Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that
new and updated data across its genetic medicines portfolio will be
presented at the American Society of Gene and Cell Therapy (ASGCT)
annual conference in Baltimore, Maryland, from May 7 to 11, 2024.
Data from 10 abstracts, including six oral presentations, provide
insight on Regeneron’s approach to overcoming obstacles to clinical
implementation of genetic medicines, from pre-dosing to delivery to
long-term sustained expression. The company will also present
updated data from the Phase 1/2 CHORD trial investigating DB-OTO in
children with profound genetic hearing loss due to mutations of the
otoferlin gene.
“Genetic medicine approaches including gene therapy, gene
editing and gene silencing hold incredible promise for people with
serious, genetically driven diseases, but some common barriers to
practical implementation remain, such as delivery to tissues beyond
the liver and waning efficacy over time,” said Christos Kyratsous,
Ph.D., Senior Vice President and Co-Head of Regeneron Genetic
Medicines. “Regeneron continues to advance methods to overcome
these obstacles through our proprietary delivery approaches
utilizing next-generation viral vectors, particularly specific
retargeting antibodies and innovative payloads. Our data at ASGCT
also details efforts to sustain expression of treatment over time
and better modulate immune response via adeno-associated virus
delivery.”
“We are continuing to dose patients in our clinical trial of
DB-OTO gene therapy for profound hearing loss due to otoferlin
deficiency and are advancing additional gene therapy programs
toward the clinic. The ASGCT presentation will build on promising
early results in the first patient,” said Aris Baras, M.D., Senior
Vice President, Co-Head of Regeneron Genetic Medicines and Head,
Regeneron Genetics Center®. “These results raise hope and
enthusiasm for the field, and we believe that findings from the
program will help us unlock paths forward for gene therapies and
genetic medicines for more patients and diseases.”
Regeneron presentations at ASGCT:
Abstract title |
|
Abstract |
|
Presenting/LeadAuthor |
|
Presentationdate/time(ET) |
|
Oral Presentations |
Intracochlear Administration of DB-OTO Gene Therapy in Pediatric
Patients with Profound Hearing Loss Due to Otoferlin Mutations: The
CHORD Phase 1/2 Open-Label Trial |
|
10 |
|
Lawrence Lustig, Columbia University |
|
Wednesday, May 8, 9:30-9:45AM ET |
|
Antibody-Based AAV Retargeting to Transferrin Receptor Mediates
Efficient Blood Brain Barrier Crossing and In Vivo Gene Delivery to
the CNS in Mice and Non-Human Primates |
|
118 |
|
Kalyani Nambiar |
|
Wednesday, May 8, 4:15-4:30PM ET |
|
Targeted Gene Insertion of Vectorized Monoclonal Antibodies in
Non-Human Primates Overcomes AAV Genome Silencing in the Liver and
Supports High, Sustained In Vivo Expression of Functional
Antibodies |
|
197 |
|
Rachel Sattler |
|
Thursday, May 9, 5:15-5:30pm ET |
|
Retargeting of AAV Using Bispecific Antibodies |
|
218 |
|
Sven Moller-Tank |
|
Thursday, May 9, 5:15-5:30PM ET |
|
Tissue De-Targeting Abrogates Hepatotoxicity and Complement-Related
Thrombotic Complications Associated with High-Dose AAV Gene
Therapies |
|
298 |
|
Andrew Baik |
|
Friday, May 10, 4:15-4:30PM ET |
|
Orthogonal B Cell and Plasma Cell Immunosuppression Strategies
Prevent and Suppress High-Titer Antibody Immunity to Enable AAV
Vector Re-Dosing |
|
353 |
|
Nicholas Giovannone |
|
Saturday, May 11, 8:15-8:30AM ET |
|
Poster Presentations |
AAV Conjugated to Antibodies Against p75NTR: A New Platform to
Deliver Pain Therapeutics to Nociceptive Sensory Neurons |
|
638 |
|
Adina Buxbaum |
|
Wednesday, May 8, 12:00-7:00PM ET |
|
A Process for Identifying AAV and Transgene Integrations in Mouse
and Human Genomes Using Long Read Oxford Nanopore Sequencing |
|
897 |
|
Terrence Turner |
|
Wednesday, May 8, 12:00-7:00PM ET |
|
DNA Leakage of rAAV Under Freeze/Thaw Stress and Analytical Method
Development for Free DNA Characterization |
|
898 |
|
Shuai Li |
|
Wednesday, May 8, 12:00-7:00PM ET |
|
Identification of Degradation Pathways of rAAV8 to Aid Stable Drug
Product Formulation Development |
|
899 |
|
Ariel Chen |
|
Wednesday, May 8, 12:00-7:00PM ET |
|
Lectures |
Engineering CAR-T Cells with Novel Receptor Architectures |
|
N/A |
|
Philip Gregory |
|
Thursday, May 9, 10:55-11:25AM ET |
|
Pressing Challenges in Gene Therapy |
|
N/A |
|
Jim Wang |
|
Saturday, May 11, 8:00-9:45AM ET |
|
About RegeneronRegeneron (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, many 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, 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.
Forward-Looking Statements and Use of Digital
MediaThis 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, including without limitation the
investigational gene therapy DB-OTO as discussed in this press
release as well as Regeneron’s other genetic medicine programs
referenced 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, such as DB-OTO in children with profound genetic hearing
loss due to mutations of the otoferlin gene; the extent to which
the results from the research and development programs conducted by
Regeneron and/or its collaborators or licensees (including those
referenced in this press release) may be further replicated and/or
lead to advancement of product candidates to clinical trials,
therapeutic applications, or regulatory approval; the potential of
the Company’s novel genetic medicine delivery systems and
approaches to immune response modulation discussed or referenced in
this press release; 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), including the studies discussed or referenced in this
press release, on any of the foregoing or any potential regulatory
approval of Regeneron’s Products and Regeneron’s Product Candidates
(such as DB-OTO); 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 (such as DB-OTO) 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;
therapeutic applications, or regulatory approval; 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 potential for any license,
collaboration, or supply agreement, including Regeneron’s
agreements with Sanofi and Bayer (or their respective affiliated
companies, as applicable), to be cancelled or terminated; 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).
|
|
Contacts:Media
Relations Ella
CampbellTel: +1
914-572-4003ella.campbell@regeneron.com |
Investor RelationsVesna TosicTel:
+1 914-847-5443vesna.tosic@regeneron.com |
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