Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...

This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...

Research in genetics and biotechnology has advanced by leaps and bounds in recent years, offering new hope for the treatment ...

But if this therapy works, it will heal the muscle ... To fix the dysferlin mutation, Escobar uses CRISPR-Cas9, which is often described as "gene-editing scissors" and for which a Nobel Prize ...

CRISPR-Cas9 genome editing exploits the CRISPR-Cas ... activity to SpCas9 and can be effectively applied in targeted therapy, demonstrating its potential as a powerful gene editing tool.

Vertex Pharmaceuticals (VRTX) announced a reimbursement agreement with NHS England for eligible sickle cell disease patients to access the ...

Vertex Pharmaceuticals Inc. (VRTX), a commercial-stage global biotechnology company, announced Friday a reimbursement agreement with ...

Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) shares are trading higher Friday following two major developments that strengthen ...

People in England with sickle cell will be able to get the world’s first CRISPR-based medicine under an agreement between the ...

Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell ...