Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial Under Investigation for the Treatment of Rett Syndrome
June 05 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 that the first
patient has been dosed with TSHA-102 in the Phase 1/2 REVEAL trial
evaluating the safety and preliminary efficacy of TSHA-102 in adult
patients with Rett syndrome. TSHA-102 is a self-complementary
intrathecally delivered AAV9 investigational gene transfer therapy
that utilizes a novel miRNA-Responsive Auto-Regulatory Element
(miRARE) platform designed to regulate cellular MECP2 expression.
The study is being conducted at CHU Sainte-Justine, the Université
de Montréal mother and child university hospital centre in
Montreal, Canada.
“Dosing of the first adult patient marks the beginning of
clinical evaluation of TSHA-102 in the Phase 1/2 REVEAL trial, and,
to our knowledge, the first time a gene therapy has ever been
evaluated in a clinical setting for the treatment of Rett
syndrome,” said Sukumar Nagendran, M.D., President, and Head of
R&D. “By targeting the regulation of gene expression on a
cell-by-cell basis, we believe our miRARE technology has the
ability to enable safe expression of MECP2, which may help address
the risks associated with both under and overexpression resulting
from the mosaic pattern of MECP2 silencing. This is a significant
milestone that furthers our quest to bring a potentially
transformational gene therapy to patients and families living with
Rett syndrome. We look forward to sharing initial available
clinical safety data from the Phase 1/2 REVEAL trial at our R&D
Day on June 28, 2023.”
The Phase 1/2 REVEAL trial is a first-in-human, open-label,
randomized, dose-escalation and dose-expansion study evaluating the
safety and preliminary efficacy of TSHA-102 in adult females with
Rett syndrome due to MECP2 loss-of-function mutation. Participants
will receive a single lumbar intrathecal injection of TSHA-102.
Dose escalation will evaluate two dose levels of TSHA-102
sequentially, with an initial dose of 5x1014 total vector genomes
(vg) and the second dose of 1x1015 vg. The maximum tolerated dose
(MTD) or maximum administered dose (MAD) established will then be
administered during dose expansion. Per the protocol, an
independent data monitoring committee will review available safety
data from the first patient at approximately six weeks post-dosing
to determine if the Company can proceed with dosing the second
patient. Initial available clinical safety data will be reported at
Taysha’s upcoming R&D Day on June 28, 2023. To register for the
event, please click here.
Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor
Neuroscience and Pediatrics, and Principal Investigator of the
REVEAL study added, "Based on its unique and compelling technology
targeting the genetic root cause of Rett syndrome, TSHA-102 has the
potential to transform care by addressing a significant unmet
medical need for patients with this devastating and currently
incurable disease. The dosing of the first patient in this
important clinical trial represents a critical advancement in
evaluating the potential of gene therapy for Rett syndrome. It is a
privilege to be part of this important endeavor. In the name of all
affected families, I thank Taysha for bringing this potentially
transformative therapy from the bench to the bedside.”
Sabrina Millson, President of Ontario Rett Syndrome Association
further added, “This is a momentous day for the Rett syndrome
community. As a mom to a daughter living with Rett syndrome and the
president of the Ontario Rett Syndrome Association here in Canada,
I know first-hand how this disease leads to debilitating symptoms,
including difficulties in communication, mobility and breathing.
The potential for a treatment that addresses the underlying cause
of disease and slows progression or potentially prevents the onset
of disease with early intervention is truly remarkable. We’re
pleased to collaborate with Taysha Gene Therapies in an effort to
bring a gene therapy treatment that could meaningfully change the
lives of patients and their caregivers.”
About TSHA-102TSHA-102 is a self-complementary
intrathecally delivered AAV9 investigational gene transfer therapy
in clinical evaluation for Rett syndrome. TSHA-102 utilizes a novel
miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed
to regulate cellular MECP2 expression. TSHA-102 has received Orphan
Drug and Rare Pediatric Disease designations from the United States
(U.S.) Food and Drug Administration (FDA) and has been granted
Orphan Drug designation from the European Commission. We are
advancing TSHA-102 in the REVEAL Phase 1/2 clinical trial under a
CTA approved by Health Canada. A CTA submission to United Kingdom
(UK) MHRA in pediatric patients with Rett syndrome is expected in
mid-2023, and an Investigational New Drug (IND) application to the
FDA is anticipated in the second half of 2023.
About Rett SyndromeRett 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.
About the CHU Sainte-JustineThe
Centre hospitalier universitaire Sainte-Justine is the largest
mother-child hospital in Canada. A member of the Université de
Montréal extended network of excellence in health (RUIS), CHU
Sainte-Justine has 6759 employees, including 1770 nurses and
nursing assistants; 1131 other healthcare professionals;
531 physicians, dentists and pharmacists; 931 residents and over
280 researchers; 170 volunteers; and 3 406 interns and students in
a wide range of disciplines. CHU Sainte-Justine has 484 beds,
including 67 at the Centre de réadaptation Marie Enfant (CRME), the
only exclusively pediatric rehabilitation centre in Québec. The
World Health Organization has recognized CHU Sainte-Justine as a
“health-promoting hospital.” chusj.org
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 of our product candidates, including TSHA-102, 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, 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.
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
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