Fully non-viral approach to genetic medicine
employs differentiated gene delivery, gene editing and gene
insertion technology
Progressing two fully non-viral programs in
rare disease with significant unmet patient need
Virtual R&D Day featuring academic experts
and Poseida's leadership and scientific teams to be held today at
10:00am ET / 7:00am PT
SAN
DIEGO, April 17, 2024 /PRNewswire/ -- Poseida
Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell therapy
and genetic medicines company advancing a new class of treatments
for patients with cancer and rare diseases, today announced that
the Company plans to highlight progress across its proprietary
non-viral genetic engineering and delivery platform and rare
disease pipeline during a virtual R&D Day to be held today
at 10:00am ET / 7:00am PT.
"Poseida is forging ahead with a renewed focus on our genetic
medicine portfolio. Our system of proprietary, non-viral tools used
individually or together has the capacity to treat rare genetic
diseases as well as address much more prevalent diseases," said
Kristin Yarema, Ph.D., President
& Chief Executive Officer of Poseida Therapeutics. "In the
short-term, we are laser-focused on progressing our two lead
non-viral candidates within areas of significant opportunity:
P-KLKB1-101 for Hereditary Angioedema, and P-FVIII-101, which
utilizes our fully non-viral stable gene insertion technique to
treat Hemophilia A, a condition affecting approximately 30,000
adults and children in the U.S. alone."
"Poseida has developed a broad suite of fully non-viral,
differentiated genetic engineering technologies, including stable,
potentially site-specific insertion of whole genes, high-fidelity
gene editing, and strength in delivery systems including lipid
nanoparticles," said Blair Madison,
Ph.D., Chief Scientific Officer of Gene Therapy at Poseida
Therapeutics. "We believe this uniquely positions us in the
industry to deliver on the hope and promise of genetic medicines
and bring much-needed therapies to patients in need."
The event will highlight the Company's proprietary genetic
engineering and delivery platform, including its non-viral gene
insertion and gene editing programs. External expert speakers will
include:
- Marc Riedl, M.D., M.S.,
Professor of Medicine and Clinical Director of the U.S. HAEA
Angioedema Center at University of California,
San Diego; and
- Steven W. Pipe, M.D., Professor
of Pediatrics and Pathology, University of
Michigan
Key Gene Therapy R&D Day Topics and Highlights
Gene Therapy Programs
The Company will present advancements in fully non-viral
liver-directed gene therapies highlighting the potential for
functional cures across commercially viable indications.
- P-KLKB1-101 is the Company's lead liver-directed
investigational gene therapy program for the treatment of
hereditary angioedema (HAE), a rare, inherited disorder which
results in the swelling of the limbs, intestinal tract, and airways
which can be both debilitating and life-threatening. The Company
will share data highlighting durable disease correction and high
fidelity in pre-clinical studies using the Company's enhanced
editing technology, Cas-CLOVERTM.
- P-FVIII-101, the Company's second non-viral gene therapy
program, is a liver-directed investigational in vivo gene therapy
for the treatment of Hemophilia A, a hereditary disorder caused by
a deficiency in Factor VIII (FVIII) production resulting in
excessive bleeding occurring either spontaneously or due to trauma.
The Company will share data demonstrating durability and
restoration of FVIII deficiency to near-normal levels in adult
mouse models.
Technology Innovation in Gene Therapy
The Company will highlight significant advancements in its
emerging platform technologies.
- Site-specific Super piggyBac® is a single
enzyme fusion system for site-specific integration, executing clean
DNA gene insertion without double strand breaks, unintended
mutations, or the need for DNA repair. The Company will announce
that the current molecular evolution of its platform yields a
30-fold improvement of DNA expression and efficient targeted cargo
integration at single- and multi-copy sites. These data support the
potential for the Company's non-viral insertion technology as an
efficient and safe approach to achieve sustained DNA integration
and expression to remediate disease.
- Cas-CLOVER is a proprietary high-fidelity nuclease for
enabling clean site-specific gene editing that is engineered for
high specificity. The Company will present data confirming and
validating the benefits and advantages of Cas-CLOVER in multiple
applications and disease areas.
- Novel Lipids and DNA Delivery: The Company is leveraging
proprietary lipids notable for their low immunogenicity, dose
titration potential, and ability to be manufactured at scale and
favorable cost. The Company has achieved multiple breakthroughs in
its delivery technology, including novel lipids for in vivo
delivery of its technology as well as innovations enabling improved
in vivo non-viral DNA delivery.
Video Webcast and Replay
This virtual event and access
to the live webcast is available through the following registration
link: https://wsw.com/webcast/cc/pstx6/1466622.
Registration for this virtual event and access to a replay of
the live webcast will be available on the Investors & Media
section of www.poseida.com. A replay of the webcast will be
available for approximately 90 days following the presentation.
About Poseida Therapeutics, Inc.
Poseida Therapeutics is a clinical-stage biopharmaceutical company
advancing differentiated cell therapies and genetic medicines with
the capacity to cure certain cancers and rare diseases. The
Company's pipeline includes investigational allogeneic CAR-T cell
therapies for both solid tumors and hematologic cancers as well as
investigational in vivo genetic medicines that address patient
populations with high unmet medical need. The Company's approach is
based on its proprietary genetic editing platforms, including its
non-viral piggyBac® DNA Delivery System, Cas-CLOVER™
Site-Specific Gene Editing System, Booster Molecule and
nanoparticle gene delivery technologies, as well as in-house GMP
cell therapy manufacturing. The Company has formed a global
strategic collaboration with Roche to unlock the promise of cell
therapies for patients with hematologic malignancies. Learn more at
www.poseida.com and connect with Poseida on X and
LinkedIn.
Forward-Looking Statements
Statements contained in
this press release regarding matters that are not historical facts
are "forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995. Such forward-looking
statements include statements regarding, among other things,
expected plans with respect to clinical trials, including timing of
clinical data updates; anticipated timelines and milestones with
respect to the Company's development programs and manufacturing
activities and capabilities; the potential capabilities and
benefits of the Company's technology platforms and product
candidates, including the efficacy and safety profile of such
product candidates; the quotes from Drs. Yarema and Madison; and
the Company's plans and strategy with respect to developing its
technologies and product candidates. Because such statements are
subject to risks and uncertainties, actual results may differ
materially from those expressed or implied by such forward-looking
statements. These forward-looking statements are based upon the
Company's current expectations and involve assumptions that may
never materialize or may prove to be incorrect. Actual results
could differ materially from those anticipated in such
forward-looking statements as a result of various risks and
uncertainties, which include, without limitation, the Company's
reliance on third parties for various aspects of its business;
risks and uncertainties associated with development and regulatory
approval of novel product candidates in the biopharmaceutical
industry; the Company's ability to retain key scientific or
management personnel; and the other risks described in the
Company's filings with the Securities and Exchange Commission.
All forward-looking statements contained in this press release
speak only as of the date on which they were made. The Company
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made, except as required by law.
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SOURCE Poseida Therapeutics, Inc.
