Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.

Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."

The German major will collaborate with the US biotech to develop CRISPR gene-editing therapies, solidifying its interest in next-generation therapeutics.

Heading into the J.P. Morgan Healthcare conference, pharmas have been focusing on discovery alliances. J&J’s deals look into novel tissue-targeted drugs, ionic liquid delivery technology and cancer vaccine R&D. Lilly teams with Entos on novel methods for delivering neurology drugs.