Profile: ProQR Therapeutics NV (PRQR.A)
15 May 2019
ProQR Therapeutics N.V. (ProQR), formerly ProQR Therapeutics B.V., is a pre-clinical stage biopharmaceutical company. The Company is engaged in the discovery and development of Ribonucleic Acid (RNA)-based therapeutics for the treatment of severe genetic disorders. Utilizing its RNA repair technologies, the Company is developing a pipeline in severe genetic disorders, such as cystic fibrosis (CF) and Leber's congenital amaurosis (LCA). The Company designs its therapeutic candidates to specifically target and repair the defective messenger RNA, or Messenger Ribonucleic Acid (mRNA), that is transcribed from a mutated gene in order to restore the expression and function of normal, or wild-type protein. The Company's product candidates include QR-010 and QR-110. The Company is focusing on developing QRX- 911, QRX- 021, QRX- 313 for Epidermolysis bullosa, QRX- 704 for Huntington's disease, QRX- 812, QRX- 411 for Usher syndrome, QRX- 504 for Fuchs, QRX- 604 for Friedreich's ataxia and QRX- 203 for Alzheimer's disease.
The Company's lead product candidate, QR-010, is an RNA-based oligonucleotide. QR-010 is designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the delta-F508 mutation in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene of CF patients. The delta-F508 mutation is a deletion of over three of the coding base pairs or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-110 is designed to bind to the mutated location in the pre-mRNA, thereby leading to normally spliced or wild-type mRNA, which could produce wild-type or normal protein. QR-110 is designed to be administered through intravitreal injections in the eye.
The Company's product candidate, QR-110, is an oligonucleotide. QR-110 is designed to treat the disease by repairing the underlying cause in the mRNA, which results in the production of wild-type centrosomal protein 290 (CEP290 protein). QR-110 is designed to bind to the mutated location in the pre-mRNA, masking the cryptic splice site, thereby leading to normally spliced or wild-type mRNA, which could produce wild-type or normal protein. QR-110 is designed to be administered through intravitreal injections in the eye.
The Company competes with Vertex Pharmaceuticals Inc., Novartis International AG, Hoffmann-LaRoche Ltd., Pfizer Inc., Galapagos, AbbVie Laboratories, Shire, Sanofi, Bayer AG, Proteostasis, Corbus Pharmaceuticals, Nivalis and Gilead.
ProQR Therapeutics NV
LEIDEN 2333 CK