Pharmaceutical Technology

JULY 29, 2022

In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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RNA Gene Expression Gene Gene Therapy 245

The Pharma Data

JULY 12, 2021

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. “Academic labs and pharmaceutical and? Nature Communications, 2021.

Drugs 52

Drugs Gene Expression Big Data Gene 52

The Pharma Data

OCTOBER 15, 2020

“For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”. As with dry AMD, there is no FDA-approved therapy. “We Source link.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

Roots Analysis

JANUARY 14, 2024

Given the ongoing scientific advancements and the rise of FDA-approved biologics, the pharmaceutical industry seems to be approaching the era of biologics. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Protein Manufacturing Production 40

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Therapeutics.

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Life Science Vaccination Vaccine Gene 111