FDA approves first-ever US use of CRISPR gene-editing treatment for sickle cell disease

[Commissar SFLUFAN]

U.S. approves first gene-editing treatment, Casgevy, for sickle cell disease

WWW.CNBC.COM

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Casgevy uses the Nobel Prize-winning technology CRISPR to treat sickle cell disease, a blood disorder that affects about 100,000 Americans.

 

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The U.S. Food and Drug Administration on Friday approved the country’s first gene-editing treatment, Casgevy, for use in patients with sickle cell disease.

 

The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the treatment could be challenging given the potential hurdles — including cost — of administering the complex therapy.

 

Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’s genes to treat disease. The treatment was approved by U.K. regulators last month.

 

Sickle cell, an inherited blood disorder, causes red blood cells to become misshapen half moons that get stuck inside blood vessels, restricting blood flow and causing what are known as pain crises. About 100,000 Americans are estimated to have the disease.

 

Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients.

 

The FDA approved the treatment for people 12 years and older.

 

 

[Kal-El814]

So rad. Now make a therapy that eliminates the possibility for me to get sunburned or tick borne illnesses and I will be unstoppable.

[DarkStar189]

57 minutes ago, Kal-El814 said:

So rad. Now make a therapy that eliminates the possibility for me to get sunburned or tick borne illnesses and I will be unstoppable.

Tick borne illnesses can fuck right off. I’ve had knee problems for close to 2 years and finally found out a few months ago it was Lyme disease. Did 2 months of oral antibiotics and I’m still not happy with where I’m at. Might need to see a specialist or give it more time.

[Kal-El814]

18 minutes ago, DarkStar189 said:

Tick borne illnesses can fuck right off. I’ve had knee problems for close to 2 years and finally found out a few months ago it was Lyme disease. Did 2 months of oral antibiotics and I’m still not happy with where I’m at. Might need to see a specialist or give it more time.

 

There should be a newer Lyme vaccine coming out soon, which is great news especially as the globe continues to cook.

[legend]

4 hours ago, Kal-El814 said:

 

There should be a newer Lyme vaccine coming out soon, which is great news especially as the globe continues to cook.

 

 

My father in law gets Lyme disease at a rate that feels biweekly, so this would be awesome.

[Kal-El814]

1 hour ago, legend said:

 

 

My father in law gets Lyme disease at a rate that feels biweekly, so this would be awesome.

 

Indeed. There was one back in the 80's / 90's (can't remember when specifically) that apparently didn't find a big enough market. Gonna be an ongoing, bigger problem since tick season is gonna be longer and longer.