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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Fierce Pharma

| Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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After delay, Bluebird submits sickle cell gene therapy for FDA approval

Bio Pharma Dive

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

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FDA approves bluebird's sickle cell disease gene therapy. Can Lyfgenia overcome CRISPR’s halo?

Fierce Pharma

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival (..)

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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FDA approves first gene therapies for sickle cell disease (with a warning)

Drug Discovery World

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Vertex, CRISPR prep filing for gene-editing blood disorder therapy

pharmaphorum

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Commercialization of novel gene-editing technology in beta-thalassemia

pharmaphorum

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. The move marks a continuation in its ongoing commitment to exploring gene-editing technology. DOWNLOAD THE FULL ARTICLE HERE.