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Casgevy

All articles tagged with #casgevy

health2 years ago

"FDA Expands CRISPR Gene-Editing Approval for Beta Thalassemia and Second Disease"

The FDA has expanded its approval of the CRISPR-based medicine Casgevy to include the treatment of beta thalassemia, an inherited blood disorder, following its initial approval for sickle cell disease in December. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the therapy's expanded approval comes ahead of the FDA's decision deadline and is seen as a promising development for patients with thalassemia.

via STAT|
#beta-thalassemia#casgevy#crispr
health-and-medicine2 years ago

"Revolutionary CRISPR Gene-Editing Breakthrough Offers New Hope for Sickle Cell Treatment"

The FDA has approved a new gene-editing treatment called Casgevy for sickle cell disease, which uses CRISPR technology to edit DNA in adult stem cells. This treatment works by turning off an inhibitor of fetal hemoglobin production, allowing the body to produce hemoglobin that can effectively carry oxygen, unlike the mutated form in sickle cell patients. The approval marks a significant advancement in gene therapy, offering a potential cure for sickle cell disease while adhering to ethical standards by only affecting the treated individual and not future generations.

via WORLD News Group|
##casgevy#crispr
health-and-medicine2 years ago

"CRISPR Faces Regulatory Delays, Approval Timeline Uncertain"

The FDA's recent approval of Casgevy, a CRISPR gene-editing therapy for sickle cell anemia, marks a historic milestone, but a report by GlobalData suggests that further approvals for similar CRISPR-based drugs are not expected soon. With only 24 CRISPR drugs in Phase 2 trials and the majority in early-stage development, the pipeline for these therapies is relatively immature. CRISPR Therapeutics' CTX-110 is identified as the next potential candidate for approval, possibly by the end of 2025, but it faces uncertain prospects. The company has shifted focus to other therapies, indicating ongoing development and evaluation within the CRISPR drug landscape.

via Seeking Alpha|
#casgevy#clinical-trials#crispr
health2 years ago

FDA Approves CRISPR Gene-Editing for Sickle Cell Disease Treatment

The US Food and Drug Administration (FDA) has approved two groundbreaking gene-editing treatments, Casgevy and Lyfgenia, for sickle cell disease. Casgevy, based on the CRISPR gene editing tool, targets the problematic gene in a patient's bone marrow stem cells, resulting in the production of properly functioning hemoglobin. Lyfgenia utilizes a harmless virus to insert a gene into patients' stem cells. Sickle cell disease is a rare and life-threatening blood disorder that primarily affects people of African or Caribbean descent. The approval of these treatments offers hope for individuals with sickle cell disease, who previously had limited treatment options.

via DW (English)|
#casgevy#crispr#gene-editing
health2 years ago

"Breakthrough Gene-Editing Treatment Offers Hope for Sickle Cell Disease Patients"

The U.S. Food and Drug Administration has approved the first gene-editing treatment, Casgevy, for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy uses CRISPR technology to edit a person's genes and turn on fetal hemoglobin, helping red blood cells maintain their healthy shape. Clinical trials have shown that Casgevy eliminates pain crises in most patients. However, challenges remain in reaching the thousands of people who could benefit from the treatment, including potential cost barriers and the complexity of administering the therapy. The FDA has also approved a separate gene therapy by Bluebird Bio, called Lyfgenia, for the treatment of sickle cell disease.

via CNBC|
#casgevy#crispr#fda
health2 years ago

FDA Approves Groundbreaking Gene Therapies for Sickle Cell Disease, Revolutionizing Treatment Options

The FDA has approved two groundbreaking gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD) in patients aged 12 and older. Casgevy is the first FDA-approved therapy to use CRISPR/Cas9 genome editing technology, while Lyfgenia utilizes a lentiviral vector for genetic modification. Both therapies involve modifying the patient's own blood stem cells and delivering them back as a one-time infusion. These treatments aim to increase the production of fetal hemoglobin or introduce a gene-therapy derived hemoglobin to prevent the sickling of red blood cells. The approvals mark a significant advancement in gene therapy and offer hope for individuals with SCD, a rare and debilitating blood disorder.

via FDA.gov|
#casgevy#fda#gene-therapy
health2 years ago

"Vertex and CRISPR's Gene-Editing Therapy Nears FDA Approval, Paving the Way for a Patent Battle"

The gene-editing therapy Casgevy, developed by Vertex and CRISPR Therapeutics, is expected to receive FDA approval for the treatment of severe cases of sickle cell disease. Casgevy would be the first gene-editing therapy authorized by US regulators and uses the CRISPR tool. In clinical trials, Casgevy demonstrated remarkable results, completely relieving 29 out of 30 sickle cell patients of debilitating pain episodes for at least one year. However, the expected high price tag raises concerns about equity in medicine. While Casgevy offers hope for a potential cure, ethical concerns and access to the treatment remain important considerations.

via The Boston Globe|
#casgevy#crispr#fda-approval
health2 years ago

"Breakthrough: CRISPR Therapy Approved, Revolutionizing Medical Treatment"

The world's first CRISPR gene-editing therapy, Casgevy, has received regulatory approval from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to treat sickle cell disease and transfusion-dependent beta-thalassemia. The treatment involves editing a gene called BCL11A to enable the production of fetal hemoglobin, which is crucial for patients with these blood disorders. While the therapy has shown promising results in clinical trials, concerns remain about its long-term safety and affordability. Casgevy is currently under review by other regulatory bodies, including the FDA, and its availability will depend on its cost. Other CRISPR therapies, such as base editing and prime editing, are also being developed for various inherited diseases.

via Livescience.com|
#beta-thalassemia#casgevy#crispr