Spark Therapeutics - 2014 Fierce 15

Spark Therapeutics
Spark joins a new wave of gene therapy players out to transform medicine

CEO: Jeffrey Marrazzo
Based: Philadelphia
Founded: 2013
Clinical focus: Gene therapy

The scoop: Some biotechs are born out of small gambles and plenty of NIH-funded basic academic research. A rare few leap onto the biotech scene with a big bankroll and a pipeline of clinical-stage assets that runs right into Phase III. Count Philadelphia's Spark Therapeutics in the latter camp.

Children's Hospital of Philadelphia (CHOP) clearly felt that its roster of researchers--including the notable Dr. Jean Bennett--at its Center for Cellular and Molecular Therapeutics were well on their way with a late-stage program for retinal dystrophies when it decided to spin out the program and a core group of staffers. A hemophilia B program is coming up behind that lead treatment.

What makes Spark Fierce: In just a few short years, gene therapy has gone from an extraordinarily high-risk field with a long, troubled past to a clinical effort worth watching closely. Bluebird bio, out of the Third Rock stable of companies, has helped demonstrate how fast you can travel on this track. And Spark has plans to go all the way, building a fully integrated biopharma company in the process.

Behind the two clinical stage programs you'll find a full slate of preclinical projects underway. The lead therapy is for RPE65-related blindness, with follow-ups in the retinal disease field. In addition to hematologic disorders there's also an early-stage effort focused on neurodegenerative diseases.

Flush with cash from CHOP and a group of venture backers that includes Sofinnova, the company has been building a new HQ facility, complete with 28,000 square feet of space to house the growing team. Marrazzo is also building a staff of 50 for the venture, which plans to go the distance to become a fully integrated biopharma business. Along the way he expects to ink a few industry collaborations, including more partnerships like the one struck with Genable, which wanted to take advantage of Spark's knowledge of vectors.

"What we've seen is that many of the initial challenges, while not all solved, many of them have solutions," CEO Jeffrey Marrazzo told FierceBiotech after he landed a $73 million round from some A-list investors. "This is an evolution from research and scientific development to new product development. That's the phase we're in."

Marrazzo's board includes two key players from Sofinnova, Lars Ekman and Anand Mehra. Elliott Sigal, a venture partner at NEA who established a legendary rep as head of R&D at Bristol-Myers Squibb, is also on the board. "I was burned on (gene therapy) myself 20 years ago," he told Forbes recently. "When they told me gene therapy was back, I couldn't believe it."

As Sigal, Bennett and others in the field well understand, solid outcomes for even a handful of patients can help demonstrate the potential of gene therapies. Now Spark plans to be in the first wave of companies to start commercializing these potentially revolutionary treatments. If it makes it, that will confront the company with a big challenge in establishing how these pricey treatments can be paid for. For now, though, the focus is staying on R&D. And it's going head-to-head with some of the best in the biotech business. 

Investors: Sofinnova Ventures, Brookside Capital, Deerfield Management, Rock Springs Capital, funds and accounts managed by T. Rowe Price Associates, Wellington Management and two undisclosed funds joined with Children's Hospital of Philadelphia to fund the company through its B round.

For more:
Upstart Spark grabs a $73M round to back rapid expansion, PhIII study

-- John Carroll (email | Twitter)

Spark Therapeutics - 2014 Fierce 15

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