Seelos Therapeutics Announces Initiation of Preclinical Study of SLS-004 in Parkinson’s Disease
May 28 2020 - 7:01AM
Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage
biopharmaceutical company focused on the development of therapies
for central nervous system disorders and rare diseases, today
announced the initiation of a preclinical study of SLS-004 in
Parkinson’s disease (PD) through an all-in-one lentiviral vector
targeting the synuclein alpha (SNCA) gene. Seelos is constructing a
bimodular viral system harboring an endogenous alpha-synuclein
(α-synuclein) transgene and inducible regulated repressive
CRISPR/Cas9-unit to achieve constitutive activation and inducible
suppression of PD-related pathologies. “There has been a high level
of interest in the alpha-synuclein approach to Parkinson's and
beginning further work on our first gene therapy program
is exciting,” said Raj Mehra, Ph.D., Chairman and CEO of Seelos.
“Initiating this preclinical study earlier than expected
is also very significant." Previously, it was shown that
enrichment in DNA methylation at intron 1 of the α-synuclein gene
SNCA, through SLS-004, facilitated robust and precise repression of
SNCA expression, which coincides with rescuing of PD-phenotypes.
The SNCA gene, which encodes α-synuclein expression, has been
implicated as a highly significant risk factor for PD. Accumulative
evidence suggests that elevated levels of wild-type α-synuclein are
causative in the pathogenesis of PD. The role of SNCA
overexpression in PD pathogenesis and the need to maintain normal
physiological α-synuclein protein levels emphasize the so far unmet
need to develop new therapeutic strategies, such as SLS-004,
targeting the regulatory mechanisms of SNCA expression.
About Alpha-synuclein (α-synuclein)
Alpha-synuclein (α-synuclein) is a protein which is of great
interest to Parkinson's researchers because it is a major
constituent of Lewy bodies and Lewy neurites, protein clumps that
are the pathological hallmark of synucleinopathies, such as
Parkinson's disease, dementia with Lewy bodies (DLB) and multiple
system atrophy (MSA). In the several years since its discovery,
alpha-synuclein has been the focus of intensive efforts by PD
researchers working to definitively characterize the protein's role
in Parkinson's and its potential as a target for neuroprotective
therapies. In 2017, The Michael J. Fox Foundation convened leaders
from academia and industry to form the Alpha-Synuclein Clinical
Path Working Group. The group's goal was to lay out a roadmap to
advance drugs targeting alpha-synuclein proteins and increase their
odds of success. For more information, please visit:
https://www.michaeljfox.org/news/roadmap-alpha-synuclein
Forward Looking Statements Statements made in this
press release, which are not historical in nature, constitute
forward-looking statements for purposes of the safe harbor provided
by the Private Securities Litigation Reform Act of 1995. These
statements include, among others, those regarding the initiation
and completion of the preclinical study of SLS-004, expectations
regarding the efficacy of SLS-004 and a bimodular viral system to
suppress PD-related pathologies, the role of SNCA gene in PD
pathogenes is the ability of SLS-004 to target the regulatory
mechanisms of SNCA expression These statements are based on Seelos'
current expectations and beliefs and are subject to a number of
factors and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements.
Risks associated to Seelos' business include, but are not limited
to, the risk of not successfully executing its preclinical and
clinical studies and not gaining marketing approvals for its
product candidates, the risks associated with the implementation of
a new business strategy, the risks related to raising capital to
fund its development plans and ongoing operations, risks related to
Seelos’ current stock price, risks related to the global impact of
COVID-19, as well as other factors expressed in Seelos' periodic
filings with the U.S. Securities and Exchange Commission, including
its Annual Report on Form 10-K and Quarterly Reports on Form
10-Q. Although we believe that the expectations reflected in
our forward-looking statements are reasonable, we do not know
whether our expectations will prove correct. You are cautioned not
to place undue reliance on these forward-looking statements, which
speak only as of the date hereof, even if subsequently made
available by us on our website or otherwise. We do not undertake
any obligation to update, amend or clarify these forward-looking
statements, whether as a result of new information, future events
or otherwise, except as may be required under applicable securities
laws. Contact Information: Anthony Marciano Head of Corporate
Communications Seelos Therapeutics, Inc. (Nasdaq: SEEL) 300 Park
Ave., 12th Fl New York, NY 10022 (646) 293-2136
anthony.marciano@seelostx.comhttps://seelostherapeutics.com/https://twitter.com/seelostxhttps://www.linkedin.com/company/seelos
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