Cystic Fibrosis: ProQR Therapeutics IPO
ProQR Therapeutics B.V., a biopharmaceutical company (Leiden, Netherlands) founded in 2012, is developing RNA-based therapeutics for the treatment of genetic disorders. ProQR Therapeutics announces on September 18th the pricing of its Initial Public Offering (IPO) of 7.5 million shares at $13/share, raising approximately $98 million. ProQR Therapeutics plans on listing on the NASDAQ under the symbol “PRQR”.
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:
“ …. 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 September 2013, ProQR Therapeutics receives FDA Orphan Drug Designation (ODD) for QR-010 for CF:
ProQR Therapeutics FDA ODD Database Record
| Generic Name: | Anti-sense oligonucleotide consisting of 2’0-Me RNA with a phosphorothioate backbone |
| Trade Name: | n/a |
| Date Designated: | 09-23-2013 |
| Orphan Designation: | Treatment of cystic fibrosis |
| Orphan Designation Status: | Designated |
| FDA Orphan Approval Status: | Not FDA Approved for Orphan Indication |
| Sponsor: | ProQR Therapeutics B.V. Darwinweg 24, 2333 CR Leiden The Netherlands NETHERLANDS The sponsor address listed is the last reported by the sponsor to OOPD. |
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The EMA gives QR-010 ODD in October 2013.
In August 2014, Cystic Fibrosis Foundation Therapeutics (CFFT), a subsidiary of the Cystic Fibrosis Foundation (CFF), enters into an agreement with ProQR Therapeutics to provide up to $3 million to support the clinical development of QR-010.
ProQR Therapeutics is also developing QR-110 as a treatment for the rare genetic eye disorder, Leber’s Congenital Amaurosis (LCA). Infants affected by LCA are often blind at birth. LCA is usually inherited as an autosomal recessive genetic condition.
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