Canadian biotechnology company Specific Biologics Inc announced on Wednesday that together with Western University it has been awarded a grant of more than CAD1.8m from Genome Canada and Ontario Genomics Genomic Applications Partnership Program (GAPP) to develop a machine learning (ML)-enabled prediction platform for Dualase genome editors.

The computational platform will be developed in collaboration with a research team led by Dr David Edgell at Western University, whose lab discovered Dualase editors, and builds on a longstanding partnership between the Edgell lab and Specific. The Dualase genome editing platform is designed to enable precise, efficient and programmable repair of small and large DNA sequences, offering broad therapeutic potential. Its two-site mechanism precisely targets and collapses toxic DNA repeat expansions with no off-target effects detected.

Specific Biologics' lead preclinical programme targets the toxic DNA repeat expansion in C9ORF72 amyotrophic lateral sclerosis (ALS), the most common genetic cause of terminal ALS with no disease-modifying therapies available.

The ML prediction platform will integrate large-scale genomic datasets, structural modelling, and experimental training data to develop predictive algorithms capable of rapidly identifying potent and highly specific Dualase editors for virtually any new therapeutic target. This is expected to accelerate lead optimisation beyond ALS to create new drug candidates for patients with high morbidity and mortality genetic diseases, including other repeat expansion diseases like Huntington's Disease.