UPDATE: Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021
October 04 2021 - 9:04AM
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.
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
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