Orchard licenses the gene therapy technology from GlaxoSmithKline 

Orchard Therapeutics has permitted lentiviral stable cell line technology from GlaxoSmithKline for its use in hematopoietic stem cell gene therapies. GSK filed for patents on the tech before selling its rare disease gene therapy portfolio to Orchard in the year 2018.

The use of lentiviral vectors for modification of hematopoietic stem cells outside of the body has resulted in gene therapies, which have shown positive outcomes in people with multiple disorders, including complex genetic diseases. Nevertheless, the complexity of clinical-grade lentiviral vector production has been a barrier to the approach and driven up the time and money required to make the therapies.

The researchers at GSK and Orchard shared a look at a way to address the barrier last year. The idea is to introduce all the required lentiviral vector components into the cell at once rather than implement a sequential series of gene transfer steps. Orchard perceives significant advantages to the approach.

The breast cancer cells evade immune defences

Immune cells use a protein on their surface called CCR2 to direct them to sites of inflammation and tumors. However, the receptor is also expressed by breast cancer cells, and its functions are not precise.

However, now, the scientists at Cold Spring Harbor Laboratory have found that breast cancer cells can hijack CCR2 to do a different job—assisting them to evade detection by the immune system as published in the Journal of Experimental Medicine.

Comprehending the immune suppressive role of CCR2 signalling in cancer cells could open up new sites for developing immuno-oncology therapies as the researchers believe.

In a mouse model of breast cancer, a loss of even one piece of the gene encoding for CCR2 in cancer cells considerably decreased tumour growth rates, the Cold Spring Harbor researchers found. They further observed the biological process behind this difference.

Glympse Bio secures USD 46 Million to boost its injectable diagnostics for NASH, cancer and more

Glympse Bio, developers of an injectable chemical biomarker platform for diagnosing the body’s hidden diseases, has raised USD 46.7 million in new funding for its efforts.

This includes tests targeted at non-alcoholic steatohepatitis—a severe form of fatty liver disease known as NASH that can lead to tissue scarring, organ failure and cancer—and other targets in oncology and new programs in infectious diseases.

The company had presented preclinical data of its technology’s uses in NASH, presenting it could help detect the disease early, as well as monitor its progression and the effects of drug therapies in animal models.

The liver test of Glympse begins with a mixture of engineered particles, which interact with the biological mechanisms of disease as they pass through the body. If certain enzymes and reactions linked to NASH are present, they will leave their mark on the injected particles that will then carry the diagnostic signal out of the body when ejected through the urine.

The kidney failure med of Mallinckrodt squeaks by FDA panel in 8-7 vote

The FDA staffers lately flagged multiple concerns around terlipressin of Mallinckrodt for a type of kidney failure, including if meeting the surrogate endpoint in its phase 3 study translated to patients living longer or faring better. Still, the FDA panel narrowly supported its approval in an 8-7 vote.

The panel vote is not binding. However, the FDA has the predisposition to follow committee recommendations. A recent exception is volanesorsen of Akcea Therapeutics, which the agency rejected after an advisory panel voted with a wider margin in favour of its approval.

Mallinckrodt is seeking a green signal in hepatorenal syndrome type 1 (HRS-1), a quickly progressing kidney disease, which affects people with cirrhosis. It can lead to life-threatening kidney failure within days and can only be cured with a liver transplant that addresses the liver disease and its effects on kidney function.