SLS-004 / Seelos Therap - LARVOL DELTA

One Year In Vivo Safety After CRISPR Based Parkinson's Disease Treatment (ASGCT 2024) - "The current abstract shows one-year in-vivo safety when administered in normal animals implying safe use of this CRIPSR based epigenetic down-regulation of alpha-synuclein in-vivo for long-term administration in disease modifying therapies.SLS-004 can be personalized to down-regulate the genetic expression by 30-50% based on individual over-expression phenotype and disease severity. SLS-004 can recover biological phenotype of dopamine neurons and reduce over expression back to physiological normal levels of protein in several synucleinopathies including in PD.This one year safety data in vivo with CRISPR provides novel validation of the safety of this epigenetic therapy for synucleinopathies"

Preclinical • Alzheimer's Disease • Behavior Disorders • CNS Disorders • Cognitive Disorders • Dementia • Gene Therapies • Lewy Body Disease • Movement Disorders • Parkinson's Disease • Psychiatry

Seelos Therapeutics to Present a Poster on SLS-004 at the 11th Annual Alzheimer's & Parkinson's Drug Development Summit (PRNewswire) - "Seelos Therapeutics, Inc...today announced that it has been selected to present a poster on SLS-004 at the 11th Annual Alzheimer's & Parkinson's Drug Development Summit in San Francisco, CA, February 21-23, 2023."

Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Announces Data Demonstrating Downregulation of Alpha Synuclein in an In Vivo Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Parkinson's Disease (PRNewswire) - "Seelos Therapeutics, Inc...announced in vivo data demonstrating that a single dose of SLS-004 downregulated the production of alpha-synuclein (α-synuclein). This reduction of α-synuclein by SLS-004 in an established α-synuclein overexpressing animal model of Parkinson's disease (PD) resulted in substantial increase and recovery of degeneration in tyrosine hydroxylase positive (TH+) dopaminergic neurons....The preliminary findings indicate that a single dose of SLS-004 administered in the SNpc of the test hemisphere in the brain produced a substantial increase in and recovery of degenerating TH+ dopaminergic neurons compared to the administration of the control vector in the SNpc of the other hemisphere. The nigrostriatal degeneration of TH+ midbrain dopaminergic neurons in the SNpc is implicated in PD pathology and attributed to the cardinal signs of parkinsonian symptoms."

Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Releases the Itinerary for its Research and Development Update Conference Call and Webcast to be Held on December 15, 2022 (PRNewswire) - “Seelos Therapeutics, Inc…released the itinerary for its research and development update conference call and webcast to be held on Thursday, December 15th from 1pm to 3pm ET….SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion): Raj Mehra, Ph.D. will provide an update of the ongoing registration directed studies of SLS-005 in amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia (SCA). Jeffrey H. Kordower, Ph.D…will discuss recent developments Seelos has made with SLS-005 in Huntington's disease and Alzheimer's disease. SLS-004 and SLS-007 (gene therapy approaches for Parkinson's disease): Krishna Subramanian, Ph.D…will discuss recent data and Raj Mehra, Ph.D. will discuss next steps for the programs.”

Clinical • Clinical data • Trial status • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • Ataxia • CNS Disorders • Huntington's Disease • Parkinson's Disease

Seelos Therapeutics Receives a Research and Development Grant from The Michael J. Fox Foundation for Parkinson's Research for SLS-004 (PRNewswire) - "Seelos Therapeutics...today announced it was selected to receive a grant from The Michael J. Fox Foundation for Parkinson's Research to advance preclinical research and development of its gene therapy delivered SLS-004 program.'The selection of SLS-004 to receive grant funding from The Michael J. Fox Foundation is a strong validation of our program and should significantly raise the profile of our program,' said Raj Mehra, Ph.D., Chairman and CEO of Seelos. 'We look forward to sharing additional data from our ongoing preclinical studies later this year.'"

Financing • Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Announces Data Demonstrating Statistically Significant Downregulation of mRNA and Reduction of Alpha Synuclein in an In Vitro Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 in Dementia with Lewy Bodies (PRNewswire) - "Seelos Therapeutics, Inc...announced data demonstrating a statistically significant (p<0.01) 19% downregulation of mRNA and a ~40% reduction of alpha synuclein (α-synuclein) in an in vitro study of SLS-004, its gene therapy program utilizing CRISPR-dCas9, in dementia with Lewy bodies (DLB)....The preliminary findings showed that following two weeks of differentiation into cholinergic neurons, there was a statistically significant (p<0.01) 19% downregulation of mRNA and a ~40% reduction of α-synuclein protein compared to the no treatment/repressor groups....Seelos released positive preclinical in vivo data with SLS-004 in downregulation of overexpressed α-synuclein in a Parkinson's disease model and plans to release additional data in the second half of 2022."

Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Announces Mid-Year 2021 Business and Clinical Update (PRNewswire) - "On June 15th, Seelos completed a Type C meeting with the FDA to discuss the regulatory pathway for the SLS-002 program. SLS-005 (IV trehalose): In the third quarter, Seelos is expected to commence dosing of a pivotal Phase IIb/III trial in amyotrophic lateral sclerosis (ALS) as part of the HEALEY ALS Platform Trial led by Harvard Medical School at Massachusetts General Hospital at several dozen trial sites across the U.S....Additional studies have commenced to explore efficacy of SLS-004 in the induced Parkinsonism in an in vivo rodent model."

FDA event • New P2/3 trial • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Depression • Parkinson's Disease

Seelos Therapeutics to Participate in Two Investor Conferences in August (PRNewswire) - “Seelos Therapeutics…will participate in two investor conferences: the BTIG Virtual Biotechnology Conference on August 9th and 10th and the Canaccord Growth Conference on August 11th and 12th. Raj Mehra, Ph.D., Chairman and CEO, will present and host one on one meetings to provide a corporate update and overview of the company's programs, specifically Part 2 of the recently initiated registrational study of SLS-002 (intranasal racemic ketamine) in Adults with Major Depressive Disorder at imminent risk of suicide [NCT04669665], the recently released in vivo data utilizing CRISPR-dCas9 gene therapy technology where a single dose of SLS-004 produced therapeutically desirable reductions in SNCA mRNA and SNCA protein expressions...in Parkinson's disease, and the upcoming initiation of the SLS-005 Phase IIb/III pivotal study in amyotrophic lateral sclerosis (ALS) in the Healey ALS Platform Trial.”

Preclinical • Review • Amyotrophic Lateral Sclerosis • CNS Disorders • Depression • Parkinson's Disease

Seelos Therapeutics Announces Positive In Vivo Data Demonstrating Down-Regulation of SNCA mRNA and Protein Expression from a Gene Therapy Study of SLS-004 Utilizing CRISPR-dCas9 for Parkinson's Disease Pathology (PRNewswire) - "Seelos Therapeutics...announced positive in vivo data demonstrating down-regulation of SNCA mRNA and protein-expression from a study of SLS-004 in an in-vivo rodent model utilizing CRISPR-dCas9 gene therapy technology. A single dose of SLS-004 produced a therapeutically desirable 27% reduction on SNCA mRNA and a 40% reduction in SNCA protein expression....'We are highly encouraged by these preliminary findings demonstrating downregulation of SNCA mRNA and SNCA protein expression in this in vivo model,'..."

Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Announces Year End 2020 Business Update (PRNewswire) - “Seelos Therapeutics…announced its year end 2020 business update….SLS-005 (IV trehalose): Pivotal Phase IIb/III trial in ALS to begin in the first half of 2021 in the HEALEY ALS Platform Trial...(Parkinson's disease gene therapy programs)...SLS-004 Preclinical data expected mid-2021."

New P2/3 trial • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Parkinson's Disease

Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson's Disease (PRNewswire) - "Seelos Therapeutics, Inc....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....'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.'"

Licensing / partnership • Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics announces initiation of preclinical study of SLS-004 in Parkinson’s disease (GlobeNewswire) - "Seelos Therapeutics, Inc....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....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."

Preclinical • CNS Disorders • Parkinson's Disease

Seelos Therapeutics acquires rights for Parkinson’s Gene Therapy program (Parkinson's News Today) - "'Aggregation of misfolded alpha-synuclein appears to be a key pathogenic mechanism leading to neuronal dysfunction and death. Inhibition of synuclein production, such as through SLS-004, is an attractive therapeutic target that may reduce aggregation and slow disease progression,' Robert A. Hauser..."

Media quote