Newsletter Signup - Under Article / In Page"*" indicates required fields For decades, cystic fibrosis has remained a relentless, life-limiting disease. But now, the future of cystic fibrosis care could be shifting from symptom management to something far more revolutionary. Gene therapy has emerged as a promising frontier in cystic fibrosis research, offering the potential to not just alleviate symptoms, but to address the genetic root of the disease itself.In this article, we will explore how gene therapy works, the biotechs progressing their genetic drug candidates, and why it might just be the breakthrough that people with cystic fibrosis have been waiting for.Cystic fibrosis: targeting the root of the diseaseAround 1,000 new cases of cystic fibrosis are diagnosed in the U.S. each year. People with cystic fibrosis tend to have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This gene codes for the CFTR protein, which is responsible for regulating the flow of salt in and out of the cells in the body

Newsletter Signup - Under Article / In Page"*" indicates required fields Many respiratory diseases have a huge effect on the lungs and other parts of the respiratory system and can be caused by various factors, including infection, exposure to air pollution, or smoking tobacco. This therapeutic area is very broad and there are numerous companies working on developing innovative therapies for different types of respiratory diseases, with cystic fibrosis and idiopathic pulmonary fibrosis being among the most popular indications for biotechs. In this article, we take a look at eight respiratory disease companies advancing novel therapies in 2025. Arrowhead Pharmaceuticals Technology: RNAi  Respiratory disease candidates: ARO-RAGE and ARO-MMP7Recent news: Collaboration with Sarepta Therapeutics that includes ARO-MMP7Equipped with a targeted RNAi molecule platform that leverages a natural pathway to “silence” genes causing disease, Arrowhead Pharmaceuticals has several focus areas, one of which is pulmonary diseases. The company has two clinical candidates in development related to respiratory diseases – one for inflammatory pulmonary diseases and one for idiopathic pulmonary fibrosis. The inflammatory pulmonary disease candidate is called ARO-RAGE. It is an RNAi therapeutic targeting the receptor for advanced glycation end-products (RAGE), which is implicated as an upstream mediator of type 2 and non-type 2 inflammatory cascades and is involved in the pathogenesis of asthma and numerous inflammatory diseases. The therapy is currently being investigated in a phase 1/2 study for the treatment of asthma

The Cystic Fibrosis Foundation has agreed to invest up to $15 million in ReCode Therapeutics to support their gene editing collaboration with Intellia