Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and ...
April 28 2021 - 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
four upcoming oral presentations at the 24th Annual Meeting of the
American Society of Gene & Cell Therapy (ASGCT), to be held
virtually between May 11th to May 14th, 2021.
The AXO-AAV-GM1 presentation will include a review of
patient-level data on safety and efficacy at 6 months follow up
from the low-dose cohort of the Company’s ongoing clinical study.
Additionally, Dr. Cynthia Tifft, the lead investigator for the
study, will present 6-month biomarker data from cerebrospinal fluid
(CSF) in the 5 children who received intravenous AAV9 gene
therapy.
Oral Presentation Details:
Presentation Title: AXO-AAV-GM1 for the
Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase
I-II trialAbstract Number:
162Session: Clinical Trials and Advanced
Preclinical Studies for Neurologic DiseasesPresenting
Author: Cynthia Tifft, MD, PhD, Deputy Clinical Director,
National Human Genome Research InstitutePresentation Date
and Time: Thursday, May 13, 2021 6:15 PM – 6:30 PM EDT
Presentation Title: AXO-Lenti-PD gene therapy
for Parkinson’s disease: efficacy, safety, and tolerability data
from the second cohort in open-label dose evaluation study
SUNRISE-PD at 6 months post administrationAbstract
Number: 163Session: Clinical Trials and
Advanced Preclinical Studies for Neurologic
DiseasesPresenting Author: Gavin Corcoran, MD,
Chief R&D Officer Presentation Date and Time:
Thursday, May 13, 2021 6:30 PM – 6:45 PM EDT
Presentation Title: Immune Modulation Preceding
AAV9-GLB1 Gene Therapy Preserves the Possibility for Re-Dosing in
Children with GM1 GangliosidosisAbstract Number:
179Session: Immunotherapy and
VaccinesPresenting Author: Precilla D’Souza, DNP,
MSN, CRNP, National Human Genome Research
InstitutePresentation Date and Time: Thursday, May
13, 2021 7:00 PM – 7:15 PM EDT
Presentation Title: A GLP Safety and
Biodistribution Study of AXO-Lenti-PD Manufactured via Two
Processes Delivered at a Higher Volume and Flow
RateAbstract Number: 256Session:
Pharmacology/Toxicology Studies or Assay
DevelopmentPresenting Author: Thomas Pack, PhD,
Sio Gene TherapiesPresentation Date and Time:
Friday, May 14, 2021 from 1:45 PM – 2:00 PM EDT
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 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 both Type I and Type II 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-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 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,” "estimate," “may be” 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 of its
upcoming clinical 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 February 9, 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 Analysts
Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief
Commercial Officer investors@siogtx.com
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