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Clinical-stage gene therapy company launched

11 November 2013

By Dr Naqash Raja

Appeared in BioNews 730

The Children's Hospital of Philadelphia (CHOP) has invested US $50 million in a biotech start-up in an effort to commercialise gene therapy.

Spark Therapeutics will use the investment to take over two clinical trials for inherited blindness and haemophilia B.

'The creation of Spark is the culmination of a decade-long commitment by CHOP and our founding team to drive the field of gene therapy forward during a time when many in the industry had moved away,' said Jeffrey Marrazzo, CEO of Spark Therapeutics.

Only one gene therapy has been commercialised in the West: uniQure's Glybera (reported in BioNews 680), but this has not yet been approved for use in the US.

Spark Therapeutics is likely to seek US Food and Drug Administration approval for gene therapy with the phase III study for treatment of inherited blindness caused by a mutation of the RPE65 gene.

This stage builds on earlier work that saw 12 patients with RPE65-related retinal degeneration demonstrate 'notable improvement in visual function', as reported by Spark Therapeutics. The company is also conducting early-stage clinical trials for haemophilia and studies to address neurodegenerative conditions, blood disorders and other forms of inherited blindness.

CHOP has taken a more hands-on approach in this venture with most of Spark Therapeutics' management and advisers coming from its Center for Cellular and Molecular Therapeutics.

'It's a bold move', Gary Kurtzman, managing director for healthcare at Safeguard Scientifics told The Philadelphia Inquirer. 'Based on the technology and the assets and the expertise, I think it's a very smart move'.

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