UK authorises gene therapy for blood disorders in world first

Crispr Therapeutics AG

16/11/2023 08:17

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.
    Sickle cell disease and β-thalassemia are genetic conditions
caused by errors in the genes for haemoglobin, which is used by
red blood cells to carry oxygen around the body. 
    "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.
    The MHRA said no significant safety concerns were identified
during the trials, adding that it was closely monitoring the
safety of the medicine.
    The medicine is administered by taking stem cells out of a
patient’s bone marrow and editing a gene in the cells in a
laboratory, with the modified cells then infused back into the
patient after conditioning treatment to prepare the bone marrow.
    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.

 (Reporting by Muvija M; editing by Sarah Young)
 ((Muvija.M@thomsonreuters.com; within the UK: +44 7776813338,
outside the UK: +91 80 61822698; Twitter: https://twitter.com/muvija_m;))