Biotech vet Astley-Sparke takes U.S. lead for pioneering gene therapy player

Philip Astley-Sparke--courtesy of Forbion

Longtime biotech exec Philip Astley-Sparke will take the U.S. lead for uniQure, the Amsterdam-based developer, which recently won the first European approval for a new gene therapy. Named as its new U.S. president, Astley-Sparke--who ran BioVex up until Amgen ($AMGN) bought it out in a $1 billion deal and recently was named a Forbion venture partner--tells FierceBiotech that his first task will be to secure a new manufacturing site for the pioneering uniQure, which is planning to file for its first U.S. approval later this year.

"The whole platform has been de-risked," says Astley-Sparke, citing the EU approval of uniQure's Glybera, a gene therapy that treats rare cases of lipoprotein lipase deficiency, an enzyme deficiency that prevents patients from metabolizing fatty blood particles, triggering inflammation that can lead to a long list of serious ailments. Only one or two people in a million suffer from the condition, and Glybera is expected to test payers' appetite for rare disease drugs once it hits the market with a price expected to approach $1 million, according to a recent report from Reuters.

Amsterdam Molecular Therapeutics had failed on several occasions to win an approval for Glybera. Early in 2012, Forbion financed a transfer of the AMT assets into uniQure, which is now one of several upstart biotechs which believe that the decades-long roller coaster ride for gene therapies is ending as a new generation of treatments starts to win regulatory approval. Cambridge, MA-based bluebird bio is also committed to pushing new gene therapies to regulators. And Astley-Sparke says his task will be to build the infrastructure of uniQure as it plots more product launches over the next 5 years.

"Hemophilia B is the next priority," says Astley-Sparke, with treatments for other conditions like porphyria to follow. His responsibilities as U.S. president also include scouting for fresh capital, which may involve a possible IPO at some point as uniQure builds up its commercial arm.    

- here's the press release

Related Articles:
Million-dollar therapy will test payers' tolerance of orphan drug prices
European Commission stamps approval on first gene therapy for rare disease
Biotech's gene therapy grabs first regulatory nod in Europe
Million-dollar therapy will test payers' tolerance of orphan drug prices
UniQure drug first gene therapy to get approval in West