ProQR Therapeutics

Recipharm and Clinical Trial Consultants partner to streamline clinical trials

Monday, September 25, 2017

Recipharm, a contract development and manufacturing organization (CDMO), recently announced its new partnership with Swedish CRO Clinical Trial Consultants (CTC) for the development of a comprehensive list of services for first-in-human clinical trials. The partnership’s project, titled Recipharm Pathway to Clinic, is geared toward streamlining clinical trials and facilitating research from the early phase of formulation development to trial implementation.

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ProQR spins out Amylon Therapeutics for CNS therapeutics

Wednesday, September 13, 2017

ProQR Therapeutics has spun out Amylon Therapeutics, a privately-held company focused on the development of therapies for central nervous system (CNS) disorders, with seed funding from a group of institutional and private investors. As part of the transaction, ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders.

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ProQR’s QRX-421 for Usher syndrome receives FDA/EMA Orphan Designation

Tuesday, September 5, 2017

ProQR Therapeutics announced that investigational drug QRX-421 for Usher syndrome has received orphan drug designation (ODD) from the FDA and EMA. This marks the third drug candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA. QR-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Usher syndrome is the leading cause of combined deafness and blindness. 

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