Taysha Gene Therapies to Present Biodistribution Data from an Analysis Evaluating AAV9 Gene Therapy Delivery at the Upcoming 31st Annual ESGCT Congress
22 Outubro 2024 - 9:00AM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company),
a clinical-stage biotechnology company focused on advancing
adeno-associated virus (AAV)-based gene therapies for severe
monogenic diseases of the central nervous system (CNS), today
announced that it will present biodistribution data from an
analysis of 28 non-human primates (NHP) evaluating the delivery of
AAV9 gene therapy vectors across five different studies using
intrathecal (IT) delivery by lumbar puncture (four studies) or
intra-cisterna magna (ICM) injection (one study). The data will be
presented during a poster presentation at the upcoming 31st
Annual Congress of the European Society of Gene &
Cell Therapy (ESGCT), taking place in Rome, Italy from October
22-25, 2024. These findings reinforce Taysha’s clinical development
approach utilizing IT administration to deliver AAV-based gene
therapies designed to treat the genetic root cause of CNS diseases,
including the TSHA-102 program in clinical evaluation for children,
adolescents and adults living with Rett syndrome.
“Findings from an analysis across five NHP studies showed that
both IT and ICM administration led to comparable, consistent and
widespread biodistribution of AAV9 vector throughout the brain and
spinal cord regions,” said Sukumar Nagendran, M.D., President and
Head of Research & Development at Taysha. “Importantly, these
findings further support the clinical potential of IT
administration as an effective, safe and minimally invasive
delivery approach for broad targeting of the CNS that has potential
for outpatient use in both children and adults. We believe the NHP
biodistribution data, together with our additional preclinical
data, reaffirm our clinical development strategy utilizing IT
administration and provide translational support for the broad
clinical effect reported following treatment with TSHA-102 in both
our REVEAL adolescent/adult trial and REVEAL pediatric trial."
Poster presentation details are as follows:
Title: Broad CNS Biodistribution of AAV9-based
Gene Therapies Delivered by Intrathecal Lumbar Puncture in
Non-Human Primates
Presenters: Nino Devidze, MS, Ph.D., Clinical
Development Lead and Emdadul Haque, Ph.D., Senior Director,
Translational Sciences at Taysha Gene Therapies
Date and Time: Wednesday October 23, 2024, from
13:30 to 15:00 CEST
Poster Session: CNS & Sensory Diseases
Poster Number: P0284
Additional details on the meeting can be found at the 31st
Annual ESGCT Congress website.
About TSHA-102TSHA-102 is a self-complementary
intrathecally delivered AAV9 investigational gene transfer therapy
in clinical evaluation for Rett syndrome. Designed as a one-time
treatment, TSHA-102 aims to address the genetic root cause of the
disease by delivering a functional form of MECP2 to cells in the
CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory
Element (miRARE) technology designed to mediate levels of MECP2 in
the CNS on a cell-by-cell basis without risk of overexpression.
TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast
Track and Orphan Drug and Rare Pediatric Disease designations from
the FDA, Orphan Drug designation from the European Commission and
Innovative Licensing and Access Pathway designation from the
Medicines and Healthcare products Regulatory Agency.
About Rett SyndromeRett syndrome is a rare
neurodevelopmental disorder caused by mutations in the X-linked
MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is
essential for regulating neuronal and synaptic function in the
brain. The disorder is characterized by loss of communication and
hand function, slowing and/or regression of development, motor and
respiratory impairment, seizures, intellectual disabilities and
shortened life expectancy. Rett syndrome progression is divided
into four key stages, beginning with early onset stagnation at 6 to
18 months of age followed by rapid regression, plateau and late
motor deterioration. Rett syndrome primarily occurs in females and
is one of the most common genetic causes of severe intellectual
disability. Currently, there are no approved disease-modifying
therapies that treat the genetic root cause of the disease. Rett
syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is
estimated to affect between 15,000 and 20,000 patients in the U.S.,
EU, and U.K.
About Taysha Gene TherapiesTaysha Gene
Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company
focused on advancing adeno-associated virus (AAV)-based gene
therapies for severe monogenic diseases of the central nervous
system. Its lead clinical program TSHA-102 is in development for
Rett syndrome, a rare neurodevelopmental disorder with no approved
disease-modifying therapies that address the genetic root cause of
the disease. With a singular focus on developing transformative
medicines, Taysha aims to address severe unmet medical needs and
dramatically improve the lives of patients and their caregivers.
The Company’s management team has proven experience in gene therapy
development and commercialization. Taysha leverages this
experience, its manufacturing process and a clinically and
commercially proven AAV9 capsid in an effort to rapidly translate
treatments from bench to bedside. For more information, please
visit www.tayshagtx.com.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Words such as
“anticipates,” “believes,” “expects,” “intends,” “projects,”
“plans,” and “future” or similar expressions are intended to
identify forward-looking statements. Forward-looking statements
include, but are not limited to, statements concerning the
potential of TSHA-102 and Taysha’s other product candidates to
positively impact quality of life and alter the course of disease
in the patients Taysha seeks to treat, its research, development
and regulatory plans for its product candidates, the potential for
these product candidates to receive regulatory approval from the
FDA or equivalent foreign regulatory agencies, and whether, if
approved, these product candidates will be successfully distributed
and marketed and the potential market opportunity for Taysha’s
product candidates. Forward-looking statements are based on
management’s current expectations and are subject to various risks
and uncertainties that could cause actual results to differ
materially and adversely from those expressed or implied by such
forward-looking statements. Accordingly, these forward-looking
statements do not constitute guarantees of future performance, and
you are cautioned not to place undue reliance on these
forward-looking statements. Risks regarding Taysha’s business are
described in detail in its SEC filings, including in
Taysha’s Annual Report on Form 10-K for the full-year
ended December 31, 2023 and Quarterly Report on Form 10-Q for
the quarter ended June 30, 2024, which are available on the
SEC’s website at www.sec.gov. Additional information will be
made available in other filings that Taysha makes from time to time
with the SEC. These forward-looking statements speak only as
of the date hereof, and Taysha disclaims any obligation to update
these statements except as may be required by law.
Company Contact:Hayleigh Collins Director, Head
of Corporate Communications and Investor RelationsTaysha Gene
Therapies, Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyInizio
EvokeCarolyn.hawley@inizioevoke.com
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