Regeneron Pharmaceuticals, Inc. (NASDAQ:
REGN) and
Mammoth Biosciences, Inc., today announced a collaboration to
research, develop and commercialize in vivo CRISPR-based gene
editing therapies for multiple tissues and cell types. Regeneron is
developing adeno-associated viral vectors (AAVs) using
antibody-based targeting to enhance delivery of genetic medicine
payloads to specific tissues and cell types. Mammoth is developing
novel ultracompact nucleases and associated gene editing systems,
with a variety of editing functionalities at a significantly
smaller size than other CRISPR-based systems, including first
generation Cas9 nucleases. By leveraging Regeneron’s expertise in
AAV and antibody engineering and Mammoth’s expertise in
ultracompact gene editing systems, the teams will endeavor to
create disease-modifying medicines that can be delivered to tissues
beyond the liver, to which most gene editing treatments are
currently limited.
“We believe in the incredible power of gene editing, which we
are utilizing in our diverse preclinical and clinical genetic
medicines pipeline. After years spent developing our
next-generation delivery approaches, we are eager to combine them
with Mammoth’s gene editing systems to better match payload,
delivery system and disease type,” said Christos Kyratsous, Ph.D.,
Senior Vice President and co-Head of Regeneron Genetic Medicines at
Regeneron. “Together, we have the potential to overcome significant
delivery hurdles and effectively reach tissues around the body,
impact multiple diseases and dramatically increase the number of
patients who could benefit from gene editing treatments.”
“Mammoth brings over a decade of scientific expertise in CRISPR,
beginning with our co-founders’ work in the Doudna Lab at
University of California Berkeley. We believe we can further our
mission to transform the lives of patients by accelerating the
discovery and development of genetic medicines in collaboration
with Regeneron. Mammoth’s ultracompact CRISPR systems address the
size constraints of viral delivery and complement Regeneron’s
targeted AAV technologies. We look forward to working with
Regeneron to enable all-in-one AAV delivery and unlock the true
potential of in vivo gene editing,” said Trevor Martin, Ph.D.,
co-founder and Chief Executive Officer of Mammoth Biosciences.
“This exciting collaboration pairs two teams with a shared
commitment to translating high science into ground-breaking in vivo
genetic medicines that can potentially address the needs of more
patients and more prevalent conditions,” said Jennifer Doudna,
Ph.D., co-founder and Chair of the Scientific Advisory Board at
Mammoth Biosciences, CRISPR genome editing co-inventor and winner
of the 2020 Nobel Prize in Chemistry.
Under the terms of the agreement, Mammoth will receive $100
million inclusive of $95 million in equity investment at signing,
and an upfront payment, and is eligible to receive up to $370
million per target in development, regulatory and commercial
milestone payments, and royalty rates ranging from single digits to
mid-teens on future net sales of all collaboration products. In
addition, Mammoth has the right to opt-in to co-funding and sharing
profits on a majority of collaboration programs in lieu of
receiving milestones and royalties. In exchange, for a period of
five and a half years, Regeneron is obtaining broad access to
Mammoth’s editing technologies, other than certain excluded
targets, with the option to extend such access for an additional
two years upon the payment of a research extension fee. The parties
will jointly select and research collaboration targets, and then
Regeneron will lead development and commercialization.
About Regeneron
Regeneron (NASDAQ:
REGN) is a leading biotechnology company that invents, develops and
commercializes life-transforming medicines for people with serious
diseases. Founded and led by physician-scientists, our unique
ability to repeatedly and consistently translate science into
medicine has led to numerous approved treatments and product
candidates in development, most of which were homegrown in our
laboratories. Our medicines and pipeline are designed to help
patients with eye diseases, allergic and inflammatory diseases,
cancer, cardiovascular and metabolic diseases, neurological
diseases, hematologic conditions, infectious diseases and rare
diseases.
Regeneron pushes the boundaries of scientific discovery
and accelerates drug development using our proprietary
technologies, including VelociSuite® which produces optimized fully
human antibodies and new classes of bispecific antibodies. We are
shaping the next frontier of medicine with data-powered insights
from the Regeneron Genetics Center® and pioneering genetic medicine
platforms, enabling us to identify innovative targets and
complementary approaches to potentially treat or cure diseases.
For more information, please visit www.Regeneron.com or follow
Regeneron on LinkedIn, Instagram, Facebook or X.
About Mammoth BiosciencesMammoth Biosciences is
a biotechnology company focused on leveraging its proprietary
ultracompact CRISPR systems to develop potential long-term curative
therapies for patients with life-threatening and debilitating
diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer
Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the
company’s ultracompact systems are designed to be more specific and
enable in vivo gene editing in difficult to reach tissues utilizing
both nuclease applications and new editing modalities beyond double
stranded breaks, including base editing, reverse transcriptase
editing, and epigenetic editing. The company is building out its
wholly owned pipeline of potential in vivo gene editing
therapeutics and capabilities and has partnerships with leading
pharmaceutical and biotechnology companies to broaden the reach of
its innovative and proprietary technology platform. Mammoth’s deep
science and industry experience, along with a robust and
differentiated intellectual property portfolio, have enabled the
company to further its mission to transform the lives of patients
and deliver on the promise of CRISPR technologies.
For more information, please visit www.mammoth.bio or follow
Mammoth on LinkedIn or X.
Regeneron Forward-Looking Statements This press
release includes forward-looking statements that involve risks and
uncertainties relating to future events and the future performance
of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”),
and actual events or results may differ materially from these
forward-looking statements. Words such as “anticipate,” “expect,”
“intend,” “plan,” “believe,” “seek,” “estimate,” variations of such
words, and similar expressions are intended to identify such
forward-looking statements, although not all forward-looking
statements contain these identifying words. These statements
concern, and these risks and uncertainties include, among others,
the nature, timing, and possible success and therapeutic
applications of products marketed or otherwise commercialized by
Regeneron and/or its collaborators or licensees (collectively,
“Regeneron’s Products”) and product candidates being developed by
Regeneron and/or its collaborators or licensees (collectively,
“Regeneron’s Product Candidates”) and research and clinical
programs now underway or planned, such as the planned research
programs with Mammoth Biosciences, Inc. to develop in vivo
CRISPR-based gene editing therapies for multiple extrahepatic
tissues and cell types as discussed in this press release; the
potential for any license, collaboration, or supply agreement,
including Regeneron’s agreements with Sanofi and Bayer (or their
respective affiliated companies, as applicable), as well as
Regeneron's collaboration with Mammoth Biosciences discussed in
this press release, to be cancelled or terminated; the extent to
which the results from the research and development programs
conducted by Regeneron and/or its collaborators or licensees
(including those to be conducted as part of the collaboration with
Mammoth Biosciences discussed in this press release) may be
replicated in other studies and/or lead to advancement of product
candidates to clinical trials, therapeutic applications, or
regulatory approval; the potential of utilizing for therapeutic
purposes Regeneron’s expertise in adeno-associated viral vectors
and antibody engineering and Mammoth Biosciences’ expertise in
ultracompact gene editing systems as discussed in this press
release; the likelihood, timing, and scope of possible regulatory
approval and commercial launch of Regeneron’s Product Candidates
and new indications for Regeneron’s Products; uncertainty of the
utilization, market acceptance, and commercial success of
Regeneron’s Products and Regeneron’s Product Candidates and the
impact of studies (whether conducted by Regeneron or others and
whether mandated or voluntary) on any of the foregoing; the ability
of Regeneron’s collaborators, licensees, suppliers, or other third
parties (as applicable) to perform manufacturing, filling,
finishing, packaging, labeling, distribution, and other steps
related to Regeneron’s Products and Regeneron’s Product Candidates;
the ability of Regeneron to manage supply chains for multiple
products and product candidates; safety issues resulting from the
administration of Regeneron’s Products and Regeneron’s Product
Candidates in patients, including serious complications or side
effects in connection with the use of Regeneron’s Products and
Regeneron’s Product Candidates in clinical trials; determinations
by regulatory and administrative governmental authorities which may
delay or restrict Regeneron’s ability to continue to develop or
commercialize Regeneron’s Products and Regeneron’s Product
Candidates; ongoing regulatory obligations and oversight impacting
Regeneron’s Products, research and clinical programs, and business,
including those relating to patient privacy; the availability and
extent of reimbursement of Regeneron’s Products from third-party
payers, including private payer healthcare and insurance programs,
health maintenance organizations, pharmacy benefit management
companies, and government programs such as Medicare and Medicaid;
coverage and reimbursement determinations by such payers and new
policies and procedures adopted by such payers; competing drugs and
product candidates that may be superior to, or more cost effective
than, Regeneron’s Products and Regeneron’s Product Candidates;
unanticipated expenses; the costs of developing, producing, and
selling products; the ability of Regeneron to meet any of its
financial projections or guidance and changes to the assumptions
underlying those projections or guidance; the impact of public
health outbreaks, epidemics, or pandemics (such as the COVID-19
pandemic) on Regeneron's business; and risks associated with
intellectual property of other parties and pending or future
litigation relating thereto (including without limitation the
patent litigation and other related proceedings relating to EYLEA®
(aflibercept) Injection), other litigation and other proceedings
and government investigations relating to the Company and/or its
operations, the ultimate outcome of any such proceedings and
investigations, and the impact any of the foregoing may have on
Regeneron’s business, prospects, operating results, and financial
condition. A more complete description of these and other material
risks can be found in Regeneron’s filings with the U.S. Securities
and Exchange Commission, including its Form 10-K for the year ended
December 31, 2023. Any forward-looking statements are made based on
management’s current beliefs and judgment, and the reader is
cautioned not to rely on any forward-looking statements made by
Regeneron. Regeneron does not undertake any obligation to update
(publicly or otherwise) any forward-looking statement, including
without limitation any financial projection or guidance, whether as
a result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and
social media outlets to publish important information about the
Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website (https://investor.regeneron.com) and its
LinkedIn page
(https://www.linkedin.com/company/regeneron-pharmaceuticals).
Regeneron Contacts:
Investors:Vesna
Tosic+1-914-847-5443vesna.tosic@regeneron.com
Media:Ella
Campbell+1-914-847-7017ella.campbell@regeneron.com
Mammoth Contacts:
Investors:Elaine
Sun+1-650-996-3188esun@mammothbiosci.com
Media: John Kang,
HDMZ+1-309-310-4537john.kang@hdmz.com
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