UK Approves World's First CRISPR-Based Medicine

Britain's drugs regulator has approved a groundbreaking treatment for two painful and debilitating lifelong blood disorders, which works by "editing" the gene that causes them. From a report: The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light for Casgevy to be used to treat sickle cell disease and beta thalassemia. It is the first medicine licensed anywhere that works by deploying gene editing that uses the "genetic scissors," known as CRISPR, for which its inventors won the Nobel prize for chemistry.

Casgevy's developers hope the pioneering treatment could banish the pain, infections and anaemia sickle cell disease brings and the severe anaemia experienced by those with beta thalassemia. About 15,000 people in the UK, almost all of African or Caribbean heritage, have sickle cell disease. About 1,000 -- mainly of Mediterranean, south Asian, south-east Asian and Middle Eastern background -- have beta thalassemia and need regular blood transfusions to treat their anaemia. Experts in the illnesses hope Casgevy may be a cure, making it no longer necessary for people with the conditions to have a bone marrow transplant. Until now this has been the only treatment available, even though the body can reject the donor marrow. The Sickle Cell Society welcomed the MHRA's decision as a "historic moment for the sickle cell community" which "offers [them] newfound hope and optimism."

Re: Cool

[ArmoredDragon]

Way too soon dude, the first step is the eugenics wars, THEN you get that.

Re:

[backslashdot]

Docile Khan can be created with gene editing and thus we can skip the eugenics wars.

Cut, Copy, Paste: The UK's Genetic Text Editor

[Press2ToContinue]

Ah, Britain, once known for conquering lands, now conquering genes with the elegance of a well-crafted Python script. They've swapped their red coats for lab coats, their muskets for molecular scissors. Welcome to the era where 'gene editing' isn't just for those pesky compile errors in your DNA.

Casgevy? Sounds like a software update for your genome. 'Version 2.0: Now with fewer genetic bugs!' This isn't your grandpa's medicine; it's more like applying a hotfix to a legacy system (and we all know how well those usually go).

Remember the days when 'cut and paste' was just for fixing code, not chromosomes? Or when 'debugging' meant something entirely different in the world of genetics? CRISPR's like the GitHub of genetics, but instead of pull requests, we're talking gene requests.

It's fascinating how we've gone from cloning Dolly the sheep to editing human genes. Next up, CRISPR dating apps? 'Swipe right to edit your potential offspring's genome.' Or maybe a subscription model for genetic modifications? 'Sign up for our Platinum DNA package for enhanced memory and muscle tone!'

All jokes aside, this is a big step for medicine, like upgrading from dial-up to fiber optics. But let's be real, if CRISPR was a software release, we'd all be nervously waiting for the patch notes.

When hackers & terrorists get ahold

[Tablizer]

...of this tech, you'll wake up one day with 3 wankers ... on your forehead.

Re:

[Tablizer]

Stoned terrorists want weirder shit.

Why do they have to edit another gene?

[Pinky's Brain]

Why do they have to reactivate fetal hemoglobine instead of fixing the HBB genes directly?

Re:Why do they have to edit another gene?

[wherrera]

CRISPR is more of a cutting tool than a patching tool. There is a much higher risk of off-target mutations with attempting to fix the hemoglobin gene directly in the way you suggest. That risk is much lower if CRISPR is used just to activate or inactivate an existing gene.

Under Review in the United States

[fropenn]

It looks to be under review in the United States as well. The FDA can be very slow, so I am not sure if there is a timeline for possible approval in the United States. But if it is approved it would be very good news for the 100,000 or so people in the United States who have sickle cell disease.

https://www.fda.gov/advisory-committees/advisory-committee-calendar/cellular-tissue-and-gene-therapies-advisory-committee-october-31-2023-meeting-announcement-10312023

Tailored?

[Hodr]

So does this treatment need to be tailored to the individuals DNA, or is this section that needs to be de-activated always accessible in the same location (or by the same detection method or what have you)?

Re:

[yagmot]

First, hematopoietic, or blood-producing, stem cells are collected from patients and sent to a lab, where the actual CRISPR editing is done. After quality testing, the Casgevy-treated stem cells are shipped for use. Before the edited cells can be administered, patients must undergo a preparatory treatment with busulfan, a chemotherapy drug, to obliterate any native stem cells that might remain in their bone marrow. This “conditioning” step is crucial because it provides space in the bone marrow for the functional, CRISPR-edited cells to engraft and grow.

But busulfan, like most chemotherapies, can cause severe side effects, including low levels of infection-fighting white blood cells. For this reason, patients must be hospitalized in sterile conditions. The actual injection of Casgevy-treated cells is quick, but patients must remain in the hospital until their immune system recovers and the risk of serious infection abates.

https://www.statnews.com/2023/... [statnews.com]

Re:

[Hodr]

Yowzers. So not going to be affordable any time soon.

Will this make them susceptible to malaria?

[MobyDisk]

Sickle-cell anemia makes you immune to malaria. Will someone who takes this drug lose that immunity? Or do they get to keep their immunity AND have functioning hemoglobin?

Re:

[fropenn]

Malaria can still be a significant risk to some people with SCD. I don't think anyone would recommend not treating SCD in order to protect against malaria.

https://bmcinfectdis.biomedcentral.com/articles/10.1186/s12879-018-3556-0

Title.

[Motleypuss]

Nice one. Hopefully gene editing can one day be used to fix prematurely-terminating COL2A1. Fingers crossed!