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WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease

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Management Comment
“We are pleased to receive Orphan Drug Designation for our first Huntington’s disease therapy and to advance what may be the first allele-targeted therapy into clinical trials, particularly as there are no approved disease-modifying treatments for HD,” said Paul Bolno, M.D., MBA, President and Chief Executive Officer of WAVE Life Sciences. “WVE-120101 is the first of our two lead allele-specific antisense programs for HD, and we are on track to file investigational new drug (IND) applications for both in late 2016. Each program distinctly targets the underlying genetic cause of HD and between these two programs, a substantial majority of HD patients would be covered. Our team at WAVE is driven by a sense of urgency and purpose as we work to advance disease-modifying treatments for HD patients.”
http://ir.wavelifesc...
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Catalyst Date
Occurred on:
Jun 21, 2016
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Related Keywords Orphan Drug Designation, Huntington’s Disease Therapy, Wve-120101, Rs362307, Htt, Single Nucleotide Polymorphism