The groundbreaking approval has been eagerly anticipated by patients and doctors alike. The treatment is expected to be extremely expensive.
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.”
There are two wolves inside me.
One of them wants to mock every person I’ve ever met who argued nothing will be cured because there’s no money in it.
The other is just jazzed as fuck about CRISPR.
Anyone who says that doesn’t know much about medicine or pharma companies. It’s not like there’s one single pharma company that controls all development and decides no cures. Companies are constantly trying to scoop each other with better or different treatments for things. This is the first crispr based treatment for a genetic disease approved. But we already have gene therapies for multiple other disorders approved and on the market already. And the silly conspiracy totally falls apart when you consider the drugs and things that often get attacked. Like sure lipitor makes a lot of money, but it prevents a huge humber of strokes and heart attacks that are waaaaaay more expensive to treat than just some lipitor. Not to mention vaccines and all our other preventative medicines. And most often something you take every day is just a way safer and easier way to treat something. Could someone do a gene editing thing to change your genes that regulate cholesterol and other lipids? Yeah I suppose it’s feasible. But gene editing and gene therapies are still new and carry a lot of risks. It’s why you see a lot of these gene therapies are getting approved for deadly things like spinal muscular atrophy and sickle cell where the risk is worth it. There’s plenty of evil terrible stuff in the pharmaceutical industry as it is without making up new conspiracies that don’t even make any sense. Alright, end of rant.
Both wolves have to be kinda sad that this CRISPR treatment is millions of dollars for now.
Computers were once the size of warehouses and significantly shittier than my phone.
Time marches on.
So, when will we have grey market gene therapies for myostatin? Asking for SWIM.
