Britain has authorised a gene therapy that aims to cure sickle-cell disease and another type of inherited blood disorder for patients aged 12 and over, the country's medical regulator said on Thursday, becoming the first in the world to do so.
LONDON, Nov 16 (Reuters) - Britain has authorised a gene therapy that aims to cure sickle-cell disease and another type of inherited blood disorder for patients aged 12 and over, the country’s medical regulator said on Thursday, becoming the first in the world to do so.
Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.
“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal,” MHRA Interim Director Julian Beach said in the statement.
In clinical trials Casgevy has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β -thalassaemia, relieving the symptoms of disease, Beach added.
U.S.-based companies Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN) welcomed the approval in a separate statement.
“I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said CRISPR CEO Samarth Kulkarni said.
The original article contains 273 words, the summary contains 179 words. Saved 34%. I’m a bot and I’m open source!
This is the best summary I could come up with:
LONDON, Nov 16 (Reuters) - Britain has authorised a gene therapy that aims to cure sickle-cell disease and another type of inherited blood disorder for patients aged 12 and over, the country’s medical regulator said on Thursday, becoming the first in the world to do so.
Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.
“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal,” MHRA Interim Director Julian Beach said in the statement.
In clinical trials Casgevy has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β -thalassaemia, relieving the symptoms of disease, Beach added.
U.S.-based companies Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN) welcomed the approval in a separate statement.
“I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said CRISPR CEO Samarth Kulkarni said.
The original article contains 273 words, the summary contains 179 words. Saved 34%. I’m a bot and I’m open source!