Michael Panzara, M.D.Chief Medical Officer at Wave Life SciencesSpeaker
Profile
Wave Life Sciences’ proprietary discovery and drug development platform PRISMTM enables the precise design, optimization and production of stereopure oligonucleotides for genetically defined diseases. PRISM combines our unique ability to construct stereopure oligonucleotides with a deep knowledge of how the interplay among oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacologic properties. Our current focus for clinical development is in neurology. We are conducting two clinical trials in Huntington’s disease (HD), advancing additional HD programs, as well as targeting C9ORF72 in amyotrophic lateral sclerosis and frontotemporal dementia. We are also advancing discovery research in inherited retinal diseases and hepatic diseases.
Agenda Sessions
Stereopure Oligonucleotides in Development for the Treatment of Genetically Defined
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