ProQR Therapeutics

ProQR Therapeutics ($PRQR)
Based: Leiden, Netherlands
IPO price: $13
Close Sept. 30: $17.19
Change: Up 32%
Raised: $98 million

Scoop: Despite its relative youth as a publicly traded company, cystic fibrosis biotech ProQR Therapeutics stands out among 2014's entrants with a sophisticated plan of action and impressive band of co-signers--and investors, who immediately bid up the company's top-of-the-range debut, would seem to be excited about its prospects. The Dutch biotech is developing an RNA-based therapy for CF, designed to correct an underlying cause of the disease by repairing defects in the CFTR gene. The lead candidate, QR-010, targets CF patients with two copies of the F508del mutation in their CFTR genes--the same population treated by Vertex Pharmaceuticals' ($VRTX) in-development combination of lumacaftor and ivacaftor, a potential blockbuster. And while its larger rival has a considerable head start, ProQr believes its compound is well-differentiated enough to stand out on the market. Among the company's founders are biotech luminaries Henri Termeer, who founded and led Genzyme, and Dinko Valerio, the man behind Crucell, which was acquired by Johnson & Johnson ($JNJ).

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Biotech IPOs: ProQR soars and Foamix thuds amid a volatile market
CF biotech ProQR shoots for a $75M IPO to challenge Vertex

ProQR Therapeutics