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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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How Centralized IBC Review Can Benefit Gene Therapy Research

Advarra

In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. As of June 2023, 416 Phase III studies are listed, representing a growing pipeline of gene therapy products preparing to undergo consideration for FDA approval. Gene therapy research is booming in the clinical setting.

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Metabolism of macrocyclic drugs

Metabolite Tales Blog

Metabolism of de novo-designed macrocyclic drugs approved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDA approved drugs are macrocycles [1]. link] Take a look at our other blogs The post Metabolism of macrocyclic drugs appeared first on Hypha Discovery. Ayushi Jain and Khalid Mumtaz, 2022.

Drugs 52
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Metabolism of de novo designed macrocyclic drugs

Metabolite Tales Blog

Metabolism of de novo-designed macrocyclic drugs approved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDA approved drugs are macrocycles [1]. link] Take a look at our other blogs The post Metabolism of de novo designed macrocyclic drugs appeared first on Hypha Discovery. Avicenna J Med 12:154–156.

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DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

NIH Director's Blog: Drug Discovery

Golub and colleagues produced the library by using a virus to insert a distinct DNA barcode, just 24 nucleotides long, stably into the genomes of each genetically distinct tumor cell line. The approach might also be put to use for optimizing drug candidates or for discovering new uses for cancer drugs that have already been FDA approved.

DNA 52
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High Content Imaging Solutions Aid in Screen for Zika Virus Inhibitors

PerkinElmer

Although the novel coronavirus is the subject of nearly every media story right now, several years ago it was the Zika virus (ZIKV) gathering a great deal of attention. Here, we describe the novel use of high content imaging solutions (HCS) in a study to identify possible Zika virus inhibitors that could be translated into therapeutic form.

Virus 52
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Use of Ultra-High-Throughput Screening in Discovery of COVID-19 Virus Structure

PerkinElmer

The virus, which can cause a severe form of pneumonia and lead to acute respiratory distress, currently has no FDA-approved targeted therapeutic or vaccine. For this reason, researchers around the globe are scrambling to understand this novel virus and figure out its potentially targetable vulnerabilities.

Virus 52