Seelos Therapeutics announced an update to its Parkinson’s Disease program SLS-007.
Seelos will begin an in-vivo study delivering SLS-007 by an Adeno-Associated Virus (AAV) vector making the program Seelos’ second gene therapy approach for Parkinson’s Disease (PD). Seelos is currently producing the viral vectors and preparing animals for this study. Data to determine delivery and target engagement is expected in the second half of this year.
“Identifying the AAV vector as the optimal route of systemic administration of SLS-007 may ultimately yield a convenient one-time delivery of the peptides,” said Raj Mehra, Ph.D., Chairman and CEO of Seelos. “This is a novel method of viral delivery that has been a collaboration between our R&D team and key opinion leaders that, if successful, could be a major advancement in the field.”
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“The study will include measurements of several key biomarkers that will assess the extent of alpha-synuclein (α-synuclein) aggregates expression in key target areas of the brain, such as the forebrain and the substantia nigra. These outcomes will help determine key target engagement and set the stage for our next steps with the program,” added Tim Whitaker, MD, Head of R&D at Seelos.
Seelos is also developing SLS-004, which is an all-in-one lentiviral vector gene therapy program that targets the SNCA gene that encodes for the production of α-synuclein.
SLS-007 is a family of rationally designed peptidic inhibitors that target the NACore (non-amyloid component core) of α-synuclein to inhibit protein aggregation in patients with PD. The overexpression of α-synuclein leads to the formation of α-synuclein aggregates which comprise Lewy bodies and neurites which are the hallmarks of the pathogenesis of PD. Recent in-vitro and cell culture research have shown that SLS-007 has the potential to stop the propagation and seeding of α-synuclein aggregates. The goal of the in-vivo animal study will be to establish pharmacokinetic (PK) and pharmacodynamic (PD) profiles and target engagement parameters of SLS-007.