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.
Novartis AG is enlisting the help of Generate:Biomedicines Inc. and its artificial intelligence platform to generate drugs for multiple undisclosed targets. The number of targets and therapeutic areas also weren’t disclosed. Novartis will pick the targets, although the targets Generate has been working on are off limits. Generate will be responsible for creating the lead candidate, at which point Novartis will take over development.
Researchers from Boehringer Ingelheim Pharma GmbH & Co KG presented the discovery and preclinical characterization of new spleen tyrosine kinase (SYK) inhibitors, BI-894416 and BI-1342561, being developed for the treatment of asthma.
Tankyrases (TNKS) are involved in relevant cellular functions such as telomere maintenance or Wnt signaling, and their inhibition may be a potential treatment strategy for the management of hyperproliferative disorders because it counteracts profibrotic changes and blocks the synthesis of extracellular matrix proteins.
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