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BioMarin’s Momentum Grows With Twin Blockbuster Plans

Dwarfism Drug Data Look Promising

Executive Summary

The frontrunner in hemophilia A gene therapy has been hit by data doubts, but this and achondroplasia drug could deliver in 2020.

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BioMarin CEO Suggests Hemophilia Gene Therapy Pricing In $2m-$3m Range

During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.

UniQure Aiming To Be First With Hemophilia B Gene Therapy

The company has come a long way since gene therapy flop Glybera, and claims best-in-class status for viral vector.

Astellas To Pay $3bn For Gene Therapy Company Audentes

Japanese company joins the ranks of big pharma buying up gene therapy pioneers

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