The UK firm has attracted more top-tier investors who are clearly impressed with its gene therapy for bubble baby syndrome and a high-quality pipeline in other very rare diseases.

This week’s announcements include board appointments by Biocom, Pliant Therapeutics and ProclaRx; senior appointments by FORMA Therapeutics and Tizona Therapeutics and various appointments in Asia by China Healthcare Reform Office, Shanghai Pharmaceuticals and the Johnson & Johnson Innovation Center in Shanghai.

Post-deal termination, approximately 14% of the drugs from those alliances go on to be approved, and of those still in development, nearly two-thirds advance at least one phase. Third in a series of follow-ups to IN VIVO’s 2014 study comparing/contrasting likelihood of approval and up-front payments on alliances, and the outcomes of relevant deals.

Investors and dealmakers are using recent discoveries about convergences between Gaucher’s disease and Parkinson’s in the lysosome to generate new therapeutic programs. Start-up Lysosomal Therapeutics thinks that the rare disorder can serve as a model system for developing new drugs to increase lysosomal function as a way to treat Parkinson’s disease and possibly other neurodegenerative disorders.