viernes, 31 de mayo de 2019

Vertex is that much closer to treating everyone with CF

The Readout
Damian Garde

Vertex is that much closer to treating everyone with CF

Vertex Pharmaceuticals has spent the last decade plus developing a mosaic of medicines for cystic fibrosis, targeting individual genetic nuances with the goal of eventually having something to offer everyone with CF. Right now, its drugs address about half of the patient population, but that number could rise to 90% next year.

Yesterday, Vertex laid out its plans to seek FDA approval for a triplet therapy that would add a drug called VX-445 to the previously approved Kalydeco and Symdeko. The company said it will submit the combination for review in the third quarter, and if everything goes according to plan, Vertex will soon have therapies for the majority of the world’s CF population.

Addressing that last 10% will be a challenge, however. Vertex’s drugs only work for patients whose genetic mutations leave at least some function in the CFTR gene, which is responsible for CF. For the minority with completely broken copies of CFTR, hope for a treatment rests on newfangled technologies like synthetic mRNA and genome editing.

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