Cystic Fibrosis: The Remaining 10% Still Lack Effective Treatments
Despite major advances in cystic fibrosis (CF) treatment, about 10% of patients remain without effective therapies due to genetic mutations that prevent them from benefiting from breakthrough drugs like Vertex Pharmaceuticals' Trikafta and Alyftrek. Emily Kramer-Golinkoff founded the nonprofit Emily's Entourage in 2011 with a mission to accelerate research for these underserved CF patients, who often face drug-resistant infections or side effects. Recent clinical trials have faced setbacks: In May, Vertex and Moderna halted an mRNA-based trial due to tolerability issues, while Boehringer Ingelheim ended its gene therapy trial in February. Despite this, Emily's Entourage has raised over $22 million since 2011, funding 51 research projects exploring antisense oligonucleotides (ASOs), small molecules, and gene therapies. One promising approach is a gene therapy developed by Spirovant Sciences currently in early-stage trials. The foundation also supports phage-based treatments for drug-resistant infections that are fatal to many CF patients.
Topics: Cystic Fibrosis Gene Therapy Nonprofits Pharmaceutical R&D