Labroots

Efficient base editing in cystic fibrosis airway epithelial cells

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...

Krystal Biotech Touts Positive KB407 CF Gene Therapy Data, Eyes Repeat Dosing and Registrational Path

Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...

Krystal Biotech Announces Positive Interim Clinical Update from KB407 Phase 1 CORAL-1 Study with Confirmation of Wild-Type CFTR Delivery to the Lungs of Patients with Cystic ...

Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutationsKB407 transduction confirmed in all ...
European Medical Journal

Residual CFTR Function Tied to Healthier Blood Sugar

Residual CFTR function in cystic fibrosis was associated with better glucose tolerance and insulin secretion, with CFTR ...
Nature

Congenital Bilateral Absence of Vas Deferens and CFTR Gene Mutations

Congenital bilateral absence of the vas deferens (CBAVD) is a recognised cause of male infertility most frequently linked to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) ...
Nasdaq

Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
Yahoo

Genetic medicine can leave people with rare mutations behind. But there's new hope

This photo provided by Emily’s Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare genetic mutation, during a trip to Maine. (Emily’s Entourage via AP) Emily ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...