Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company
committed to delivering a new class of differentiated one-time
curative genetic therapies, and Cimeio Therapeutics, a
biotechnology company that is reinventing cell therapy through its
leadership in the emerging field of epitope shielding, today
announced a research collaboration to combine their respective
technologies, including Prime Medicine’s Prime Editing platform and
Cimeio’s Shielded Cell and Immunotherapy Pairs (SCIP) platform.
The goal of the research is to improve the safety
and effectiveness of hematopoietic stem cell (HSC) transplants to
treat genetic diseases, acute myeloid leukemia (AML), and
myelodysplastic syndrome (MDS), as well as enable the in vivo
selection of edited HSCs to potentially remove the need for
transplantation entirely.
HSC transplant offers a potentially curative
approach for many debilitating and life-threatening diseases,
including malignancies such as AML and rare genetic diseases like
Gaucher’s and Hurler’s, though its clinical utility is limited by
the current need for myeloablative conditioning regimens and the
risk of post-transplant progression of malignant disease. Cimeio’s
SCIP platform combined with Prime Editing may significantly improve
the accessibility, eligibility and outcomes for patients who could
benefit from HSC transplant. These combined technologies may also
enable selection of in vivo edited HSCs, which could allow for the
treatment of genetic diseases without a transplant.
Under the terms of the agreement, Prime Medicine
will develop a Prime Editor for Cimeio’s CD117 shielding variant
that will then be evaluated by both companies. CD117 is a receptor
tyrosine kinase expressed on normal HSCs and on leukemia cells, and
therefore is an attractive target for an antibody-based
conditioning therapy for patients needing a stem cell transplant,
or for patients with AML or MDS needing new treatment options.
If the research collaboration is successful, the
companies will grant exclusive license options to each other for
their technology. Prime Medicine will receive an exclusive option
to license Cimeio’s cell shielding technology for CD117-shielded
HSC transplant, as well as in vivo editing of CD117-shielded HSCs,
for genetic diseases, not including Sickle cell disease. Cimeio
will receive an exclusive option to license Prime Medicine’s Prime
Editing technology for its CD117-shielded allogeneic HSC product
for use in AML and MDS, and, potentially, a second shielding
protein for use in AML and MDS. If the companies exercise their
exclusive license options, they will each be eligible to receive
economics on net sales of licensed products. Specific financial
terms were not disclosed.
Cimeio’s proprietary investigational
immunotherapies are designed to selectively deplete diseased HSCs,
while its novel CD117 protein variant, which can be introduced into
HSCs using Prime Editing, has the potential to protect the healthy
transplanted HSCs from depletion, allowing them to engraft while
maintaining the normal function of the CD117 receptor. Because the
edited HSCs are shielded, the diseased HSCs can be more gradually
depleted, which could reduce toxicity and increase safety. The
immunotherapies can also be administered post-transplant with the
potential to boost engraftment or treat minimal residual
disease.
Prime Editing will be uniquely suited to introduce
these novel protein variants into HSCs by virtue of its
versatility, precision and efficiency, without causing double
strand breaks and with minimal off target editing. Because Prime
Editing can be multiplexed (i.e., multiple Prime Edits can be made
with a single administration), rare genetic diseases such as beta
thalassemia, immunodeficiencies and bone marrow failure syndromes
may be corrected by Prime Editing at the same time as cell
shielding, offering the possibility of autologous transplant for
these patients without toxic conditioning.
“We believe Prime Editing is an incredibly powerful
technology that could impact the care and treatment of a wide range
of diseases. To fully exploit the potential of our technology, we
are committed to collaborating with partners who can meaningfully
expand our reach, accelerating our efforts to deliver important new
medicines to patients worldwide,” said Keith Gottesdiener, M.D.,
Chief Executive Officer of Prime Medicine. “Through this
partnership, we are gaining access to a promising platform
technology, which, when combined with Prime Editing, may allow many
more patients to benefit from the potentially curative power of HSC
transplant and, for the first time, make feasible in vivo treatment
of many genetic diseases. We are delighted to collaborate with the
Cimeio team, which includes experts in the fields of cell therapy,
gene editing and HSC transplant, and look forward to working
closely together to evaluate the synergistic potential of our
technologies.”
At the American Society of Hematology (ASH) Annual
Meeting in December 2022, Cimeio presented results from a
preclinical study demonstrating proof-of-concept for its
CD117-shielded cells. The data showed that its shielded cells
expressing a genetically engineered variant of CD117 were fully
functional in vitro and contributed to engraftment in vivo, similar
to unmodified HSCs expressing the wild-type receptor. Mice
transplanted with a mix of human HSCs expressing either wild-type
CD117 or the Cimeio shielded CD117 variant showed a selective
depletion of wild-type CD117 cells following treatment with
Cimeio’s antibody directed against wild-type CD117, while those
cells expressing the shielded CD117 variant were spared.
“Our recently disclosed data continues to show that
our unique shielding technology and paired immunotherapies have the
potential to deliver transformative therapies for patients with
many types of diseases,” said Thomas Fuchs, Chief Executive Officer
of Cimeio Therapeutics. “Through this collaboration, we are
bringing together industry leading protein engineering and genome
editing, with the potential to deliver safer, curative therapies
for patients. Our aim is to eliminate the need for toxic
chemotherapies and radiation, and enable new therapeutic approaches
post-transplant. Our goal is to reinvent HSC transplant as a more
effective and practical option for many more patients facing
debilitating and fatal diseases. We look forward to partnering with
the extremely talented team at Prime Medicine to advance our novel
CD117 program.”
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 leveraging its
proprietary Prime Editing platform, a versatile, precise and
efficient gene editing technology, to develop a new class of
differentiated, one-time, potentially 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.
Prime Medicine is currently progressing a
diversified portfolio of eighteen programs initially focused on
genetic diseases with a fast, direct path to treating patients or
with a high unmet need because they cannot be treated using other
gene-editing approaches. Over time, the Company intends to maximize
Prime Editing’s therapeutic potential and advance potentially
curative therapeutic options to patients for a broad spectrum of
diseases. For more information, please visit
www.primemedicine.com.
About Cimeio Therapeutics
Cimeio is an applied gene editing and immunotherapy
company developing a portfolio of Shielded-Cell & Immunotherapy
Pairs™ (SCIP), which has the potential to transform hematopoietic
stem cell transplant. Cimeio’s technology platform is based on the
design and expression of modified variants of naturally occurring
cell surface proteins in HSCs. These novel variants maintain their
function but are resistant to depletion when targeted by a paired
immunotherapy which has high affinity for the wild-type version of
these proteins. This technology has significant therapeutic
potential, which Cimeio is using to develop curative treatments for
patients with genetic diseases, hematologic malignancies, and
severe autoimmune disorders. Shielded Cell and Immunotherapy Pairs
and SCIP are trademarks of Cimeio Therapeutics AG. For more
information, please visit www.cimeio.com.
Cautionary Note Regarding 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 research collaboration to combine Prime
Medicine and Cimeio’s respective technologies, including Prime
Medicine’s Prime Editing platform and Cimeio’s SCIP platform, and
the goals of such collaboration; the potential benefits of such
collaboration and technology thereunder, including the ability to
cure various diseases and replace existing treatments such as
transplantation; and the exercise of the exclusive options and
payment of economics. 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: uncertainties related to the
authorization, initiation, and conduct of preclinical and other
development requirements for potential product candidates,
including uncertainties related to 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,
including the Cimeio preclinical study presented at ASH; the scope
of protection Prime Medicine is able to establish and maintain for
intellectual property rights covering its Prime Editing technology;
Prime Medicine’s ability to identify and enter into future license
agreements and collaborations; and 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 Quarterly Report on Form 10-Q, 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.
Prime Medicine Investor
ContactHannah DeresiewiczStern Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Prime Medicine Media ContactDan
Budwick, 1ABdan@1ABmedia.com
Cimeio ContactSteve
Edelsonsedelson@versantventures.com 415-801-8088
Grafico Azioni Prime Medicine (NASDAQ:PRME)
Storico
Da Apr 2024 a Mag 2024
Grafico Azioni Prime Medicine (NASDAQ:PRME)
Storico
Da Mag 2023 a Mag 2024