Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

That’s the view of analysts at Jefferies, who suggest it could “re-ignite concerns over the use of lentiviral vectors and the ... 2 trial in patients with sickle cell disease (SCD) – another ...

including a study of CRISPR Therapeutics and Vertex Pharma’s CRISPR ex vivo drug for blood disorders beta thalassaemia and sickle cell disease. Allergan and Editas say they will test AGN-151587 ...

This either uses viruses as vectors of mRNA techniques not dissimilar to mRNA vaccines ... and rarer genetic diseases. The hematology targets sickle cell disease (BEAM-101 treatment) and is expected ...

Genetic diseases caused by mutations in DNA, affect millions worldwide, leading to conditions ranging from cystic fibrosis to sickle cell disease ... of engineered viral vectors, which are ...

“Currently, we are developing a pipeline of products to transfer to our Brazilian partners, starting with those used to treat haematological cancers, but then expanding to CAR-T therapies that target ...

Accumulation of clots causes some of the most severe symptoms of sickle cell disease, including strokes, kidney failure, ...

Gallbladder problems are a common complication in individuals with sickle cell disease. These issues often arise due to the breakdown of red blood cells and the subsequent formation of gallstones ...

"This research provides crucial insights into how the immune system functions in sickle cell disease," said Dr. Karina Yazdanbakhsh, Vice President and Director of Research at New York Blood Center ...