Wave Life Sciences
Developer of oligonucleotide-based RNA medicines employing multiple RNA-targeting modalities (RNA editing, RNAi/siRNA, antisense, splicing modulation) and proprietary chemistry innovations. Maintains a clinical-stage pipeline addressing diseases such as alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington’s disease, and obesity. Operates in-house cGMP oligonucleotide manufacturing and engages in external collaborations to advance discovery and development.
Industries
Nr. of Employees
large (251-1000)
Wave Life Sciences
Cambridge, Massachusetts, United States
Patents
Products
Clinical-stage RNA editing candidate for SERPINA1 (alpha-1 antitrypsin deficiency)
A-to-I RNA editing oligonucleotide approach designed to correct a pathogenic single-base change in SERPINA1 mRNA to reduce protein aggregation in hepatocytes and restore functional circulating protein.
Antisense exon-skipping therapeutic for Duchenne muscular dystrophy (exon 53)
Antisense oligonucleotide designed to induce exon 53 skipping to restore the dystrophin mRNA reading frame and enable production of functional dystrophin protein.
Allele-selective antisense candidate targeting mutant HTT for Huntington’s disease
Allele-selective antisense oligonucleotide designed to selectively lower expression of mutant huntingtin mRNA using SNP-targeting to preserve wild-type HTT.
siRNA program targeting INHBE for obesity (GalNAc-conjugated)
siRNA approach to silence the INHBE gene to achieve sustained metabolic benefits and healthy weight-loss profile; includes development for monotherapy and combination approaches.
Clinical-stage RNA editing candidate for SERPINA1 (alpha-1 antitrypsin deficiency)
A-to-I RNA editing oligonucleotide approach designed to correct a pathogenic single-base change in SERPINA1 mRNA to reduce protein aggregation in hepatocytes and restore functional circulating protein.
Antisense exon-skipping therapeutic for Duchenne muscular dystrophy (exon 53)
Antisense oligonucleotide designed to induce exon 53 skipping to restore the dystrophin mRNA reading frame and enable production of functional dystrophin protein.
Allele-selective antisense candidate targeting mutant HTT for Huntington’s disease
Allele-selective antisense oligonucleotide designed to selectively lower expression of mutant huntingtin mRNA using SNP-targeting to preserve wild-type HTT.
siRNA program targeting INHBE for obesity (GalNAc-conjugated)
siRNA approach to silence the INHBE gene to achieve sustained metabolic benefits and healthy weight-loss profile; includes development for monotherapy and combination approaches.
Services
Onsite cGMP manufacturing capacity for oligonucleotide synthesis (high-throughput to large-scale), process development, analytical development, QC/QA, and partner manufacturing support.
Onsite cGMP manufacturing capacity for oligonucleotide synthesis (high-throughput to large-scale), process development, analytical development, QC/QA, and partner manufacturing support.
Expertise Areas
- RNA therapeutics (RNA editing, RNAi/siRNA, antisense, splice modulation)
- Oligonucleotide chemistry and stereopure synthesis
- Preclinical pharmacology and in vivo models
- Bioanalytical and biomarker assay development
Key Technologies
- A-to-I RNA editing (ADAR-based editing oligonucleotides)
- Antisense oligonucleotides
- RNA interference (siRNA)
- Stereopure oligonucleotide synthesis
News & Updates
Conference presentation and poster on INHBE silencing and other conference/booth materials.
Conference materials including poster on RNA editing of PNPLA3 decreasing hepatocyte fat content.
Peer-reviewed publication on RNA editing approaches for AATD.
Publication describing chemical modifications that improve ADAR-mediated editing.
Preclinical study evaluating stereopure antisense oligonucleotides targeting mutant huntingtin.
Conference presentation and poster on INHBE silencing and other conference/booth materials.
Conference materials including poster on RNA editing of PNPLA3 decreasing hepatocyte fat content.
Peer-reviewed publication on RNA editing approaches for AATD.
Publication describing chemical modifications that improve ADAR-mediated editing.
Preclinical study evaluating stereopure antisense oligonucleotides targeting mutant huntingtin.