Sio Gene Therapies Announces Successful Manufacture of Three GMP Batches of AXO-Lenti-PD Gene Therapy for Parkinson’s Disease
November 11 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 provided a
manufacturing and regulatory update for AXO-Lenti-PD, its
clinical-stage gene therapy for Parkinson’s disease.
“Today’s announcement is the culmination of several months of
effort by the Sio and Oxford BioMedica teams focused on the
development of a reliable, suspension-based manufacturing process
to enable scale-up of production and advancement of the
AXO-Lenti-PD program,” said Pavan Cheruvu, M.D., Chief Executive
Officer of Sio Gene Therapies. “With the successful manufacture of
three batches, all of which achieved the target titer and have
completed fill and finish, we now have a process that has generated
sufficient clinical trial material for future clinical development.
We believe these data, coupled with scientific advice from the
MHRA, help clarify the path forward and collectively represent an
inflection point for the AXO-Lenti-PD program.”
Key Highlights
- Three GMP batches have successfully completed fill and finish,
achieving target titers using the updated suspension-based
process
- Company is on-track to complete final testing of these three
batches to support certification of at least one batch for use as
clinical trial material by a Qualified Person in Q4 2021
- Additionally, the Company successfully completed a scientific
advice meeting with the Medicines and Healthcare Products
Regulatory Agency (MHRA) in the U.K. regarding the AXO-Lenti-PD
clinical development program. The MHRA provided guidance on the:
- Appropriate development pathway for completion of the Phase 1
dose-ranging study
- Acceptability of a comparability protocol between the prior
adherent and new suspension manufacturing process
- New device administration system to support bilateral
simultaneous infusions
- Expect to provide a program update in Q1 2022
About Sio Gene Therapies
Sio 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 “believe,” "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 occurrence of adverse safety events
during our current and future trials; the development of a
suspension-based manufacturing process for AXO-Lenti-PD; 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 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 Analysts
David W. NassifSio Gene Therapies Inc.Chief Financial Officer
and General Counseldavid.nassif@siogtx.com
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