CFTR Amplifiers May Be Potent CF Mono or Combo Therapy, Study Says

Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported.

Investigational CFTR Amplifier PTI-428 Receives Orphan Drug Status in Europe

Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in development for the treatment of cystic fibrosis (CF). This recognition of the therapeutic potential of PTI-428 in Europe follows previously granted orphan drug, breakthrough therapy, and fast…