Parkinson's Study Using CRISPR Technology Shows Promise, Seelos Therapeutics Says

  • Seelos Therapeutics Inc SEEL 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 a substantial increase and recovery of degeneration in tyrosine hydroxylase positive (TH+) dopaminergic neurons. 
  • TH+ dopaminergic neurons in the midbrain region, called substantia nigra pars compacta (SNpc), are known to degenerate in patients with PD. 
  • This degeneration is attributed to the cardinal Parkinsonian symptoms of tremor, rigidity, bradykinesia, and postural disturbances.
  • The preliminary findings indicate that a single dose of SLS-004 administered in the brain's SNpc test hemisphere produced a substantial increase in and recovery of degenerating TH+ dopaminergic neurons compared to the administration of the control vector.
  • In July 2021, Seelos announced positive in vivo data demonstrating the down-regulation of SNCA mRNA and protein expression from a study of SLS-004. A single dose of SLS-004 produced a therapeutically desirable 27% reduction in SNCA mRNA and a 40% reduction in SNCA protein expression.
  • Also, in June 2022, Seelos released statistically significant data in an in vitro gene therapy study of SLS-004 utilizing CRISPR-dCas9 in dementia with Lewy bodies.
  • Price Action: SEEL shares are down 6.98% at $1.20 on the last check Thursday.
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