Taysha Gene Therapies to Host Virtual R&D Day
June 17 2021 - 7:00AM
Business Wire
Two-day R&D day on June 28 and 29, 2021 at
10:00 am ET will highlight progress across its pipeline and will
feature presentations from key opinion leaders
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 will host
its first two-day virtual research and development (R&D) day
for analysts and investors. The event will be webcast live on June
28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day.
The event will highlight the company’s R&D progress, focused
on advancement of its early- and late-stage investigational
programs. Topics of discussion will include:
Day 1 – June 28, 2021
- TSHA-120 (GAN): Pivotal-stage AAV9 gene replacement therapy
program for the treatment of giant axonal neuropathy (GAN), a rare
autosomal recessive disease of the central and peripheral nervous
systems caused by loss-of-function gigaxonin gene mutations.
- TSHA-101 (GM2 gangliosidosis): The first bicistronic AAV9 gene
therapy in clinical development designed to deliver two genes, HEXA
and HEXB, comprising the alpha and beta sub-units of beta
hexosaminidase A, intrathecally for the treatment of GM2
gangliosidosis, also called Tay-Sachs or Sandhoff disease. TSHA-101
is currently in Phase 1/2 development.
- TSHA-118 (CLN1 disease): AAV9-based gene therapy designed to
express a human codon-optimized CLN1 transgene to potentially treat
CLN1 disease, a rapidly progressing rare lysosomal storage disease
with no currently approved treatments. This gene replacement
therapy program is currently under an open IND, with initiation of
a Phase 1/2 trial expected in the second half of 2021.
- TSHA-102 (Rett syndrome): AAV9-based gene therapy in
development for Rett syndrome, a severe neurodevelopmental
disorder, designed to deliver MECP2, as well as a novel miRARE
platform that regulates transgene expression on a cell-by-cell
basis. This regulated gene replacement therapy is currently in
IND/CTA-enabling studies, with an IND/CTA filing expected in the
second half of 2021.
Day 2 – June 29, 2021
- TSHA-104 (SURF1-associated Leigh syndrome): AAV9-based gene
therapy with a transgene encoding the human SURF1 protein to
potentially treat SURF1-associated Leigh syndrome, a monogenic
mitochondrial disorder with no currently approved treatments. This
gene replacement therapy program is currently in IND/CTA-enabling
studies, with an IND/CTA filing expected in the second half of
2021.
- TSHA-105 (SLC13A5 deficiency): AAV9-based gene therapy
expressing human SLC13A5 protein to potentially treat SLC13A5
deficiency, a rare autosomal recessive epileptic encephalopathy
characterized by the onset of seizures within the first few days of
life. This gene replacement therapy program is currently in
IND/CTA-enabling studies.
- TSHA-103 (SLC6A1 haploinsufficiency disorder): Gene replacement
therapy constructed from a codon-optimized version of the human
SLC6A1 gene packaged within an AAV9 viral vector for the treatment
of SLC6A1 haploinsufficiency disorder. This program is currently in
IND/CTA-enabling studies.
- TSHA-111-LAFORIN and TSHA-111-MALIN (Lafora disease):
Recombinant AAV9 viral vectors with miRNA targeting the knockdown
of GYS1 for the treatment of Lafora disease. These programs are
currently in IND/CTA-enabling studies.
- TSHA-112 (APBD): Recombinant AAV9 viral vector with miRNA
targeting the knockdown of GYS1 for the treatment of Adult
Polyglucosan Body Disease (APBD). This program is currently in
IND/CTA-enabling studies.
- Additional preclinical programs, including tauopathies
(TSHA-113, an AAV9 viral vector that utilizes AAV-mediated gene
silencing to potentially deliver life-long reduction of tau protein
levels in neurons following a single dose) and Angelman syndrome
(TSHA-106, an AAV9 viral vector designed for shRNA-mediated
knockdown of UBE3A-ATS).
The event will feature presentations from Taysha senior leaders
and the following key opinion leaders:
- Steven Gray, Ph.D. Chief Scientific Advisor, Taysha Gene
Therapies Associate Professor, Department of Pediatrics at UT
Southwestern
- Berge Minassian, M.D. Chief Medical Advisor, Taysha Gene
Therapies Division Chief, Pediatric Neurology at UT
Southwestern
- Rachel Bailey, Ph.D. ssistant Professor, Department of
Pediatrics at UT Southwestern
- Kimberly Goodspeed, M.D. Assistant Professor, Department of
Pediatrics, Neurology and Psychiatry at UT Southwestern
Registration for this event is available through LifeSci Events.
A live video webcast will be available in the “Events & Media”
section of the Taysha corporate website. An archived version of the
event will be available on the website for 60 days.
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 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, the potential market opportunity for these product
candidates, our corporate growth plans and our plans to establish a
commercial-scale cGMP manufacturing facility to provide
preclinical, clinical and commercial supply. 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, which is 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/20210617005380/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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