ProQR Therapeutics, a Dutch biotech developing mRNA treatments for CF, announced its IPO terms today. They have developed a technique that targets and repairs defective messenger RNA (mRNA) and treats the underlying genetic mutation for CF, which they believe is superior to small molecules (e.g.. Kalydeco) and gene therapy treatments. ProQR plans to file an IND with the FDA in the 4Q for its Phase 1 trial, with top-line data expected in the 4Q 2015. The company also plans to use its mRNA repair approach to develop a therapy for diseases.
This sounds very promising. Lets hope that this new biotech can deliver on its promise and ad a new (inexpensive!?) drug to the CF market that will treat the underlying cause of CF and alleviate many symptoms to improve quality and length of life for people living with CF.
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- Chris
This sounds very promising. Lets hope that this new biotech can deliver on its promise and ad a new (inexpensive!?) drug to the CF market that will treat the underlying cause of CF and alleviate many symptoms to improve quality and length of life for people living with CF.
Join the discussion on Facebook! https://www.facebook.com/knowcf/posts/10152802393388777
- Chris