Vertex Pharmaceuticals Inc.’s chief operating officer Stuart Arbuckle said during the Stifel Healthcare Conference in November that his firm’s launch last year of Casgevy (exagamglogene autotemcel) for sickle cell disease and transfusion-dependent beta-thalassemia “was the beginning of diversifying on top of our cystic fibrosis [CF] base” – a base that itself continues to grow.
It was hypothesized that the MEK1/2 inhibitor ATR-002 could reduce inflammation and clear Staphylococcus aureus infection during cystic fibrosis, thus potentially showing a dual effect.
As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.
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 Splisense Ltd. and affiliated organizations recently reported preclinical data for SPL-84, an inhaled antisense oligonucleotide drug candidate being developed for the treatment of patients with cystic fibrosis carrying the 3849 +10 kb C-to-T (3849) mutation.
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR.
Prime Medicine Inc. has announced its plans to strategically focus its efforts on a set of high value programs as it advances its pipeline of next-generation gene editing therapies.
Cystic fibrosis (CF) is characterized by lack of hydration in the airways by impaired functioning of cystic fibrosis transmembrane conductance regulator (CFTR), leading to infection, inflammation and lung tissue damage. It is hypothesized that inhibiting the epithelial sodium channel (ENaC) in the airways in CF may enhance the mucociliary clearance (MCC) and provide clinical benefit, but numerous inhaled ENaC blockers have failed in clinical trials. Enterprise Therapeutics Ltd. is developing an inhaled ENaC blocker compound, ETD-001, for the treatment of CF.
Sionna Therapeutics Inc.’s approach with small molecules in cystic fibrosis (CF) yielded the Boston-based firm an upsized and oversubscribed $182 million series C financing. The company is working on drugs that could fully restore the function of the CF transmembrane conductance regulator protein by stabilizing the first nucleotide-binding domain (NBD1). Four compounds are expected to enter the clinic this year – three NBD1 stabilizers and one ICL4 modulator.
