Biossil, based in Toronto and co-founded by Dr. Alexander Mosa (Chair, CSO and co-founder) and Anthony Mouchantaf (CEO and co-founder), is making headway for rare diseases.

Biossil’s core model is to use machine learning to re-analyze clinical data from late-stage drug candidates that were previously shelved. The premise is that some of these drugs failed not because they lacked efficacy, but because trial design, particularly patient selection, was not well aligned with underlying biological heterogeneity. Two recent examples of this are Sickle Cell Disease and MPS VI.

This is particularly relevant in Sickle Cell Disease, where treatment options remain limited relative to disease burden, and where variability in patient response is clinically significant but not always well captured in conventional trials.

Using this approach, Biossil has identified two late-stage molecules with prior clinical data that may warrant renewed development in more precisely defined patient subgroups. Both of their SCD programs are now being advanced with a focus on stratification and indication refinement, essentially assessing whether these drugs were prematurely abandoned rather than definitively unsuccessful.

MPS VI is an ultra-rare condition where development activity has been limited and patients have few options. Our platform identified a previously shelved late-stage molecule that may be relevant to this population — a finding that might not have surfaced through conventional drug development, where small patient populations often get overlooked.

Biossil has identified a portfolio of 10 molecules that they believe show promise. For more information contact: Akwasi Owusu-Bempah <[email protected]>