WVE - Study update
Načítám obsah…
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.
