Capricor Therapeutics to Present Exosome Platform Updates at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting
09 Maio 2024 - 10:15AM
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment of rare diseases, today announced that an abstract
featuring new preclinical data highlighting the therapeutic
potential of its StealthX™ exosome platform technology has
been selected for an oral presentation at the ASGCT 27th Annual
Meeting taking place in Baltimore, Maryland from May 7–11, 2024.
The findings highlight a potential exosome-based approach for the
treatment of arginase-1 deficiency (ARG1-D), a rare genetic
metabolic disease characterized by complete or partial lack of the
enzyme arginase in the liver and red blood cells.
“The findings from this preclinical study have
further characterized our StealthX™ exosome platform and provide a
novel approach for exosome-based enzyme-replacement therapies which
aim to replace a deficient or absent enzyme,” said Linda Marbán,
Ph.D., Capricor’s chief executive officer. “We believe our
StealthX™ platform provides an opportunity with potentially
broad applications and our data continues to support the concept of
exosomes as a suitable delivery vehicle for a variety of payloads.
We continue to focus our exosome platform in two main areas, the
development of therapeutics and vaccines, and our goal is to
leverage partnership opportunities to further expand and advance
these opportunities.”
Study Overview and
Findings:
- In this study, exosomes were
engineered to express human Arg1 enzyme inside of the exosomes and
were evaluated for their in vitro functionality as an approach for
the potential treatment of ARG1-D.
- Results showed the Arg1-exosomes
were enzymatically active and able to convert arginine into urea in
vitro. In addition, the Arg1-exosomes were capable of delivering
the Arg1 protein into 293F and HepG2 cells in a time and
dose-dependent manner, contrary to human recombinant Arg1 protein
alone at the same or higher dose.
- The data suggests that the
Arg1-exosomes were able to not only catalyze arginine efficiently
at a lower delivered dose to cells in vitro but also protect the
Arg1 protein to be delivered into target cells and retain its
enzymatic activity in cells.
- Taken together, Capricor’s
StealthX™ platform of engineered Arg1-exosomes as tested in
vitro have the potential to serve as an enzyme replacement therapy
to deliver Arg1 to hepatocytes and possibly have potential clinical
benefits for the treatment of ARG1-D.
Presentation details: |
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Title: |
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Exosome-mediated intracellular delivery of arginase-1: a
potential application for the treatment of arginase-1
deficiency |
Oral session: |
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Exosomes, Virus like Particles and LNPs |
Presenting author: |
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Li-En Hsieh, Ph.D., Principal Scientist |
Date: |
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May 9, 2024 at 4:45 p.m. ET |
Abstract number: |
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223 |
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The abstract will be made available on the
publications section of the Capricor website following the
conclusion of the meeting.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a
biotechnology company dedicated to advancing transformative cell
and exosome-based therapeutics to redefine the treatment landscape
for rare diseases. At the forefront of our innovation is our lead
product candidate, CAP-1002 — an allogeneic cardiac-derived cell
therapy. Extensive preclinical and clinical studies have shown
CAP-1002 to demonstrate immunomodulatory, antifibrotic, and
regenerative actions specifically tailored for dystrophinopathies
and heart disease. CAP-1002 is currently advancing through Phase 3
clinical development for the treatment of Duchenne muscular
dystrophy (DMD). Capricor is also harnessing the power of our
exosome technology, using our proprietary StealthX™ platform in
preclinical development focused on the areas of vaccinology,
targeted delivery of oligonucleotides, proteins and small molecule
therapeutics to potentially treat and prevent a diverse array of
diseases. At Capricor, we stand committed to pushing the boundaries
of possibility and forging a path toward transformative treatments
for those in need. For more information, visit capricor.com,
and follow Capricor
on Facebook, Instagram and Twitter.
Cautionary Note Regarding
Forward-Looking Statements
Statements in this press release regarding the
efficacy, safety, and intended utilization of Capricor’s product
candidates; the initiation, conduct, size, timing and results of
discovery efforts and clinical trials; the pace of enrollment of
clinical trials; plans regarding regulatory filings, future
research and clinical trials; regulatory developments involving
products, including the ability to obtain regulatory approvals or
otherwise bring products to market; manufacturing capabilities;
dates for regulatory meetings; statements about our financial
outlook; the ability to achieve product milestones and to receive
milestone payments from commercial partners; plans regarding
current and future collaborative activities and the ownership of
commercial rights; scope, duration, validity and enforceability of
intellectual property rights; future revenue streams and
projections; expectations with respect to the expected use of
proceeds from the recently completed offerings and the anticipated
effects of the offerings; and any other statements about Capricor’s
management team’s future expectations, beliefs, goals, plans or
prospects constitute forward-looking statements within the meaning
of the Private Securities Litigation Reform Act of 1995. Any
statements that are not statements of historical fact (including
statements containing the words “believes,” “plans,” “could,”
“anticipates,” “expects,” “estimates,” “should,” “target,” “will,”
“would” and similar expressions) should also be considered to be
forward-looking statements. There are a number of important factors
that could cause actual results or events to differ materially from
those indicated by such forward-looking statements. More
information about these and other risks that may impact Capricor’s
business is set forth in Capricor’s Annual Report on Form 10-K for
the year ended December 31, 2023, as filed with the Securities and
Exchange Commission on March 11, 2024. All forward-looking
statements in this press release are based on information available
to Capricor as of the date hereof, and Capricor assumes no
obligation to update these forward-looking statements.
Capricor has entered into
a partnership for the exclusive commercialization and
distribution of CAP-1002 for DMD in the United States and Japan
with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma,
Inc.), subject to regulatory approval. CAP-1002 is an
Investigational New Drug and is not approved for any indications.
None of Capricor’s exosome-based candidates have been approved for
clinical investigation.
For more information, please
contact:
Capricor Company Contact:AJ
Bergmann, Chief Financial Officerabergmann@capricor.com
858.727.1755
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