Wave Life Sciences
Wave Life Sciences is a leading RNA medicines company focused on developing innovative therapies across multiple modalities including RNA editing, splicing, RNA interference, and antisense silencing. The company aims to advance a diversified, sustainable pipeline of clinical and preclinical programs targeting diseases in muscle, liver, and the central nervous system, supported by scalable manufacturing capabilities.
Industries
Nr. of Employees
large (251-1000)
Wave Life Sciences
Boston, Massachusetts, United States, North America
Products
RNA editing program targeting SERPINA1 (alpha-1 antitrypsin deficiency)
Pipeline program employing RNA editing approaches directed at SERPINA1 for treatment of alpha-1 antitrypsin deficiency; listed patient population ~200,000 (US & Europe).
GalNAc-conjugated programs targeting PNPLA3, LDLR, and APOB
GalNAc-conjugated oligonucleotide programs aimed at liver disease targets including PNPLA3 (patient population ~9M), LDLR (heterozygous familial hypercholesterolemia; ~900K listed), and APOB (~70K listed).
RNAi program targeting INHBE (obesity)
RNA interference (siRNA) program using liver-targeted chemistry to modulate INHBE for metabolic disease; listed patient population ~175M (US & Europe).
Splicing modulation program for Duchenne muscular dystrophy (exon 53 and other exons)
Oligonucleotide splicing modulation programs targeting exon 53 (patient population ~2.3K) and additional exon targets (up to ~18K) for Duchenne muscular dystrophy.
Allele-selective silencing program targeting mutant HTT (Huntington's disease)
Allele-selective oligonucleotide silencing program for mutant huntingtin with listed symptomatic and pre‑symptomatic patient populations (~25K and ~60K respectively).
RNA editing program targeting SERPINA1 (alpha-1 antitrypsin deficiency)
Pipeline program employing RNA editing approaches directed at SERPINA1 for treatment of alpha-1 antitrypsin deficiency; listed patient population ~200,000 (US & Europe).
GalNAc-conjugated programs targeting PNPLA3, LDLR, and APOB
GalNAc-conjugated oligonucleotide programs aimed at liver disease targets including PNPLA3 (patient population ~9M), LDLR (heterozygous familial hypercholesterolemia; ~900K listed), and APOB (~70K listed).
RNAi program targeting INHBE (obesity)
RNA interference (siRNA) program using liver-targeted chemistry to modulate INHBE for metabolic disease; listed patient population ~175M (US & Europe).
Splicing modulation program for Duchenne muscular dystrophy (exon 53 and other exons)
Oligonucleotide splicing modulation programs targeting exon 53 (patient population ~2.3K) and additional exon targets (up to ~18K) for Duchenne muscular dystrophy.
Allele-selective silencing program targeting mutant HTT (Huntington's disease)
Allele-selective oligonucleotide silencing program for mutant huntingtin with listed symptomatic and pre‑symptomatic patient populations (~25K and ~60K respectively).
Services
cGMP manufacturing operations
On-site manufacturing operations for nucleic acid therapeutics under cGMP conditions, including production, facilities management, and quality oversight.
cGMP manufacturing operations
On-site manufacturing operations for nucleic acid therapeutics under cGMP conditions, including production, facilities management, and quality oversight.
Expertise Areas
- Nucleic acid therapeutic development
- RNA modality programs (editing, splicing, RNAi, antisense)
- cGMP manufacturing and scale-up
- Clinical program development for CNS, liver, and muscle indications
Key Technologies
- RNA editing
- RNA splicing modulation
- RNA interference (siRNA)
- Antisense oligonucleotide silencing