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Novartis and Precision BioSciences announce in vivo gene editing collaboration

The companies will work together to develop potentially curative treatments for disorders including sickle cell disease and beta thalassaemia


Novartis and Precision BioSciences (Precision) have announced an in vivo gene editing research and development collaboration to develop potentially curative treatments for disorders including sickle cell disease (SCD) and beta thalassaemia.

The clinical stage editing company will receive $74m upfront from Novartis to kick-start the collaboration, and is eligible to receive an additional $1.4bn in additional payments for future milestones.

As part of the agreement, Precision will develop a custom ARCUS nuclease designed to insert, in vivo, a therapeutic transgene at a “safe harbour” location in the genome as a potential one-time transformative treatment option for diseases including certain haemoglobinopathies such as SCD and beta thalassaemia.

Widely used ex vivo therapeutic gene-editing techniques such as CRISPR/Cas9 require cells to be removed from the body, edited and reinserted, in a lengthy, expensive and potentially hazardous process. Novartis hopes to develop a simpler, off-the-shelf treatment.

Commenting on the collaboration, Jay Bradner, president of the Novartis Institutes for Biomedical Research (NIBR), said: "We identify here a collaborative opportunity to imagine a unique therapeutic option for patients with haemoglobinopathies, such as SCD and beta thalassaemia – a potential one-time treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies,”

SCD is a life-long, incurable genetic illness causing red blood cells to take a distinct crescent shape which can block blood vessels and affect the way oxygen is carried around the body. This can cause problems including delayed growth, organ failure and chronic pain, known as sickle cell crises, and affects 20 million people worldwide.

“The in vivo gene editing approach that we are pursuing for SCD could have a number of significant advantages over other ex vivo gene therapies currently in development,” said Derek Jantz, chief scientific officer and co-founder of Precision. “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option.”

One in 100,000 people are affected by symptomatic cases of beta thalassaemia, a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin. Patients with the most severe form of the disease develop life-threatening anaemia and have to undergo regular blood transfusions.

Novartis will have an exclusive licence for further development of the custom ARCUS nuclease, taking charge of all subsequent research, development, manufacturing and commercialisation.

Article by
Emily Kimber

24th June 2022

From: Research, Healthcare



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