Fresh off Sanofi’s sell-off, Regeneron announced an expanded collaboration with gene editing company Intellia that gives the big biotech access to the coveted CRISPR gene editing technology for additional targets and indications, including hemophilia. The...

BioMarin’s gene therapy deal with DiNAQOR represents the first step in the company's plans to expand its gene therapy pipeline, while entering what’s becoming a hot cardiovascular indication: cardiomyopathy. The companies partnered to develop gene...

Robert Hershberg has joined Frazier Healthcare Partners as venture partner. The former EVP, CSO and head of BD and global alliances of Celgene Corp. helped coordinate the company’s $74 billion takeout by Bristol Myers Squibb...

BioMarin's hemophilia gene therapy gets Priority Review  BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) could become the first company to have a hemophilia gene therapy approved in the U.S. FDA accepted and granted Priority Review to a BLA...

Dan Spiegelman stepped down as EVP and CFO at BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to pursue other interests. SVP, Finance and Chief Accounting Officer Brian Mueller will serve as acting CFO as the rare diseases company...

A series of incremental but important improvements on the existing gene therapies are starting to address some of the technology’s key limitations. But some argue it will take a major overhaul of manufacturing practices to...

A pair of CNS approvals in the final days of 2019 has only added to the growing momentum buysiders were already tracking for 2020 in the once neglected disease area. Positive regulatory signals are converging...

Monday's basket of regulatory news includes two landmark regulatory applications for BioMarin's hemophilia gene therapy in the U.S. and EU, and FDA approval of Allergan's migraine drug. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) submitted a BLA to...

Roche cleared to complete Spark takeover  The planned merger of Roche (SIX:ROG; OTCQX:RHHBY) and Spark Therapeutics Inc. (NASDAQ:ONCE) has at last been cleared by the U.S. Federal Trade Commission and U.K. Competition and Markets Authority....

Sanofi (Euronext:SAN; NASDAQ:SNY) said Muzammil Mansuri, EVP, head of strategy and business development, will retire at the end of the month. His responsibilities will be divided between Alban de La Sablière, head of business development,...