NEW YORK, Sept. 23, 2020 /PRNewswire/ -- Seelos
Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage
biopharmaceutical company focused on the development of therapies
for central nervous system disorders and rare diseases, announced
today the signing of a Sponsored Research Agreement (SRA) with
Duke University to use the MPTP-induced
Parkinson's Disease (PD) mouse model to establish in vivo
proof-of-concept study to demonstrate that administration of
LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the
efficacy and safety of SLS-004. Subsequently, other pre-clinical
models would be utilized to further validate the investigational
product.
"We are extremely pleased to begin the in vivo target engagement
study with SLS-004 at Duke as it builds
onto the work we began in the spring focused on designing a vector
capable of inducing and suppressing Parkinson's related
phenotypes," said Raj Mehra Ph.D., Chairman and CEO of Seelos.
"Initiating this next portion of the studies at Duke should help us further validate this approach
in Parkinson's."
Seelos has also begun work on designing a vector capable of
inducing and suppressing PD-related phenotypes by carrying a unit
to overexpress the alpha-synuclein (α-synuclein) protein and a unit
to mediate inducible suppression of α-synuclein. The process aims
to create a next-generation suppressive unit, carrying a more
effective effector molecule than previously used, in the form of
DNA methyltransferase 3A and 3L (DNMT3A & L). This research may
help to advance a potential novel, effective and precise tool for
reversing SNCA pathologies that can provide a valuable new
therapeutic strategy for treating PD.
About SLS-004
SLS-004 is a novel epigenome-editing approach to modulate
expression of SNCA gene mediated by modification of
DNA-methylation. SLS-004 utilizes an all-in-one lentiviral vector
harboring dCas9-DNA methyltransferase 3A (DNMT3A) to enrich
DNA-methylation within CpGs island at the SNCA intron 1 region. The
system resulted in a precise and fine-tuned downregulation (30%) of
SNCA overexpression in hiPSC-derived dopaminergic neurons from a PD
patient with the triplication of the SNCA locus (SNCA-Tri). Most
importantly, the reduction of SNCA expression mediated by the
developed system was sufficient to ameliorate disease related
cellular phenotypes. The in vitro studies achieved several key
millstones including the establishment that DNA hypermethylation at
SNCA intron 1 allows an effective and sufficient tight
downregulation of SNCA expression levels and suggests the potential
of this target sequence combined with the CRISPR-dCas9 technology
as a novel epigenetic-based therapeutic approach for PD.
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 of a proof-of-concept study
to demonstrate whether administration of the LV-dCas9-DNMT3A virus
can prevent and/or delay PD, the potential for the LV-dCas9-DNMT3A
virus to prevent and/or delay PD, the efficacy and safety of
SLS-004, the potential use of other pre-clinical models to validate
SLS-004, the potential for the in vivo proof-of-concept study to
validate the design of a vector capable of inducing and suppressing
PD-related phenotypes, and the potential for Seelos' research to
advance a novel, effective and precise tool for reversing SNCA
pathologies. 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 with 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 risk that prior test results may not be replicated
in future studies and trials, the risks that clinical study results
may not meet any or all endpoints of a clinical study and that any
data generated from such studies may not support a regulatory
submission or approval, 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.com
www.seelostherapeutics.com
https://twitter.com/seelostx
https://www.linkedin.com/company/seelos
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