Allievex Corp., a new company advancing an enzyme replacement therapy for Sanfilippo syndrome type B licensed from Biomarin Pharmaceutical Inc. through a registrational phase II trial, has marked the first close of a series A financing co-led by Novo Holdings and the startup's founder, Pappas Capital.

As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.