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Cystic Fibrosis: ProQR Therapeutics IPO

September 22, 2014

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.


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.

Please Note: “Wall Street & Broadway” by Fletcher6 (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

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