Wave Life Sciences Ltd., a clinical stage genetic medicine company, designs, optimizes, and produces novel stereopure oligonucleotides through PRISM, a discovery and drug developing platform. It is developing oligonucleotides target ribonucleic acid to reduce the expression of disease-promoting proteins or restore the production of functional proteins, or modulate protein expression. The company also develops WVE-004, a C9orf72 molecule for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia; WVE-003, a mutant huntingtin SNP3 molecule for the treatment of Huntington's disease; WVE-N531, an Exon 53 molecule for the treatment of Duchenne muscular dystrophy; and ATXN3, a discovery stage program for the treatment of spinocerebellar ataxia 3, as well as multiple preclinical programs for CNS disorders. In addition, it focuses on developing GalNAc-conjugated AIMers to treat hepatic indications comprising Alpha-1 antitrypsin deficiency (AATD); and two preclinical programs, such as Usher syndrome type 2A (USH2A) and retinitis pigmentosa due to a P23H mutation in the RHO gene (RhoP23H) for the treatment of retinal diseases. It has collaboration agreements with Pfizer Inc., Takeda Pharmaceutical Company Limited, University of Oxford, University of Massachusetts, Western Washington University, Grenoble Institute of Neurosciences, IRBM S.p.A, University of Louisville, and University College London. The company was incorporated in 2012 and is based in Singapore.
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Wave Life Sciences Ltd., a clinical stage genetic medicine company, designs, optimizes, and produces novel stereopure oligonucleotides through PRISM, a discovery and drug developing platform. It is developing oligonucleotides target ribonucleic acid to reduce the expression of disease-promoting proteins or restore the production of functional proteins, or modulate protein expression. The company also develops WVE-004, a C9orf72 molecule for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia; WVE-003, a mutant huntingtin SNP3 molecule for the treatment of Huntington's disease; WVE-N531, an Exon 53 molecule for the treatment of Duchenne muscular dystrophy; and ATXN3, a discovery stage program for the treatment of spinocerebellar ataxia 3, as well as multiple preclinical programs for CNS disorders. In addition, it focuses on developing GalNAc-conjugated AIMers to treat hepatic indications comprising Alpha-1 antitrypsin deficiency (AATD); and two preclinical programs, such as Usher syndrome type 2A (USH2A) and retinitis pigmentosa due to a P23H mutation in the RHO gene (RhoP23H) for the treatment of retinal diseases. It has collaboration agreements with Pfizer Inc., Takeda Pharmaceutical Company Limited, University of Oxford, University of Massachusetts, Western Washington University, Grenoble Institute of Neurosciences, IRBM S.p.A, University of Louisville, and University College London. The company was incorporated in 2012 and is based in Singapore.
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Wave Life Sciences Ltd., a clinical stage genetic medicine company, designs, optimizes, and produces novel stereopure oligonucleotides through PRISM, a discovery and drug developing platform. It is developing oligonucleotides target ribonucleic acid to reduce the expression of disease-promoting proteins or restore the production of functional proteins, or modulate protein expression. The company also develops WVE-004, a C9orf72 molecule for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia; WVE-003, a mutant huntingtin SNP3 molecule for the treatment of Huntington's disease; WVE-N531, an Exon 53 molecule for the treatment of Duchenne muscular dystrophy; and ATXN3, a discovery stage program for the treatment of spinocerebellar ataxia 3, as well as multiple preclinical programs for CNS disorders. In addition, it focuses on developing GalNAc-conjugated AIMers to treat hepatic indications comprising Alpha-1 antitrypsin deficiency (AATD); and two preclinical programs, such as Usher syndrome type 2A (USH2A) and retinitis pigmentosa due to a P23H mutation in the RHO gene (RhoP23H) for the treatment of retinal diseases. It has collaboration agreements with Pfizer Inc., Takeda Pharmaceutical Company Limited, University of Oxford, University of Massachusetts, Western Washington University, Grenoble Institute of Neurosciences, IRBM S.p.A, University of Louisville, and University College London. The company was incorporated in 2012 and is based in Singapore.