Emily'sEntourage News & Analysis

10% of Cystic Fibrosis Patients Still Await Effective Treatment

Approximately 10% of cystic fibrosis patients do not benefit from recent advancements in CF treatments, which use drugs like Trikafta. These drugs, made by Vertex Pharmaceuticals, enhance the CFTR protein function for 90% of patients, transforming the disease into a more manageable condition. However, the remaining 10% of patients, born with other genetic mutations, experience a lack of effective treatment. Recent scientific efforts, including attempts by Vertex and Moderna using mRNA technology and Boehringer Ingelheim's gene therapy trials, were halted due to tolerability issues and other challenges. Emily's Entourage, a nonprofit founded by Emily Kramer-Golinkoff, has played a key role in continuing research for these patients. Since its inception in 2011, it has raised over $22 million, funding 51 research projects that explore novel treatments like antisense oligonucleotides and small molecules. Delivering drugs to affected lung cells poses significant scientific challenges because of the lungs' natural defenses and the presence of thick mucus in CF patients. This matters to ordinary investors as ongoing research might open opportunities for pharmaceutical companies to address the unmet needs of these patients, potentially leading to breakthroughs that could impact company valuations and future investment decisions.