Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage
biotechnology company with a mission to discover, develop and
deliver potentially curative therapies that address the underlying
causes of heart disease, announced that several abstracts have been
accepted for presentation at the American Society of Gene and Cell
Therapy (ASGCT) 26th Annual Meeting detailing the company’s
expanded capabilities for manufacturing and discovering genetic
medicines for heart disease.
“Tenaya was founded with a singular focus on developing novel
medicines for rare and prevalent forms of heart disease. To be a
leader in this space requires a sustained commitment to
differentiated platform capabilities that enable us to optimize the
product profile for our therapeutic candidates and to address new
conditions where there is high unmet need,” said Faraz Ali, Chief
Executive Officer of Tenaya. “The presentations at this year’s
ASGCT highlight innovations in manufacturing, capsid engineering
and gene editing, that support the advancement of our current
pipeline and create new opportunities for future pipeline
candidates. We look forward to sharing work on additional
innovative enabling technologies and genetic medicine programs
at future meetings.”
The ASGCT 26th Annual Meeting is being held in Los Angeles,
California May 16-20, 2023. Details of the company’s upcoming
presentations at ASGCT are available below:
Manufacturing and Process DevelopmentTN-201 is
an adeno-associated virus (AAV) gene therapy being advanced into
the clinic for the treatment of MYBPC3-associated hypertrophic
cardiomyopathy (HCM). Clinical supply of TN-201 was manufactured
under current Good Manufacturing Practice regulations at Tenaya’s
Genetic Medicines Manufacturing Center using the company’s
proprietary Sf9 recombinant baculovirus (Sf9/rBV) production
process at the 1000L scale. At ASGCT, Tenaya researchers will
present data detailing the scale up, formulation and purification
techniques deployed in the successful initial manufacture of
TN-201.
In addition to the Sf9/rBV manufacturing platform, Tenaya has
also internalized the HEK293 manufacturing platform up to the 200L
scale. Tenaya researchers will present data on a novel small
molecule additive that increases the productivity of HEK293-based
AAV manufacturing processes, which may have implications for
improved scalability, yields and costs.
Wednesday Poster Session, May
17, 2023, at 12 pm PTDevelopment of Cost-Effective and
Scalable Recombinant Baculovirus Production Process for the
Manufacturing of AAV (Abstract #409)Lead author:
Charles Feathers, Process Development Manager, Tenaya
Therapeutics
Development of a Comprehensive and
Risk-Based Viral Safety Assurance Strategy for the Manufacturing of
AAV Gene Therapy (Abstract #408)Lead author:
Samantha Jones, Senior Manager, Process Development, Tenaya
Therapeutics
Titer Boosting of HEK293-based AAV
Manufacturing Process using Proprietary Small Molecule Additive and
Successful Scale up to 200L (Abstract #407)Lead
author: Jackson Leong, Associate Scientist, Process
Development, Tenaya Therapeutics
Thursday Poster Session, May
18, 2023, 12 pm PTDevelopment of Rational Formulation for
the Delivery of AAV Viral Vector for Treatment of Heart Disease
(Abstract #1010)Lead author: Joseph Woods, Process
Development Associate, Tenaya Therapeutics
Capsid EngineeringAAV9 is the current capsid of
choice for cardiovascular indications and the capsid being used in
Tenaya’s TN-201 and TN-401 gene therapy product candidates. Through
rigorous testing across multiple species, Tenaya has identified
several novel AAV capsids with greater transduction efficiency for
the heart and/or de-targeting of the liver compared to AAV9, that
may be used in second generation approaches and for new product
candidates. At ASGCT, Tenaya scientists will present new data from
non-human primate studies for previously identified novel
capsids.
Wednesday Poster Session, May
17, 2023, at 12 pm PTEngineering Novel AAV Capsids for
Cardiac Gene Delivery (Abstract #463)Lead author:
Ze Cheng, Ph.D., Senior Scientist, Tenaya Therapeutics
Gene EditingPhospholamban (PLN) plays a key
role in the functioning of the heart, and carriers of the R14del
pathogenic variant of the PLN gene have a high risk of developing a
form of dilated cardiomyopathy that may result in severe
arrhythmia, heart failure, and sudden cardiac death. At ASGCT,
Tenaya scientists will show data for the first time in which
genetic editing using a single AAV vector restores heart function
and survival in a severe murine model of PLN cardiomyopathy caused
by the R14del mutation. This foundational research will inform
future drug development efforts for PLN cardiomyopathies, as well
as for additional cardiomyopathies in which gene editing may be an
advantageous approach to correcting specific pathogenetic drivers
underlying disease.
Thursday Poster Session, May
18, 2023, at 12 pm PTGene Editing of R14del Mutation in
PLN Rescues PLN-R14del-Associated Cardiomyopathy (Abstract
#1113)Lead author: Huanyu Zhou, Ph.D., Senior
Scientist, Tenaya Therapeutics
To view full event programming, please visit the ASGCT 26th
Annual Meeting website. Following the conference, Tenaya’s
presentations will be available in the “Our Science” section of the
company’s website.
About Tenaya TherapeuticsTenaya Therapeutics is
a clinical-stage biotechnology company committed to a bold mission:
to discover, develop and deliver potentially curative therapies
that address the underlying drivers of heart disease. Leveraging
its integrated and interrelated Gene Therapy, Cellular Regeneration
and Precision Medicine platforms and proprietary core capabilities,
the company is advancing a pipeline of novel therapies with diverse
treatment modalities for rare genetic cardiovascular disorders and
more prevalent heart conditions. Tenaya’s most advanced candidates
include TN-201, a gene therapy for MYBPC3-associated hypertrophic
cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated
arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301,
a small molecule HDAC6 inhibitor being initially developed for
heart failure with preserved ejection fraction (HFpEF). Tenaya also
has multiple early-stage programs progressing through preclinical
development. For more information, visit
www.tenayatherapeutics.com.
Forward-looking StatementThis press release
contains forward-looking statements as that term is defined in
Section 27A of the Securities Act of 1933 and Section 21E of the
Securities Exchange Act of 1934. Statements in this press release
that are not purely historical are forward-looking statements.
Words such as “look forward,” “may,” and similar expressions are
intended to identify forward-looking statements. Such
forward-looking statements include, among other things, statements
regarding Tenaya’s pipeline development plans; future planned
presentations; the potential implications for Tenaya’s novel small
molecule additive on the productivity of HEK293-based AAV
manufacturing processes; the therapeutic of novel capsids
identified by Tenaya and the potential to use them for new product
candidates; and statements by Tenaya’s Chief Executive Officer. The
forward-looking statements contained herein are based upon Tenaya’s
current expectations and involve assumptions that may never
materialize or may prove to be incorrect. These forward-looking
statements are neither promises nor guarantees and are subject to a
variety of risks and uncertainties, including but not limited to:
risks associated with the process of discovering, developing and
commercializing drugs that are safe and effective for use as human
therapeutics and operating as an early stage company; the
availability of data at the referenced times; the timing, scope and
likelihood of regulatory filings and approvals for Tenaya’s product
candidates; Tenaya’s ability to initiate and complete clinical
trials for its product candidates; the potential for any clinical
trial results to differ from preclinical, interim, preliminary,
topline or expected results; the level of costs associated with
Tenaya’s research, development, manufacturing and other activities
and the company’s ability to raise any additional funding it will
need to continue to pursue its business and product development
plans; Tenaya’s ability to successfully operate a manufacturing
facility for clinical or commercial supply; Tenaya’s
commercialization and marketing capabilities and strategy; the loss
of key scientific or management personnel; competition in the
industry in which Tenaya operates; Tenaya’s reliance on third
parties; Tenaya’s ability to obtain and maintain intellectual
property protection for its product candidates; general economic
and market conditions; and other risks. Information regarding the
foregoing and additional risks may be found in the section entitled
“Risk Factors” in documents that Tenaya files from time to time
with the Securities and Exchange Commission. These forward-looking
statements are made as of the date of this press release, and
Tenaya assumes no obligation to update or revise any
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by law.
Contacts
Investors |
Media |
Michelle CorralVice President,
Investor Relations andCorporate CommunicationsTenaya
TherapeuticsIR@tenayathera.com |
Wendy RyanTen Bridge
CommunicationsWendy@tenbridgecommunications.com |
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