New Orphan Drug Targeting F508delta Mutation Being Developed By ProQR Therapeutics

ProQR Therapeutics B.V., a biopharmaceutical company based in Leiden, Netherlands and founded in 2012, is developing RNA-based therapeutics for the treatment of genetic disorders. They recently announced their stock market listing, raising approximately $98 million toward the development of their new drugs.

ProQR Therapeutics’ lead candidate is QR-010, a RNA-based oligonucleotide for the treatment of Cystic Fibrosis (CF). Per the European Medicines Agency’s (EMA) website, QR-010: “ QR010…. is an ‘anti-sense oligonucleotide’, a very short piece of synthesized RNA (a type of genetic material involved in the production of proteins). This anti-sense RNA specifically attaches to the ‘sense’ RNA with the F508delta mutation which is responsible for the production of the abnormal CFTR protein in cystic fibrosis. As a result, the anti-sense RNA is expected to induce the repair of the genetic RNA abnormality, leading to the production of a fully functional CFTR protein.”

ProQR Therapeutics plans on filing an FDA Investigational New Drug (IND) application by the end of 2014 with a Phase Ib clinical trial to follow. ProQR Therapeutics is targeting the DF508 CF mutation, which is the most prevalent mutation comprising about 70% of all CF patients.

In August 2014, Cystic Fibrosis Foundation Therapeutics (CFFT), a subsidiary of the Cystic Fibrosis Foundation (CFF), entered into an agreement with ProQR Therapeutics to provide up to $3 million to support the clinical development of QR-010.

Bottom Line:

  • ProQR Therapeutics is developing a new orphan drug called QR010
  • ProQR Therapeutics plans to schedule a Phase Ib clinical trial in a few months

Cystic Fibrosis: ProQR Therapeutics IPO