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nL-PACS1-001
- Development stage
- Phase 2
- Lead developer
- n-Lorem Foundation
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
nL-PACS1-001 is a personalized antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of Schuurs-Hoeijmakers syndrome (SHMS). SHMS is a rare neurodevelopmental disorder caused by a specific de novo, heterozygous missense gain-of-function mutation (typically c.607C>T; p.Arg203Trp) in the *PACS1* gene. The ASO is designed to target and modulate the expression of the mutant *PACS1* mRNA to mitigate the toxic gain-of-function effect. It is currently being evaluated in a single-participant (N-of-1) Phase 1/2 clinical trial to assess safety and efficacy.
- Other names
- nL-PACS1-001-n-Lorem Foundation-Schuurs-Hoeijmakers syndromenL-PACS-1-001-n-Lorem Foundation-Schuurs-Hoeijmakers syndromenL-PACS 1-001-n-Lorem Foundation-Schuurs-Hoeijmakers syndrome
02
Targets
PACS1 (Phosphofurin acidic cluster sorting protein 1)
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