The biotech company said it is well-stocked to supply its approved drugs for cystic fibrosis to patients. Meanwhile, it plans to implement virtual visits and ship drugs to participants in its ongoing trials, while pausing enrollment and delaying initiation in some.
Shares of the company rose 9 percent on the news that the FDA had approved Trikafta. The drug will be an option for patients using the company's other approved CF drugs.
A new report from Relatient, A Data-Driven Guide to Patient Access Succes, highlights how focusing on data accuracy and relevance can enhance the performance of healthcare practices.
The Boston-based company said it would acquire Semma Therapeutics, which is developing a stem cell-derived therapy and device for the disease. Preclinical data were announced in July, and clinical trials are planned for 2020.
The Phase I/II trial is in beta-thalassemia, a rare blood disorder. Another Phase I/II study of the same drug, in sickle cell disease, is expected to infuse its first patient in mid-2019.
Shares of gene-editing companies were down Tuesday morning following the news, but analysts sought to reassure investors about Editas.
The agency also accepted the two companies' IND to begin the Phase I/II study of CTX001 in patients with sickle cell disease, sending CRISPR shares up more than 11 percent Thursday morning.
When it comes to managing inbound phone calls, underperformance has devastating cost implications.
Boston-based drugmaker Vertex Pharmaceuticals leads the round and also enters partnership with UK firm to aid its own drug development efforts.
FDA slaps clinical hold on Crispr Therapeutics and Vertex Pharmaceuticals' upcoming gene-editing trial.
Vertex Pharmaceuticals, known for its line of cystic fibrosis drugs, has entered the gene editing space with a new $105 million deal with CRISPR Therapeutics.
Vertex Pharmaceuticals founder Joshua Boger says that venture capitalists have too much of a say in how to shape a startup - when really, it should be left up to a solid team of scientists.
Zelis CEO Amanda Eisel shares her perspective on how the company is solving the problems of a fragmented health financial system to benefit all.
The money will push two stem-cell technologies deeper into clinical trials and create Ohio's first Pluripotent Stem Cell Facility.