Sio Gene Therapies to Present at the 18th Annual WORLDSymposium™ 2022
February 02 2022 - 7:00AM
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 data in an oral platform
presentation and two poster presentations at the 18th Annual
WORLDSymposium™ 2022, to be held February 7-11, 2022.
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. A comprehensive data update is still expected
in the first half of this year. The Company is also collaborating
with Invitae, who will present a poster on the Detect LSD
program.
Oral Platform Presentation Details:
Presentation Title: Phase 1/2 trial of
AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and
juvenile-onset GM1
gangliosidosisSession: Platform
Presentations, Clinical ResearchPresenting
Author: Cynthia Tifft, MD, PhD, National Human Genome
Research Institute, National Institutes of Health
(NIH)Presentation Date and Time: Wednesday,
February 9, 2022; 1:00 PM PST
Poster Presentation Details:
Presentation Title: Phase 1/2 trial of
AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and
juvenile-onset GM1 gangliosidosisPoster Number:
302Presenting Author: Cynthia Tifft, MD, PhD,
National Human Genome Research Institute, National Institutes of
Health (NIH)Presentation Date and Time: Wednesday,
February 9, 2022; 3:00-5:00 PM PST
Presentation Title: Detect Lysosomal
Storage Diseases: A no-charge, sponsored, testing program that
enables access to genetic testing, treatment, and clinical trials
for individuals with suspected lysosomal disordersPoster
Number: 194Presenting
Author: Heather McLaughlin, PhD, FACMG,
InvitaePresentation Date and Time: Thursday,
February 10, 2022; 3:00-5:00 PM PST
Presentations can be accessed from the WORLDSymposium™ website
at www.worldsymposia.org.
About AXO-AAV-GM1
AXO-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 achieve a broad central
and peripheral biodistribution. Preclinical studies in murine and a
naturally-occurring feline model of GM1 gangliosidosis have
supported AXO-AAV-GM1’s ability to increase β-galactosidase enzyme
activity, reduce GM1 ganglioside accumulation, improve
neuromuscular function, and extend survival.
AXO-AAV-GM1 has received Orphan Drug Designation, Rare Pediatric
Disease Designation, and Fast Track Designation from
the FDA and is the only gene therapy in clinical
development for all pediatric forms of GM1 gangliosidosis.
In 2018, Sio licensed exclusive worldwide rights from UMass Chan
Medical School for the development and commercialization of
gene therapy programs for GM1 gangliosidosis and GM2
gangliosidosis, including Tay-Sachs and Sandhoff diseases.
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 is comprised of 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. 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 StatementsThis 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 “believe,” “expect,” “intend,” “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, expected
cash burn runway, expectations regarding licensing and commercial
agreements, 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 Sio’s operations; the actual funds and/or runway
required for Sio’s clinical and product development activities and
anticipated upcoming milestones; actual costs related to Sio’s
clinical and product development activities and Sio’s 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
occurrence of adverse safety events during our current and future
trials; the scaling up of manufacturing; the outcome of
interactions with regulatory agencies and 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 November
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:
MediaJosephine 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
Officerinvestors@siogtx.com
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