This new collaboration builds on Wave’s ongoing research and development in splice correction programmes, including its lead DMD programme, WVE-210201, an investigational therapy targeting exon 51 currently in a global Phase 1 clinical trial. Wave’s next DMD programme, targeting exon 53, is expected to initiate clinical trials in Q1 2019.

Wave Life Sciences Ltd., a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, and Deep Genomics Inc., a biotechnology company that is building a new universe of genetic medicines using its machine learning-driven biomedical platform, today announced the formation of a collaboration to identify novel therapies to be developed by Wave for the treatment of genetic neuromuscular disorders.

“Wave is an industry leader in developing optimized oligonucleotides and in adopting science-based disruptive technologies,” said Brendan Frey, PhD, Scientific Founder and Chief Executive Officer of Deep Genomics. “For this reason, Wave and Deep Genomics are a good match. Wave’s efforts complement our discovery platform, which combines automation, high volume data acquisition and genome biology in a machine learning system. By working together, we aim to extend what is currently known about splicing targets in genetic neuromuscular disorders.”

“Deep Genomics is a world leader in building machine learning systems that incorporate advanced biological knowledge and data, and we are excited to use these systems for drug discovery,” said Paul Bolno, MD, MBA, Chief Executive Officer and President of Wave Life Sciences. “We believe this collaboration will enable a more profound understanding of splicing biology and illuminate new approaches to increase the size of patient populations with genetic neuromuscular disorders that may be eligible for treatment. We intend to use these new insights to expand the universe of druggable splicing targets beyond Duchenne muscular dystrophy and spinal muscular atrophy and direct our highly efficient stereopure oligonucleotides toward optimal regions or sequences within those targets.”

Under the collaboration, the companies will analyze and test oligonucleotides against potential therapeutic targets within multiple genes implicated in neuromuscular disorders. The analysis will use Deep Genomics’ machine learning platform to identify cause and effect relationships specific to neuromuscular-related targets that involve splicing regulation. Wave’s propriety chemistry platform will be used to validate targets and elucidate the implications of target intervention across different phenotypes, with the goal of expanding Wave’s pipeline of rationally designed oligonucleotides.

This new collaboration builds on Wave’s ongoing research and development in splice correction programs, including its lead DMD program, WVE-210201, an investigational therapy targeting exon 51 currently in a global Phase 1 clinical trial. Wave’s next DMD program, targeting exon 53, is expected to initiate clinical trials in Q1 2019.

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