#Casgevy

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#beta-thalassemia
MedCity News
5 months ago
Medicine

Vertex's CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy, Casgevy, has received FDA approval as a treatment for beta thalassemia.
The therapy provides a one-time treatment option for transfusion-dependent beta thalassemia patients age 12 and older. [ more ]
WIRED
7 months ago
Health

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions. [ more ]
WIRED
7 months ago
Health

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions. [ more ]
MedCity News
5 months ago
Medicine

Vertex's CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy, Casgevy, has received FDA approval as a treatment for beta thalassemia.
The therapy provides a one-time treatment option for transfusion-dependent beta thalassemia patients age 12 and older. [ more ]
WIRED
7 months ago
Health

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions. [ more ]
WIRED
7 months ago
Health

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions. [ more ]
morebeta-thalassemia
WIRED
6 months ago
Medicine

The First Crispr Medicine Is Now Approved in the US

Casgevy uses Crispr technology to modify patients' cells and produce healthy hemoglobin.
Casgevy has shown promising results in a clinical trial, with 29 out of 31 patients being free of pain crises for at least a year. [ more ]
MedCity News
7 months ago
Medicine

U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders

The UK has authorized a gene-editing medicine for treating sickle cell disease and beta thalassemia, marking the first approval worldwide for a therapy based on CRISPR technology.
The therapy, known as Casgevy, involves editing a gene in a patient's stem cells to produce high levels of fetal hemoglobin.
Casgevy offers a potentially curative treatment option for these inherited blood disorders, reducing the need for chronic therapies and blood transfusions. [ more ]
www.nytimes.com
7 months ago
Health

Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval

Regulators in Britain have approved the first treatment derived from CRISPR, called Casgevy, for sickle-cell disease and beta thalassemia.
About 2,000 patients in Britain are expected to be eligible for Casgevy treatment.
The FDA is expected to approve Casgevy for sickle-cell patients in the US in early December. [ more ]
www.nytimes.com
7 months ago
Health

Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval

Regulators in Britain have approved the first treatment derived from CRISPR, called Casgevy, for sickle-cell disease and beta thalassemia.
About 2,000 patients in Britain are expected to be eligible for Casgevy treatment.
The FDA is expected to approve Casgevy for sickle-cell patients in the US in early December. [ more ]
#CRISPR gene editing
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
moreCRISPR gene editing
time.com
7 months ago
Medicine

CRISPR Therapy Gets U.K. Approval, the First in the World

Britain's medicines regulator has approved the world's first gene therapy for sickle cell disease, offering hope to thousands of patients.
Casgevy is the first treatment licensed using the gene editing tool CRISPR, and it targets the problematic gene in a patient's bone marrow stem cells.
Both sickle cell disease and thalassemia are caused by genetic mutations that affect the production of hemoglobin in red blood cells. [ more ]
time.com
7 months ago
Medicine

CRISPR Therapy Gets U.K. Approval, the First in the World

Britain's medicines regulator has approved the world's first gene therapy for sickle cell disease, offering hope to thousands of patients.
Casgevy is the first treatment licensed using the gene editing tool CRISPR, and it targets the problematic gene in a patient's bone marrow stem cells.
Both sickle cell disease and thalassemia are caused by genetic mutations that affect the production of hemoglobin in red blood cells. [ more ]
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