Merus NV added Gilead Sciences Inc. to its collaboration roster, entering a deal potentially worth more than $1.5 billion. While its previous agreements have focused primarily on bispecific antibodies, the Gilead alliance takes aim at trispecifics, antibodies capable of binding three targets at once. In other news, shares of Biomx Inc. (NYSE:PHGE) jumped 194% March 6, ending the day at 68 cents, up 45 cents, on news that it was merging with fellow phage-focused company Adaptive Phage Therapeutics Inc. and raised $50 million in a concurrent private placement.
Intellia Therapeutics Inc. and Recode Therapeutics Inc. have established a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis.
Vertex Pharmaceuticals Inc. upped its own ante with pivotal phase III data in its strong suit, cystic fibrosis (CF). Taking on Trikafta, the company’s bestseller, Vertex’s once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza) combo hit all primary and key secondary endpoints in two randomized controlled studies of those ages 12 and older.
The Cystic Fibrosis Foundation (CF Foundation) has agreed to provide Prime Medicine Inc. with up to $15 million to support the development of prime editors for the treatment of cystic fibrosis (CF).
ΔF508-CFTR is the most common CFTR mutation in cystic fibrosis (CF), which leads to destabilization of CFTR’s first nucleotide-binding domain (NBD1), contributing centrally to defective ΔF508-CFTR folding, trafficking and function.
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 identified cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
Vertex Pharmaceuticals Inc. has divulged cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
With more than 70,000 people living with cystic fibrosis (CF) worldwide, according to the Cystic Fibrosis Foundation, the introduction of CF transmembrane conductance regulator (CFTR) modulator therapies has revolutionized treatment of the disease. However, these drugs are not effective for around 10% of CF patients, driving a significant unmet therapeutic need. One startup hoping to address this is Anoat Therapeutics.
