FDA approves cure for sickle cell disease, the first treatment to use gene-editing tool CRISPR

searcher · Dec 8, 2023

FDA approves cure for sickle cell disease, the first treatment to use gene-editing tool CRISPR

The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.

The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in chemistry in 2020.

“I think this is a pivotal moment in the field,” said Dr. Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex. “It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product.”

More:

FDA approves cure for sickle cell disease, the first treatment to use CRISPR

The groundbreaking approval has been eagerly anticipated by patients and doctors alike. The treatment is priced at $2.2 million.

www.nbcnews.com

pmbug · Dec 8, 2023

Some old notes about CRISPR (caveat emptor) from June 2018:

Editing cells’ genomes with CRISPR-Cas9 might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn — a potential game-changer for the companies developing CRISPR-based therapies.

In the studies, published in Nature Medicine, scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to seed tumors inside a patient. That could make some CRISPR’d cells ticking time bombs, according to researchers from Sweden’s Karolinska Institute and, separately, Novartis.
...

A serious new hurdle for CRISPR: Edited cells might cause cancer, two studies find

Scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to seed tumors inside a patient.

www.statnews.com

A study at Karolinska Institutet in Sweden has revealed that CRISPR-Cas9 gene editing could favor mutations in the most common cancer-causing gene. What does it mean for the first CRISPR therapy trials in humans?

CRISPR-Cas9 has taken the life sciences field by storm, making gene editing simpler and faster than ever. Although CRISPR-Cas9 gene editing was only first described in 2012, the first human trials using CRISPR as a therapy were started in China last year and Europe is due to run the first one later this year. It seems like we might have rushed into it too soon, as a new study points out that therapies using this popular gene editing tool could inadvertently cause cancer.

The research, published today in Nature Medicine, describes that the use of CRISPR-Cas9 in human cells can activate the protein p53. This protein is involved in the repair of DNA breaks and can interfere with the efficiency of CRISPR to cut DNA.

This means that when selecting cells whose DNA has been correctly modified using CRISPR, those where p53 is mutated and therefore inactive will be more likely to be selected. Given that p53 mutations are the most common genetic alteration in cancer, the process used to modify and select CRISPR-edited cells could be favoring the selection of potentially cancerous cells.
...

Scientists Warn CRISPR Therapy Could Cause Cancer as First Human Trials Take Place

A study at Karolinska Institutet in Sweden has revealed that CRISPR-Cas9 gene editing could favor mutations in the most common cancer-causing gene. What does it mean for the first CRISPR therapy trials in humans?

www.labiotech.eu

Goldhedge · Dec 8, 2023

The first treatment...? What was the jab all about anyway....?

an aside...

How do they reintroduce the altered gene into ones body? There are billions of genes to 'alter'... A shot in the arm? IV?

FDA approves cure for sickle cell disease, the first treatment to use gene-editing tool CRISPR

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