Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency From the European Commission
August 25 2021 - 7:00AM
Business Wire
Designation supports unmet need for treatment
options for patients with rare form of genetic epilepsy
TSHA-105 is first program in Taysha’s pipeline
to receive designation from European Commission
No approved treatments for the underlying cause
of the disease
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric,
pivotal-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) in both rare
and large patient populations, today announced that it has been
granted orphan drug designation from the European Commission for
TSHA-105, an AAV9-based gene therapy in development for
SLC13A5-related epilepsy.
“SLC13A5 deficiency leads to a debilitating form of genetic
epilepsy in children that results in persistent seizures and
developmental delays, requiring constant supervision and care. With
no available disease modifying treatments for this disease, we are
pleased that TSHA-105 has been granted orphan drug designation from
both the FDA and European Commission, highlighting the global need
and important potential of TSHA-105 in helping treat this form of
epilepsy,” said RA Session II, President, Founder and CEO of
Taysha. “We look forward to working with regulatory agencies to
advance this promising gene replacement strategy as expeditiously
as possible.”
SLC13A5 deficiency is a form of infantile epilepsy caused by
mutations in the SLC13A5 gene. As an autosomal recessive disorder,
two copies of the mutated gene must be inherited for an infant to
be affected. This type of epilepsy manifests as developmental
delay, and seizures beginning within the first few days of life.
SLC13A5 deficiency is a rare disorder, with an estimated prevalence
of 1,900 patients in the United States and in Europe. Current
standards of care are anti-seizure medications which only target
the symptoms and do not address the underlying cause of the
disease.
The European Commission grants orphan drug designation for
medicines being developed for the diagnosis, prevention or
treatment of life-threatening or chronically debilitating
conditions that affect fewer than 5 in 10,000 people in the
European Union. Orphan designation in the European Union includes
benefits such as protocol assistance, reduced regulatory fees and
market exclusivity.
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 to build an extensive, AAV gene therapy pipeline focused on
both rare and large-market indications. Together, we leverage our
fully integrated platform—an engine for potential new cures—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,” and “future” or similar expressions are
intended to identify forward-looking statements. Forward-looking
statements include statements concerning the potential of our
product candidates, including TSHA-105, 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, TSHA-105’s eligibility for accelerated
approval in the United States and Europe, 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, 2020 and our Quarterly Report on
Form 10-Q for the quarter ended June 30, 2021, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20210825005275/en/
Company Contact: Kimberly Lee, D.O. SVP, Corporate
Communications and Investor Relations Taysha Gene Therapies
klee@tayshagtx.com
Media Contact: Carolyn Hawley Canale Communications
carolyn.hawley@canalecomm.com
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