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A Visual Guide to Gene Delivery

Codon

Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream. This changed with the discovery of AAVs.

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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.

DNA
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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. Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs.

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FDA Approves First Over-the-Counter Fully At-Home Test for COVID-19

The Pharma Data

FDA Approves First Over-the-Counter Fully At-Home Test for COVID-19. Last week, the FDA approved a different at-home test, but it requires samples to be mailed to a lab to get the results. Last week, the FDA approved a different at-home test, but it requires samples to be mailed to a lab to get the results.

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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.

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Oncorus Pivots to Scale Up Manufacturing with New GMP Facility

The Pharma Data

Process development and quality control will begin in 2021, and full manufacturing capabilities at the 88,000-square-foot facility will be available in 2023. In addition to its lead program, which began Phase I testing in 2020, the company expects to nominate three clinical candidates in 2021.

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Pfizer and BioNTech Initiate Rolling Submission of Biologics License Application for U.S. FDA Approval of Their COVID 19 Vaccine

The Pharma Data

The information contained in this release is as of May 7, 2021. For a discussion of these and other risks and uncertainties, see BioNTech’s Annual Report on Form 20-F for the Year Ended December 31, 2020, filed with the SEC on March 30, 2021, which is available on the SEC’s website at www.sec.gov. Pfizer Disclosure Notice.