In experiments with mice, scientists found a protein that triggers vision loss caused by diabetic retinopathy and retinal vein occlusion, two diseases characterized by the closure of blood vessels in the retina.
The first US Food and Drug Administration-approved gene therapy for an inherited disorder will cost you $425,000 per eye.
GlaxoSmithKline Plc has received clearance by the UK for its most expensive treatment, for an illness known as “bubble-boy disease.”
Novartis has received U.S. Food and Drug Administration approval for its breakthrough therapy designation for treating some breast cancer patients.
MilliporeSigma has signed a commercial supply agreement with bluebird bio for viral vector manufacturing, in the hopes of accelerating treatments for serious diseases.
British doctors have seen a breakthrough in heamophilia A, a genetic defect that sees patients not producing a protein to stop bleeding.
Selectively destroying myofibroblasts that drive the fibrosis seemed to halt or even reverse fibrosis’s damaging effects in mice.
A biotech firm based in Boulder, CO is providing free access to MAD7 a novel enzyme in the gene editing field.
The largest organisation of scientists who work in gene and cell therapy have come out against DIY gene therapies.
Precision Medicine: A simple test aims to predict which treatment for cystic fibrosis is most likely to work for each patient.
We’ve caught up with Mike before his participation in the panel: ‘Can we fully integrate genomics into the NHS within 5 years?’ at the Festival of Genomics 2018.
A potentially new treatment that could delay or even prevent Alzheimer’s is being hotly pursued.
Spark Therapeutics’ gene therapy for patients with haemophilia has seen some early success in clinical trials, after being tested in 10 male patients