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Levers for Biological Progress

Codon

More specifically, it reveals that the price to sequence a human genome fell from $100 million in 2001 to $700 by 2021; a stunning collapse in price. Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease. The cost of sequencing a human genome.

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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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Improved prime editing system makes gene-sized edits in human cells at therapeutic levels

Broad Institute

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene. Tags: Gene editing Gene therapy David Liu Nature Biomedical Engineering.

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ViGeneron signs global collaboration agreement for ophthalmic gene therapy development

The Pharma Data

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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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.

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Neurophth and AAVnerGene Enter Strategic AAV Capsids Partnership for Next-Generation Ophthalmic Gene Therapy

The Pharma Data

Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. 19, 2021 /PRNewswire/ — Neurophth Therapeutics, Inc. ,