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Cystic Fibrosis: Successes, Opportunities And Challenges

 
• By Lucie Ellis-Taitt

What is next for the treatment of cystic fibrosis, a rare disease market that has boomed in recent years on the back of new drug approvals with hefty price tags? Pipelines are full, but securing reimbursement is a challenge.

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Vertex Pharmaceuticals Inc.has quickly established itself as the one to beat across the cystic fibrosis market, and the company is expecting critical data readouts for some of its pipeline products this year. Still, the early-stage pipeline for this rare disease indication is plentiful, including activity from Vertex's former hepatitis C rival, AbbVie Inc.

In parallel to new therapies coming to the fore only a few years after the launch of Vertex's standard-of-care-altering Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor) products, market access issues for efficacious...

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Rising Leaders 2025: Pedro Valencia’s ADC Vision At AbbVie

 
• By Jo Shorthouse

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Leaders At The Frontier: Conversations From SynBioBeta 2025

 
• By David Wild

Mini-profiles of five synthetic biology companies and their leaders from SynBioBeta 2025 reveal how AI integration, data-driven platforms and interdisciplinary teams are revolutionizing drug discovery and manufacturing.