Taysha Gene Therapies Announces Fast Track Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome
August 24 2023 - 8:00AM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene
therapy company focused on developing and commercializing AAV-based
gene therapies for the treatment of monogenic diseases of the
central nervous system (CNS), today announced the U.S. FDA has
granted Fast Track Designation (FTD) to TSHA-102, a
self-complementary intrathecally delivered AAV9 gene transfer
therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes
the 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.
FTD is designed to help treatments reach patients faster by
facilitating the development and expediting the review of therapies
with potential to address unmet medical needs for a serious or
life-threatening condition. Benefits of FTD to programs include
early and frequent interactions with the FDA during the clinical
development process and, if relevant criteria are met, the FDA may
also review portions of a marketing application before the sponsor
submits the complete application.
“We are pleased to receive FTD from the FDA, which underscores
the significant unmet medical need in patients with Rett syndrome
and the potential of TSHA-102 to serve as a meaningful treatment
option,” said Sukumar Nagendran, M.D., President and Head of
R&D of Taysha. “Initial data from the first adult patient in
Canada with severe disease dosed with TSHA-102 is encouraging, and
we expect to dose the second patient in our ongoing REVEAL Phase
1/2 adult trial in the current quarter. We look forward to
expanding the clinical evaluation to earlier stages of disease
progression following recent FDA clearance to initiate clinical
development of TSHA-102 in pediatric patients in the United
States.”
Rumana Haque-Ahmed, Senior Vice President, Regulatory Affairs of
Taysha, added, “Rett syndrome is a devastating neurodevelopmental
disorder that can lead to motor and respiratory impairment, loss of
communication, and ultimately shortened life expectancy. Currently,
there are no approved disease-modifying therapies that treat the
genetic root cause of the disease. Receiving FTD for important
aspects of the disease is a critical milestone that furthers our
ability to accelerate the development of TSHA-102 with the
potential to address a serious condition and significant unmet
medical need in patients living with this devastating disease. We
look forward to having continued discussions with the FDA, with the
goal of bringing TSHA-102 to patients as safely and expeditiously
as possible.”
TSHA-102 is being evaluated in the REVEAL Phase 1/2 adult trial
in Canada. The U.S. FDA cleared the IND application for
TSHA-102 in pediatric patients with Rett syndrome, and the Company
expects to dose the first pediatric patient in the first quarter of
2024.
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by
mutations in the X-linked MECP2 gene, which is a gene that’s
essential for neuronal and synaptic function in the brain. The
disorder is characterized by intellectual disabilities, loss of
communication, seizures, slowing and/or regression of development,
motor and respiratory impairment, and shortened life expectancy.
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 UK.
About Taysha Gene
Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to
eradicate monogenic CNS disease. With a singular focus on
developing curative medicines, we aim to rapidly translate our
treatments from bench to bedside. We have combined our team’s
proven experience in gene therapy drug development and
commercialization with the world-class UT Southwestern Gene Therapy
Program. Together, we leverage our fully integrated platform with a
goal of dramatically improving patients’ lives. More information is
available at www.tayshagtx.com.
Forward-Looking Statements
This 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 statements concerning the
potential benefits of FTD for TSHA-102, the potential of our
product candidates, including the reproducibility and durability of
any favorable results initially seen in our first patient dosed in
the REVEAL trial and including our preclinical product candidates,
to positively impact quality of life and alter the course of
disease in the patients we seek to treat, our research, development
and regulatory plans for our 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 these 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 our business are
described in detail in our Securities and Exchange Commission
(“SEC”) filings, including in our Annual Report on Form 10-K for
the full-year ended December 31, 2022, and our Quarterly Report on
Form 10-Q for the quarter ended June 30, 2023, both of which are
available on the SEC’s website at www.sec.gov. Additional
information will be made available in other filings that we make
from time to time with the SEC. Such risks may be amplified by the
impacts of the COVID-19 pandemic. These forward-looking statements
speak only as of the date hereof, and we disclaim any obligation to
update these statements except as may be required by law.
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communications carolyn.hawley@canalecomm.com
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