows:
European Society of Gene and Cell Therapy
31st Annual Congress (ESGCT);
October 22-25, 2024 in Rome
- Presentation Title: LNP delivered Prime
Editors restore glycemic control in humanized rodent models of
Glycogen Storage Disease Type 1b (GSD1b)Date &
Time: October 24, 2024, 2:00 p.m. CEST
- Poster Title: Development of PM359, a Prime
Edited CD34+ cell drug product for the treatment of p47phox Chronic
Granulomatous DiseaseDate & Time: October 24,
2024, 2:00 – 2:30 p.m. CEST
- Poster Title: An all-Prime Editing one-step
approach for non-viral generation of a multiplex-edited allogeneic
CAR-T cell productDate & Time: October 24,
2024, 2:00 – 2:30 p.m. CEST
- Poster Title: Methods for genome-wide
detection of single strand breaks induced by gene editors reveals
the specificity of SpCas9 nuclease domains and provides
comprehensive lists of potential off-targets for Prime
EditorsDate & Time: October 24, 2024, 2:00 –
2:30 p.m. CEST
- Poster Title: Exploring the roles of DNA
repair processes across diverse Prime Editing strategies with
pooled screens and Knock-Knock PrimeDate &
Time: October 24, 2024, 6:00 – 7:30 p.m. CEST
- Poster Title: Prime Editing advancements
enable in vivo therapeutic correction of ATP7B p.H1069Q and p.R778L
mutations in Wilson’s DiseaseDate & Time:
October 24, 2024, 6:00 – 7:30 p.m. CEST
- Poster Title: Prime Editors precisely correct
pathogenic mutations in RHO and USH2A associated Retinitis
Pigmentosa and prevent retinal degenerationDate &
Time: October 24, 2024, 6:00 – 7:30 p.m. CEST
American Association for the Study of Liver
Diseases (AASLD); November 15-19, 2024 in San Diego
- Presentation Title: LNP delivered Prime
Editors restore glycemic control in humanized rodent models of
Glycogen Storage Disease Type 1b (GSD1b)Date &
Time: Sunday, November 17, 2024, 5:00 – 6:30 p.m. PT
- Presentation Title: Advances in Prime Editing
enable in vivo therapeutic correction of the ATP7B p.H1069Q and
p.R778L mutations causing Wilson’s DiseaseDate &
Time: Monday, November 18, 2024, 8:00 – 9:30 a.m. PT
About Prime Medicine
Prime Medicine is a leading biotechnology company
dedicated to creating and delivering the next generation of gene
editing therapies to patients. The Company is deploying its
proprietary Prime Editing platform, a versatile, precise and
efficient gene editing technology, to develop a new class of
differentiated one-time curative genetic therapies. Designed to
make only the right edit at the right position within a gene while
minimizing unwanted DNA modifications, Prime Editors have the
potential to repair almost all types of genetic mutations and work
in many different tissues, organs and cell types. Taken together,
Prime Editing’s versatile gene editing capabilities could unlock
opportunities across thousands of potential indications.
Prime Medicine is currently progressing a
diversified portfolio of investigational therapeutic programs
organized around our core areas of focus: hematology, immunology
and oncology, liver and lung. Across each core area, Prime Medicine
is focused initially on a set of high value programs, each
targeting a disease with well-understood biology and a clearly
defined clinical development and regulatory path, and each expected
to provide the foundation for expansion into additional
opportunities. Over time, the Company intends to maximize Prime
Editing’s broad and versatile therapeutic potential, as well as the
modularity of the Prime Editing platform, to rapidly and
efficiently expand beyond the diseases in its current pipeline,
potentially including additional genetic diseases, immunological
diseases, cancers, infectious diseases, and targeting genetic risk
factors in common diseases, which collectively impact millions of
people. For more information, please visit
www.primemedicine.com.
© 2024 Prime Medicine, Inc. All rights reserved.
PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are
trademarks of Prime Medicine, Inc. All other trademarks referred to
herein are the property of their respective owners.
Forward Looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, implied and express
statements about Prime Medicine’s beliefs and expectations
regarding: the timing, progress, and results of its Wilson’s
Disease program, including the timing of IND-enabling activities
and opening an IND and/or CTA application; the initiation, timing,
progress, and results of its research and development programs,
preclinical studies and future clinical trials; the modularity of
the Prime Editing platform and the benefits thereof; the potential
for its modular universal LNP to be used broadly across current and
future programs; the potential for Prime Editors to more precisely
and effectively achieve genetic modification; the potential for
Prime Editors to repair genetic mutations and offer curative
genetic therapies for a wide spectrum of diseases; the potential of
Prime Editors to reproducibly correct disease-causing genetic
mutations across different tissues, organs and cell types; its
expectations regarding the breadth of Prime Editing technology and
the implementation of its strategic plans for its business,
programs, and technology; and the potential of Prime Editing to
unlock opportunities across thousands of potential indications. The
words “may,” “might,” “will,” “could,” “would,” “should,” “expect,”
“plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,”
“seek,” “predict,” “future,” “project,” “potential,” “continue,”
“target” and similar words or expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words.
Any forward-looking statements in this press
release are based on management’s current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, risks associated with: the authorization, initiation,
and conduct of preclinical and IND-enabling studies and other
development requirements for potential product candidates,
including uncertainties related to opening INDs and obtaining
regulatory approvals; risks related to the development and
optimization of new technologies, the results of preclinical
studies, or clinical studies not being predictive of future results
in connection with future studies; the scope of protection Prime
Medicine is able to establish and maintain for intellectual
property rights covering its Prime Editing technology; and the
effect of unfavorable macroeconomic conditions or market volatility
resulting from general economic, industry and market conditions,
including rising interest rates, inflation, and adverse
developments affecting the financial services industry. These and
other risks and uncertainties are described in greater detail in
the section entitled “Risk Factors” in Prime Medicine’s most recent
Annual Report on Form 10-K and Quarterly Report on Form 10-Q for
the quarter ended June 30, 2024, as well as any subsequent filings
with the Securities and Exchange Commission. In addition, any
forward-looking statements represent Prime Medicine’s views only as
of today and should not be relied upon as representing its views as
of any subsequent date. Prime Medicine explicitly disclaims any
obligation to update any forward-looking statements subject to any
obligations under applicable law. No representations or warranties
(expressed or implied) are made about the accuracy of any such
forward-looking statements.
Investor ContactHannah
DeresiewiczPrecision
AQ212-362-1200hannah.deresiewicz@precisionaq.com
Media ContactDan Budwick,
1ABdan@1ABmedia.com
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