precision medicines
Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE
The rare inherited disease affects approximately 11,000 people in the UK
Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval
The rare inherited disease affects more than 109,000 people worldwide
Vertex’s triple combination cystic fibrosis therapy Alyftrek approved by MHRA
Over 11,000 people in the UK are affected by the rare genetic disease
NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease
About 15,000 people in England are living with the inherited blood disorder
NICE recommends Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia
An estimated 460 patients in England with severe cases of the blood disorder could be eligible for Casgevy
Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m
The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology
Vertex and Genomics expand collaboration to accelerate precision medicines
The partners have been working together since 2018 to develop treatments for serious diseases
Vertex to expand autoimmune and inflammatory disease pipeline with $4.9bn Alpine acquisition
The company will gain rights to Alpine’s lead molecule, povetacicept, to treat IgA nephropathy
EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders
There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy
Vertex shares positive late-stage results for triple combination cystic fibrosis treatment
The company compared the treatment to its current triple cystic fibrosis therapy
UKHSA publishes five-year strategy to detect risk-posing pathogens in UK
The strategy will help to improve the UK's response to infectious disease threats
FDA approves Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia
The companies estimate that approximately 1,000 patients in the US will be eligible for the one-time treatment