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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 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.

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Recent Advances in Gene Therapy

DrugBank

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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

The Pharma Data

This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Safety Profile and Precautions Like all biologic therapies, ENFLONSIA comes with safety considerations.

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Grifols’ Fibrinogen Phase 3 Results Published in eClinicalMedicine

The Pharma Data

Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).

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The Altascientist Issue 36: Navigating the Key Considerations of Your Nonclinical Cell and Gene Therapy Studies

Alta Sciences

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?

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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.