Nano cap Proteostasis Therapeutics (NASDAQ:PTI) is up 6% premarket on modest volume ahead of a presentation of results from an ex vivo study of its cystic fibrosis transmembrane conductance regulator (CFTR) modulators in organoids from individuals with CF who are ineligible for current standard-of-care CFTR modulator therapies due to their genotype (~2,300 European adults). The data, based on samples from up to 500 patients, are being presented at the Keystone Symposia on Tissue Organoids in Vancouver, BC.
The study is assessing responsiveness to the company’s CFTR potentiator dirocaftor, corrector posencaftor and amplifier nesolicaftor.
The European Commission is funding the organoid program as part of a strategic initiative aimed at building a road map for personalized treatments for CF.
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