Wave Life Sciences will use Deep Genomics’ machine learning-driven biomedical platform to discover novel therapies for treating genetic neuromuscular disorders, the companies said today, through a collaboration whose value was not disclosed.
Under the collaboration, the companies have agreed to analyze and test oligonucleotides against potential therapeutic targets within multiple genes implicated in neuromuscular disorders. Wave will develop the new therapies using its propriety chemistry platform to validate targets and elucidate the implications of target intervention across different phenotypes, the companies said.
Wave aims to expand its pipeline of rationally designed oligonucleotides, which are intended to treat serious, genetically-defined diseases.
The analysis of oligonucleotides will use Deep Genomics’ machine learning platform to identify cause and effect relationships specific to neuromuscular-related targets that involve splicing regulation.
Deep Genomics has developed a platform designed to help discover drug candidates that target the genetic determinants of disease at the level of RNA or DNA. The platform produces on-target and genome-wide off-target effect data for every compound identified.
The platform is being used by Deep Genomics to evaluate over 69 billion molecules against 1 million targets, in silico. The effort, dubbed “Project Saturn,” is designed to generate a library of 1,000 compounds that are experimentally verified to manipulate cell biology as intended.
According to Deep Genomics, Project Saturn automatically identifies all ASOs that achieve a particular change, tailored to individual genotype and leveraging up to six biological mechanisms, and counting. Project Saturn is also analyzing more than 100,000 standard protein isoforms for altered expression, plus thousands of novel and anticipated isoforms.
Based in Toronto, Deep Genomics was launched in 2015, and as of last month, said it had 27 employees with advanced degrees and industry experience in artificial intelligence, automation, software engineering, genomics, molecular and cell biology, human genetics, molecular diagnostics, and organic chemistry. Deep Genomics completed a $13 million Series A financing round in September 2017 led by Khosla Ventures, with participation by True Ventures.
The company says it benefits from its location in the MaRS Discovery District, next to four research hospitals, three medical research institutes, and the AI research labs of Google, Uber and the Vector Institute for Artificial Intelligence.
Last month, Deep Genomics announced it will spend C$10 million ($7.9 million) toward expanding its preclinical platform and developing therapies for metabolic and neurodegenerative disorders. The company committed to using its platform toward unlocking new classes of anti-sense oligonucleotide therapies that were previously inaccessible, and advancing them into the clinic.
The collaboration builds on Wave’s ongoing research and development in splice correction programs—which include its lead program WVE-210201, a Duchenne muscular dystrophy (DMD) candidate targeting exon 51.
In November 2017, Wave launched a global Phase I clinical trial assessing WVE-210201. The multicenter, double-blind, placebo-controlled clinical trial is designed to assess the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in DMD patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients between the ages of 5 and 18 years.
Data from the Phase I trial for WVE-210201 is expected in the third quarter. If the trial is successful, Wave has said, the data will facilitate a rapid transition to a double-blind, placebo-controlled, multi-dose efficacy study where dystrophin expression and clinical outcomes will be assessed.
The clinical program is designed to allow patient participants in the Phase I trial to enroll in an open-label extension study in which dosing with WVE-210201 will continue. Both the open-label extension study and the planned efficacy study are expected to generate an interim efficacy readout of dystrophin expression from muscle biopsies in the second half of 2019.
Wave’s next DMD program, targeting exon 53, is expected to initiate clinical trials in Q1 2019. The company has said it is exploring both intravenous and subcutaneous administration for the WVE-210201 and exon 53 programs.