The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has significantly improved the therapeutic scenario for CF patients in the past decade. However, around 10% of patients harboring nonsense and splice-site mutations are nonresponsive to CFTR modulators.
Researchers from Arcturus Therapeutics Inc. and affiliated organizations presented preclinical data for LUNAR-CFTR, a novel mutation-agnostic, aerosolized CFTR replacement therapy for the treatment of cystic fibrosis (CF).
It is well known that mutations in the cystic fibrosis transmembrane regulator (CFTR) gene are causative of cystic fibrosis, a lethal autosomal recessive Mendelian disorder. Several studies have also pointed to an association between CFTR mutations and inflammatory bowel disease (IBD).
Vertex Pharmaceuticals Inc. has received clearance from the FDA for its IND application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF).
A study published in Nature Communications revealed a new antisense oligonucleotide therapy applicable to the W1282X mutation of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis.
Jonathan Moore, who was employee No. 36 when he came to Vertex Inc. in 1990 and ended up spending the next 28 years at the company, realized he wasn’t done with his work in ATP-binding cassette (ABC) transporters and has now formed Rectify Pharmaceuticals Inc. The new company just closed on a $100 million series A financing to fund development of a pipeline of therapies to restore ABC transporter function for treating genetic diseases.
