PRQR 简况
ProQR Therapeutics N.V. (ProQR), formerly ProQR Therapeutics B.V., is a pre-clinical stage biopharmaceutical company based in the Netherlands. The Company is engaged in the discovery and development of ribonucleic Acid (RNA)-based therapeutics for the treatment of severe genetic disorders. It 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. Its product candidates include QR-010, an RNA-based oligonucleotide for the treatment of cystic fibrosis (CF), QR-110, an oligonucleotide and for the treatment of Leber’s congenital amaurosis (LCA).
展开