Gene therapy

Advanced therapy medicinal products fastest growing area of biopharma, says report

Employment in the UK’s gene therapy business, and other advanced therapy medicinal products was the only part of the biopharma sector to witness an increase in jobs in the past five years, according to a government report published on August 4 2017.…

‘Living drug’ may soon be approved by the FDA for treatment of acute lymphoblastic leukaemia

CAR-T cell immunotherapy recommended for approval and may get FDA go ahead by the end of 2017.…

An examination of the scientific history of medical genetics

Life histories of genetic disease: patterns and prevention in postwar medical genetics by Andrew J Hogan…

Conventional therapies and emergent precision medicines for cystic fibrosis: challenges and opportunities

Around 27 years ago, the discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene – responsible for causing cystic fibrosis (CF) – marked a significant moment in the history of the condition. Despite the early promise of this target for gene therapy, a universally…

The history of the study of the gene

The gene: an intimate history by Siddhartha Mukherjee…

Developing gene therapy to treat cystic fibrosis: challenges and successes

More than 25 years after the gene for cystic fibrosis was discovered, there are signs that correcting this genetic deficit could soon be possible.…

‘One-size-fits-all’ approach will not work for drug evaluation and funding

Evaluation and funding of the most promising and innovative medicines will require a separate approach to other drugs in the future, according to the chair of the government-commissioned accelerated access review.…

Advanced therapy medicinal products present challenges for pharmacists

Few pharmacists have experience handling advanced therapy medicinal products (ATMPs) — medicines based on genes, cells or tissue engineering — but the field is likely to grow and presents special challenges for pharmacists.…

Gene therapy for ‘bubble boy’ syndrome on track for EU approval

Children with an extremely rare immune deficiency disease are set to have access to gene therapy, following a recommendation for approval by the European Medicines Agency.…

Researchers unravel function of HIV defence protein

Researchers investigate why TRIM5α protein lost ability to degrade HIV in rhesus monkey study.…