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The challenges and trends of cell & gene therapies 

Drug Discovery World

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

Therapies 130
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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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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Current insights and molecular docking studies of HIV?1 reverse transcriptase inhibitors

Chemical Biology and Drug Design

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. million people worldwide were living with HIV in 2021. Molecular insights of HIV Reverse transcriptase and it's inhibitors.

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Gilead Sciences Announces Second Quarter 2021 Financial Results

The Pharma Data

econd Quarter 2021 Product Sales Increased 21% Year-Over-Year Primarily Driven by Veklury. Nasdaq: GILD) announced today its results of operations for the second quarter 2021. “We Second Quarter 2021 Financial Results. Total second quarter 2021 revenue of $6.2 Total second quarter 2021 revenue of $6.2

Science 52
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Meet the Researcher: Liam Tremble

Drug Discovery World

I was part of the team responsible for the spin out of Poolbeg from hVIVO plc in 2021 and it has been a privilege to adopt the role of lead scientist on a number of our clinical and pre-clinical programmes. The highlight of my career has undoubtedly been taking on the role of Principal Scientist at Poolbeg Pharma.

Research 221
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SIGA’s monkeypox treatment gets UK approval  

Drug Discovery World

Pharmaceutical company SIGA Technologies has announced that its oral therapy tecovirimat has been approved in the UK for the treatment of smallpox, monkeypox, cowpox. . The therapy has also been approved for the treatment of complications following vaccination against smallpox in adults and children with a body weight of at least 13kg. .

Treatment 130