Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021
October 04 2021 - 7:30AM
Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company
focused on developing gene therapies to radically transform the
lives of patients with neurodegenerative diseases, today announced
that it will present new clinical and preclinical data in two oral
presentations and one poster presentation at the upcoming European
Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021,
to be held virtually from October 19-22, 2021.
Oral presentations will include an update on the
Phase 1/2 trial of AXO-AAV-GM1, the company’s adeno-associated
viral vector (AAV)9-based gene therapy for the treatment of Type I
(early infantile onset) and Type II (late infantile and juvenile
onset) GM1 gangliosidosis. Presentation will include new data from
the low- and high-dose cohorts. The Company will also present a
poster review of patient-level data up to 24 months from the Phase
1/2 study of AXO-Lenti-PD gene therapy for the treatment of
Parkinson’s disease.
Oral Presentation Details:
Presentation Title: Phase 1/2 Trial of
AXO-AAV-GM1 Gene Therapy for the Treatment of Infantile- and
Juvenile-onset GM1 GangliosidosisPresentation
Number: OR28Session: Session 4a: CNS
& Sensory IIPresenting Author: Erica De
Boever, Ph.D., DDS, MPH, Vice President of Clinical Development at
Sio Gene TherapiesPresentation Date and Time:
Thursday October 21, 2021; 9:00-11:00 AM CEST
Presentation Title: Bicistronic AAV Gene
Therapy for Tay-Sachs and Sandhoff Diseases in a Sheep
ModelPresentation Number:
OR30Session: Session 4a: CNS & Sensory
IIPresenting Author: Toloo Taghian, Ph.D.,
University of MassachusettsPresentation Date and
Time: Thursday, October 21, 2021; 9:00-11:00 AM CEST
Poster Presentation Details:
Presentation Title: Phase 1/2 Open-label Dose
Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinson’s
Disease: Efficacy, Safety, and Tolerability Data up to 24
MonthsPoster Number: P254Presenting
Author: Gavin Corcoran, MD, Chief R&D Officer of Sio
Gene Therapies
Copies of the presentation materials will be made available
under the Events and Presentations section of Sio’s website.
About AXO-AAV-GM1AXO-AAV-GM1 delivers a
functional copy of the GLB1 gene via an adeno-associated viral
(AAV) vector, with the goal of restoring β-galactosidase enzyme
activity for the treatment of GM1 gangliosidosis. The gene therapy
is delivered intravenously, which has the potential to broadly
transduce the central nervous system and treat peripheral
manifestations of the disease as well. Preclinical studies in
murine and a naturally-occurring feline model of GM1 gangliosidosis
have supported AXO-AAV-GM1’s ability to improve β-galactosidase
enzyme activity, reduce GM1 ganglioside accumulation, improve
neuromuscular function, and extend survival.
AXO-AAV-GM1 has received both Orphan Drug Designation and Rare
Pediatric Disease Designation from the Food and Drug Administration
and is the only gene therapy in clinical development for all
pediatric forms of GM1 gangliosidosis. In 2018, Sio licensed
exclusive worldwide rights from the University of Massachusetts
Medical School for the development and commercialization of gene
therapy programs for GM1 gangliosidosis and GM2 gangliosidosis,
including Tay-Sachs and Sandhoff diseases.
About AXO-AAV-GM2AXO-AAV-GM2 is an
investigational gene therapy for GM2 gangliosidosis (also known as
Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric
neurodegenerative genetic disorders caused by defects in the HEXA
(leading to Tay-Sachs disease) or HEXB (leading to Sandhoff
disease) genes that encode the two subunits of the β-hexosaminidase
A (HexA) enzyme. These genetic defects lead to progressive
neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims
to restore HexA function by introducing a functional copy of the
HEXA and HEXB genes via delivery of two co-administered AAVrh8
vectors.
About AXO-Lenti-PDAXO-Lenti-PD is an
investigational gene therapy for the treatment of Parkinson’s
disease that is designed to deliver three genes (tyrosine
hydroxylase, cyclohydrolase 1, and aromatic L-amino acid
decarboxylase) via a single lentiviral vector to encode a set of
critical enzymes required for dopamine synthesis, with the goal of
reducing variability and restoring steady levels of dopamine in the
brain. The investigational gene therapy aims to provide patient
benefit for years following a single administration. Axovant
expects to dose the first patient in EXPLORE-PD, a randomized, sham
controlled study in 2021.
About Sio Gene TherapiesSio Gene Therapies
combines cutting-edge science with bold imagination to develop
genetic medicines that aim to radically improve the lives of
patients. Our current pipeline of clinical-stage candidates
includes the first potentially curative AAV-based gene therapies
for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are
rare and uniformly fatal pediatric conditions caused by single gene
deficiencies. We are also expanding the reach of gene therapy to
highly prevalent conditions such as Parkinson’s disease, which
affects millions of patients globally. Led by an experienced team
of gene therapy development experts, and supported by
collaborations with premier academic, industry and patient advocacy
organizations, Sio is focused on accelerating its candidates
through clinical trials to liberate patients with debilitating
diseases through the transformational power of gene therapies. For
more information, visit www.siogtx.com.
Forward-Looking Statements
This press release contains forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
The use of words such as "expect," "estimate," "may" and other
similar expressions are intended to identify forward-looking
statements. For example, all statements Sio makes regarding costs
associated with its operating activities, funding requirements
and/or runway to meet its upcoming clinical milestones, and timing
and outcome of its upcoming clinical and manufacturing milestones
are forward-looking. All forward-looking statements are based on
estimates and assumptions by Sio’s management that, although Sio
believes to be reasonable, are inherently uncertain. All
forward-looking statements are subject to risks and uncertainties
that may cause actual results to differ materially from those that
Sio expected. Such risks and uncertainties include, among others,
the impact of the Covid-19 pandemic on our operations; the actual
funds and/or runway required for our clinical and product
development activities and anticipated upcoming milestones; actual
costs related to our clinical and product development activities
and our need to access additional capital resources prior to
achieving any upcoming milestones; the initiation and conduct of
preclinical studies and clinical trials; the availability of data
from clinical trials; the development of a suspension-based
manufacturing process for AXO-Lenti-PD; the scaling up of
manufacturing; the expectations for regulatory submissions and
approvals; the continued development of our gene therapy product
candidates and platforms; Sio’s scientific approach and general
development progress; and the availability or commercial potential
of Sio’s product candidates. These statements are also subject to a
number of material risks and uncertainties that are described in
Sio’s most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on August 12, 2021, as updated
by its subsequent filings with the Securities and Exchange
Commission. Any forward-looking statement speaks only as of the
date on which it was made. Sio undertakes no obligation to publicly
update or revise any forward-looking statement, whether as a result
of new information, future events or otherwise, except as required
by law.
Contacts:
Media Josephine Belluardo, Ph.D. LifeSci
Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com
Investors and AnalystsParag V. Meswani,
Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officer
investors@siogtx.com
Sio Gene Therapies (NASDAQ:SIOX)
Historical Stock Chart
From Jun 2024 to Jul 2024
Sio Gene Therapies (NASDAQ:SIOX)
Historical Stock Chart
From Jul 2023 to Jul 2024