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FDA Approves Merck’s ENFLONSIA™ to Prevent RSV in Infants

The Pharma Data

FDA Approves Merck’s ENFLONSIA™ to Protect Infants from Severe RSV Illness Merck operating as MSD outside the United States and Canada, has received a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).

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A Visual Guide to Gene Delivery

Codon

For the former, clinicians extract a patient’s cells, engineer them in a laboratory, and then infuse them back into the body. Some diseases are caused by mutations in large genes that exceed the packaging limits of existing vectors like adeno-associated virus (AAV). Vectors also differ in the duration of their expression.

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Prime editing treats childhood brain disease in mice

Broad Institute

& Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, to correct the root cause of AHC in mice. There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.&

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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.

DNA
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Levers for Biological Progress

Codon

” Nobody really knows without trying it out in the laboratory. For one, life itself grows and develops slowly, and the many steps required to “do” biological research in the laboratory are often tedious and manual. Even in vitro experiments — or those done in the laboratory — are relatively slow because E.

DNA
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FDA Approves Veklury (remdesivir) for the Treatment of COVID-19

The Pharma Data

FDA Approves Veklury (remdesivir) for the Treatment of COVID-19. Food and Drug Administration (FDA) has approved the antiviral drug Veklury (remdesivir) for the treatment of patients with COVID-19 requiring hospitalization. The speed and rigor with which Veklury has been developed and approved in the U.S.

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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases. Labcompare.