Taysha Gene Therapies Announces Multiple Data Presentations & Workshop Presentations at the 24th Annual Meeting of the Americ...
May 05 2021 - 7:00AM
Business Wire
TSHA-104 increased COX1 activity in brain and
muscle and restored elevation of blood lactate on exhaustive
exercise in dose-dependent manner in SURF1 knockout mice
TSHA-105 significantly reduced plasma citrate
levels, normalized EEG brain activity, and reduced the number of
seizures and seizure susceptibility in SLC13A5 knockout mice
On track to file IND/CTA for TSHA-104 in
SURF1-associated Leigh syndrome in second half of 2021
IND/CTA-enabling studies for TSHA-105 in
SLC13A5 deficiency are ongoing
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 preclinical
data from its investigational gene therapy programs will be
presented at the 24th Annual Meeting of the American Society of
Gene & Cell Therapy (ASGCT), which will be held virtually May
11-14, 2021.
“Presentations at this year’s ASGCT will highlight the positive
preclinical results for TSHA-104 in SURF1-associated Leigh syndrome
and TSHA-105 in SLC13A5 deficiency that support our advancement of
these programs,” said RA Session II, President, Founder and Chief
Executive Officer of Taysha. “Among the compelling data, we have
shown that TSHA-104 as a single intrathecally administered gene
replacement therapy was effective in improving SURF1
deficiency-related dysfunctions, such as diminished COX1 activity
in brain and muscle and blood lactate on exhaustive exercise in a
dose-dependent manner in SURF1 knockout mice. In SLC13A5
deficiency, CSF-delivered TSHA-105 resulted in improved EEG
activity and reduced seizure susceptibility in SLC13A5 knockout
mice. We remain on track to file an IND/CTA in SURF1-associated
Leigh syndrome in the second half of this year and continue to
advance TSHA-105 in SLC13A5 deficiency towards the clinic.”
Summary of Abstracts and Posters (all times in Eastern
Time)
Thursday, May 13, 2021 at 7:00 – 7:15
pm
- Abstract Presentation title: Gene Replacement Therapy for
SURF1-Related Leigh Syndrome Using AAV9
- Session title: Clinical Trials and Advanced Preclinical Studies
for Neurologic Diseases
- Abstract number: 165
- Authors: Qinglan Ling, Matthew Rioux, Steven Gray
- Presenter: Qinglan Ling, Ph.D., of UT Southwestern Medical
Center
- COX activity was partially and significantly rescued in all
tested tissues of AAV9/hSURF1-treated mice via intrathecal (IT)
delivery
- AAV9/hSURF1-treated mice demonstrated a dose-dependent increase
in hSURF1 mRNA expression, restoration of MT-CO1 protein expression
in the brain
- Gene replacement treatment also mitigated the lactic acidosis
upon exhaustive exercise at mid-age
Thursday, May 13, 2021 at 7:00 – 7:15
pm
- Abstract Presentation title: scAAV9 Gene Replacement Therapy
for Epileptic SLC13A5 Deficiency
- Session title: AAV Therapies for Neurological and Sensory
Diseases
- Abstract number: 137
- Authors: Rachel Marion Bailey, Lauren Bailey, Morgan
Schackmuth, Irvin Garza
- Presenter: Rachel Bailey, Ph.D., of UT Southwestern Medical
Center
- TSHA-105-treated knockout mice demonstrated significantly
decreased plasma citrate levels compared to knockout mice treated
with vehicle
- TSHA-105-treated knockout mice had reduced spike train activity
and seizure frequency on electroencephalogram (EEG)
Tuesday, May 11, 2021 at 8:00 – 10:00
am
- Poster title: Novel AAV Capsids for Enhanced Gene Transfer to
the Cerebellum, Spinal Cord, and Schwann Cells
- Session: AAV Vectors – Virology and Vectorology
- Abstract number: 314
- Authors: Xin Chen, Thomas Dong, Widler Casy, Yuhui Hu, Daphne
Chen, Thomas McCown, Steven Gray
- A capsid DNA shuffling and directed evolution process was
pursued to generate new AAV variants for nervous system gene
transfer. In the IT cohort, more than 15 variants had
biodistribution values at least ten times greater than AAV9 with
reduced biodistribution to the liver
- Results demonstrated that some variants could be used to treat
cerebellar diseases with Purkinje cell involvement and other
variants could be used to treat peripheral demyelinating
neuropathies with Schwann cell involvement
The ASGCT abstracts are now available at
https://www.cell.com/molecular-therapy-family/molecular-therapy/issue?pii=S1525-0016(21)X0002-0.
ASGCT-Sponsored Pre-Meeting Workshops (all times in Eastern
Time)
Monday, May 10, 2021 at 12:55 – 1:05
pm
- Workshop title: Transitioning From Academics to Industry
- Session 2: Learning From Experience: Case Studies of
Transitions from Academia to Industry
- Topic: Moving from Academic Vector Production to Commercial
Scale Manufacturing
- Speaker: Frederick Porter, Ph.D., Chief Technical Officer of
Taysha Gene Therapies
Monday, May 10, 2021 at 3:00 – 3:15
pm
- Workshop title: Emerging Issues in Market Access
- Session 1: Gene Therapy Investment and Capital
- Keynote: Attracting Capital and Building a Company in the Gene
Therapy Space
- Speaker: RA Session II, President, Founder and Chief Executive
Officer of Taysha Gene Therapies
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, 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, 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/20210505005401/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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