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- Seelos recently released in vivo gene therapy data of SLS-004 utilizing CRISPR-dCas9 in Parkinson's disease demonstrating downregulation of alpha synuclein and recovery of tyrosine hydroxylase-positive (TH+) dopaminergic neurons.
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 that it has been selected to present a poster on SLS-004 at the 11 th Annual Alzheimer's & Parkinson's Drug Development Summit in San Francisco, CA February 21-23, 2023 .
Seelos will present the following Poster on February 22 nd at 5:00 pm PT :
Title : SLS-004 - CRISPR-based epigenetic alpha-synuclein downregulation recovers loss of midbrain dopamine neurons in a humanized A53T Parkinson's rodent model
Co-Authors : Krishna Subramanian, Ph.D., Jeffrey H. Kordower , Ph.D., Yaping Chu , Ph.D., David Biondi , DO, FAAN, Boris Kantor, Ph. D, Ornit Chiba-Falek, Ph.D., Raj Mehra, Ph.D.
Presenter :Â Krishna Subramanian, Ph.D.
For additional information: https://alzheimers-parkinsons-summit.com/
About SLS-004
SLS-004 is an investigational novel epigenome-editing approach to modulate expression of alpha-synuclein (α-synuclein) protein produced by SNCA gene. This approach is mediated by hypermethylation of the SNCA genetic code that has demonstrated in preclinical models a controlled and regulated decrease in α-synuclein production. SLS-004 utilizes an all-in-one lentiviral vector harboring dCas9 and an effector to enrich DNA-methylation within CpGs (cytosine-phosphate-guanine site) 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 Parkinson's disease (PD) related cellular phenotypes in these in-vitro cellular models. The in vitro studies achieved several key milestones, including the establishment that DNA hyper-methylation at SNCA intron 1 allows for an effective and sufficiently tight downregulation of SNCA expression levels. These data suggest the potential of SLS-004, a CRISPR-dCas9 technology, as a future novel epigenetic-based therapeutic approach for PD.
About Seelos Therapeutics
Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system (CNS) disorders and other rare diseases. The Company's robust portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder (MDD), amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia (SCA), as well as early-stage programs in Huntington's disease, Alzheimer's disease, and Parkinson's disease.
For more information, please visit our website: https://seelostherapeutics.com , the content of which is not incorporated herein by reference.
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 safety and efficacy of SLS-004 and its ability to downregulate or reduce SNCA mRNA and SNCA protein expression and its potential for a disease-modifying gene therapy in PD. These statements are based on Seelos' current expectations and beliefs and are subject to a number of risks 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 clinical results may not be replicated in future studies and trials (including the risk that the results from the preclinical study of SLS-004 are not replicated or are materially different from the results of 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 Seelos' 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 most recent 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
Chief Communications Officer
Seelos Therapeutics, Inc. (Nasdaq: SEEL)
300 Park Avenue, 2 nd Floor
New York, NY 10022
(646) 293-2136
anthony.marciano@seelostx.com
https://seelostherapeutics.com/
https://x.com/seelostx
https://www.linkedin.com/company/seelos
Mike Moyer
Managing Director
LifeSci Advisors, LLC
250 West 55th St., Suite 3401
New York, NY 10019
(617) 308-4306
mmoyer@lifesciadvisors.com
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SOURCE Seelos Therapeutics, Inc.
