gene editing
Lilly and MeiraGTx partner in $475m ophthalmology deal
The companies will develop and commercialise therapies for genetic conditions
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
The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter
By Sarah Mohamad and Lauren Coleman-Bennett
What do people look for in gene therapy materials? Results from our survey of patients and relatives
By Sam Fraser
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
CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix
The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US
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
Scientists discover new form of gene editing to recombine and rearrange DNA
The bridge RNA system allows researchers to edit, delete and convert DNA recombinases without using CRISPR