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GEN News Highlights : Jan 21, 2014

BioMarin Nabs Repligen's HDACi Compound Library

BioMarin Pharmaceutical is buying Repligen's histone deacetylase inhibitor (HDACi) library, along with the intellectual property related to it. BioMarin is paying Repligen $2 million up front and, per their agreement, will also provide Repligen any potential future milestone and royalty payments for developing, selling, and getting regulatory approval for the compounds.

BioMarin's CEO Jacques Bienaime said in a statement that the agreement is part of the firm's strategy to acquire preclinical programs that it can develop for people with serious unmet medical needs. The compounds, the firm says, could potentially be applied to Friedreich's ataxia and other neurological disorders.

"Fredreich's ataxia is a neurological disorder that affects tens of thousands of young people in the developed world, and we look forward to the opportunity to move a compound into the clinic in this indication," Bienaime added.

Biomarin has been busy lately: In November the FDA voted to approve its investigational drug Vimizim for the treatment of the ultra-rare lysosomal storage disorder Morquio A syndrome. The next day, Myriad Genetics announced a research collaboration with BioMarin to use Myriad's HRD (Homologous Recombination Deficiency) test to identify tumor types that may be sensitive to BioMarin's investigational product candidate, BMN-673.