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Beta Thalassemia

All articles tagged with #beta thalassemia

health-and-pharmaceuticals2 years ago•1 min saved

"CRISPR Therapy Gains Second FDA Approval for Gene Editing"

The FDA approved Crispr Therapeutics' drug, Casgevy, developed with Vertex Pharmaceuticals, for patients age 12 and older with beta thalassemia, marking the second gene-editing approval for Crispr. The drug was expected to be approved in March but gained early approval, following its approval for sickle cell disease last year. While CRSP stock slipped, VRTX stock rose, with Vertex's stock surging since mid-December. CRSP stock has a strong Relative Strength Rating, and MarketSmith.com shows it sitting on a floor at its 50-day moving average.

via Investor's Business Daily|

#beta-thalassemia #crispr #fda-approval

health2 years ago•0 min saved

"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

healthcare2 years ago•0 min saved

"FDA Approves CRISPR Gene-Editing Therapy for Beta-Thalassemia and Sickle Cell Disease"

The FDA has approved Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy, a gene editing therapy, to treat beta-thalassemia, marking the first-ever CRISPR-edited therapy for the rare genetic blood disorder. Priced at $2.2 million for the one-time ex vivo treatment, the approval is for patients 12 years and older who require regular blood transfusions to survive, and it will compete with a gene-edited therapy from startup bluebird bio.

via Endpoints News|

#beta-thalassemia #crispr-therapeutics #fda

health2 years ago•6 min saved

"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

health2 years ago•3 min saved

U.K. Approves Groundbreaking Gene Therapy for Sickle-Cell Disease

The U.K. has approved the first treatment derived from CRISPR, a gene-editing method, for sickle-cell disease and beta thalassemia. The treatment, called Casgevy, is expected to be approved by the FDA in the United States next month. The manufacturers, Vertex Pharmaceuticals and CRISPR Therapeutics, anticipate that about 2,000 patients in the U.K. will be eligible for the treatment. Sickle-cell disease affects around 100,000 Americans, mostly Black and Hispanic individuals, and causes episodes of extreme pain. The treatment relies on CRISPR to activate a gene that produces an alternative form of hemoglobin. However, the treatment is onerous and expensive, requiring intense chemotherapy and hospital stays. The price in the U.S. is expected to be millions of dollars per patient.

via The New York Times|

#beta-thalassemia #crispr #gene-editing

health2 years ago•6 min saved

Revolutionary Gene Therapy: A Fairytale Cure for Children's Blood Disease

Two girls with beta thalassemia, a deadly blood disease, have become the first patients on the West Coast to receive a pioneering gene therapy offered by UCSF Benioff Children's Hospital Oakland. The therapy, called Zynteglo, uses an engineered virus to deliver a healthy gene into patient cells, aiming to permanently cure the disease. The treatment has shown promising results, with one of the girls already feeling better after 10 weeks. The therapy offers hope for an estimated 1,300 to 1,500 patients with beta thalassemia and opens up possibilities for treating other inherited disorders caused by single gene mutations. However, the treatment is expensive, costing $2.8 million for a single-use vial.

via The Mercury News|

#beta-thalassemia #gene-therapy #health

healthcare2 years ago•1 min saved

Price Battles Jeopardize Access to Gene Therapies in Europe

Gene therapy makers are concerned about the profitability of their treatments in Europe, while patients fear being left behind. Bluebird Bio's gene therapy, Zynteglo, was approved in Europe in 2019 for the treatment of beta thalassemia. The therapy has shown promising results in clinical trials, offering the potential for a life free of blood transfusions. However, the high cost of gene therapies and uncertainties surrounding reimbursement have raised concerns about patient access and the financial viability of these treatments in Europe.

via STAT|

#beta-thalassemia #european-approval #gene-therapy

health2 years ago•3 min saved

CRISPR Gene-Editing Drug Shows Promise for Sickle Cell and Thalassemia Treatment

The FDA has indicated that it could approve Crispr Therapeutics' gene-edited sickle cell treatment, exa-cel, as early as December. The drug works by increasing hemoglobin levels in patients with sickle cell disease and beta thalassemia. If approved, exa-cel would be the first drug using CRISPR-based gene editing to hit the market. The companies are also working to file for approval in Europe and the U.K.

via Investor's Business Daily|

#beta-thalassemia #crispr #fda