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Find Clinical Drug Pipeline Developments & Deals by Wave Life Sciences
The net proceeds from this offering will be used to advance RNA editing pipeline of programs, including WVE-006 and to accelerate the development of Wave's INHBE program into the clinical.
The net proceeds from the offering will be used to advance RNA editing pipeline of programs, including WVE-006 and to accelerate the development of Wave's INHBE program into the clinical.
WVE-004 is an ASO designed with Wave’s proprietary and best-in-class chemistry, which selectively targets transcriptional variants containing the hexanucleotide repeat expansion (G4C2) associated with the C9orf72 gene, thereby sparing normal C9orf72 protein.
High muscle concentrations of WVE-N531 and exon skipping were observed six weeks after initiating biweekly multidosing at 10 mg/kg, achieving proof-of-concept in the study. WVE-N531 also appeared safe and well-tolerated.
The collaboration includes 2 components, first is a discovery collaboration which enables GSK to advance upto 8 programmes and Wave upto 3 programmes, using Wave’s PRISM platform in genetics and genomics. GSK will in-license Wave’s preclinical programme WVE-006 for AATD.
WVE-004 is stereopure antisense oligonucleotide designed to selectively target transcriptional variants containing a hexanucleotide repeat expansion associated with C9orf72 gene, thereby sparing C9orf72 protein.
WVE-003 is working as intended, to selectively reduce the toxic mHTT protein while avoiding targeting the healthy, wild-type huntingtin protein, thereby preserving its beneficial effects in the central nervous system.
Preclinical data published in Molecular Therapy Nucleic Acids demonstrate that WVE-004 potently reduces C9orf72 transcriptional variants and poly(GP) dipeptide repeat proteins in mice for at least six months while maintaining C9orf72 protein levels.
WVE-004, a stereopure antisense oligonucleotide designed, including PN backbone chemistry modifications, to selectively target transcriptional variants containing a hexanucleotide repeat expansion (G4C2) associated with the C9orf72 gene, thereby sparing normal C9orf72 protein.