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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

s specific mutation, creating a mouse model of the disease, determining the optimal base editor, performing extensive safety analyses, working with Danaher to manufacture the therapeutic, conducting toxicity studies, and securing FDA approval for the trial. This unprecedented feat required diagnosing K.J.’s

Treatment 133
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Regeneron Reports Third Quarter 2020 Financial and Operating Results

The Pharma Data

FDA accepted for priority review Libtayo ® (cemiplimab-rwlc) for both advanced non-small cell lung cancer and basal cell carcinoma. FDA approved Inmazeb for Ebola ( Zaire ebolavirus). REGN-COV2 trial in the COVID-19 outpatient setting met primary and key secondary endpoints. Regeneron Pharmaceuticals, Inc.

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Beyond Steel Tanks

Codon

In the case of semaglutide, those cells are Saccharomyces cerevisiae— also known as Baker’s yeast — engineered to secrete a peptide precursor that is later purified, chemically modified, packaged into an injectable or tablet form, and then shipped around the world. Continuing this method may not scale.

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Emergent BioSolutions Announces 2021 Financial Guidance, Provides Preliminary 2020 Results

The Pharma Data

CDMO Services assumes continued growth in Development Services (DVS), Drug Substance (DS) manufacturing, and Drug Product (DP) manufacturing and Packaging for both clinical- and commercial-stage projects on behalf of a growing list of pharmaceutical and biotechnology innovators and government/NGO customers. FOOTNOTES. (1)

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Making a “Miracle” HIV Medicine

Codon

Because of PEPFAR’s effectiveness, the United Nations (UN) set an ambitious goal in 2022 to end HIV transmission and eliminate AIDS-related deaths by 2030. Zidovudine showed promise against multiple HIV strains in cultured cells, and the Food and Drug Administration (FDA) approved it for human studies within five months.