'Genius' Kevin Eggan to Lead BioMarin’s Early R&D

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It’s been a rollercoaster two months for BioMarin Pharmaceutical. In August, the FDA blocked the much-anticipated hemophilia gene therapy Roctavian in a shocking move over concerns that it may not truly be a one-and-done treatment. On the upswing, a few days ago the FDA granted fast-track designation for the company's PKU investigational gene therapy, BMN 307. Today, more good news followed as BioMarin brings in MacArthur Fellowship “genius grant” recipient Kevin Eggan, Ph.D., as Group Vice President, Head of Research and Early Development.  

Eggan, a Harvard University professor, has garnered international recognition for his work in stem cell biology and cellular reprogramming, which led his research group into an entirely new strategy for studying human disease. His lab became the first to demonstrate that human somatic cells could be reprogrammed to an embryonic stem cell state, which inspired the discovery of factors used to generate induced pluripotent stem cells (iPSCs). Eggan’s team became the first to generate patient-specific iPSCs and use them to produce the cell type that degenerated in that individual.  

"We are thrilled to welcome Kevin to BioMarin. Throughout his career, Kevin has demonstrated a consistent track record of groundbreaking scientific research.  His philosophy of 'Dish to Bedside' illustrates how optimizing basic pre-clinical models can enhance therapeutic candidates and selection of trial participants," said Lon Cardon, Ph.D., Chief Scientific Strategy Officer, Senior Vice President at BioMarin. 

Eggan has kept busy with his work at Harvard in addition to his position as director of stem cell biology for the Stanley Center for Psychiatric Research at the Broad Institute and an institute member of the Broad Institute of MIT and Harvard. He co-founded Q-State Bioscience, Quralis and Enclear Therapies. Eggan has served on multiple boards, published upwards of 130 scientific articles and holds 13 patents.  

Eggan is excited to start his role at BioMarin.

“The possibilities for science to change the way we think about disease are plentiful, and BioMarin is an ideal place to translate research into groundbreaking therapies," Eggan said. "The company is big enough to devote resources into scientific discovery and small enough to allow innovation to flourish.”

BioMarin currently has six products on the market and three clinical trials in their pipeline. In addition to their hemophilia A that hit a speedbump in its approval with the FDA, vosoritide is in Phase III for the treatment achondroplasia (dwarfism) and in Phase I for treating dominant inherited short stature. Also in early phases are candidates for the treatment of PKU and hereditary angioedema. BioMarin focuses on first-in-class or best-in-class therapies to treat rare genetic diseases, which mostly affect children. 

Eggan stated, “As a researcher in academia, I love the freedom to follow the science.  In industry, I love the focus on establishing a clear path from biological understanding to a practical solution that can address unmet medical needs.  Now I get to do both." 

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