Vertex Pharmaceuticals Inc

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

There is no clear path for African patients to get access to the treatments, which have multimillion-dollar price tags and are highly complex to manufacture and deliver.

F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing

People with the genetic disease have new opportunities to eliminate their symptoms, but the treatments come with obstacles that limit their reach.

Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval

The first treatment that relies on CRISPR is expected to receive U.S. approval next month. But it may cost millions of dollars per patient.

Panel Tells F.D.A. That CRISPR Sickle Cell Cure Is Safe Enough for Patients

The decision by an advisory committee may lead to Food and Drug Administration approval of the first treatment for humans that uses the CRISPR gene-editing system.

‘Miracle’ Cystic Fibrosis Drug Kept Out of Reach in Developing Countries

Vertex Pharmaceuticals is not making its drug, Trikafta, available in poorer countries, where thousands of diagnosed patients stand to benefit.