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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

In 2018, Liu, who works on the same floor as Minikel and Vallabh at Broad, approached them and proposed a collaboration. Building on previous work by the vector-engineering lab of Ben Deverman at the Broad, the team developed a pair of adeno-associated viruses (AAVs) to package and deliver the base-editing machinery to brain cells.

Disease 144
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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The molecular machinery of the epigenetic system can selectively package specific regions of DNA away, making them inaccessible and less active. This platform opens the door to treating complex multigenic diseases, effective tissue engineering, and fine-tuning immune cell differentiation. 2018;:15–25. Nature Communications.

DNA 105
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Takeda Manufacturing Facilities in Japan and Ireland Recognized With Category Awards for 2021 Facilities of the Year I Takeda

The Pharma Data

OSAKA, Japan, April 28, 2021 – Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it was awarded by the International Society for Pharmaceutical Engineering (ISPE) for the 2021 Facility Of the Year Awards (FOYA) in two categories. About the ISPE Facility of the Year Awards Program.

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Mastering Responses to FDA 510(k) AI Letters: A Strategic Approach

FDA Law Blog: Biosimilars

Performance Metric FY 2018 FY 2019 FY 2020 FY 2021 FY 2022 Average Number of FDA Days to MDUFA IV Decision 72.62 Team meetings with subject matter experts can be scheduled to discuss response strategies for deficiencies that require extensive clinical, scientific, or engineering input. 510(k) Time to MDUFA IV Decision.

FDA 75
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Commercial interests contribute to drug use and addiction

National Institute on Drug Abuse: Nora's Blog

Cannabis products are often sold in colorful packages that mimic kid-friendly snack foods, for example, making them appealing to children and to young people. A new analysis by the National Academies of Sciences, Engineering, and Medicine found that early legalization efforts have prioritized economic outcomes over public health.

Drugs 106
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The Book of Nimbus

LifeSciVC

Fourteen years on, this corporate experiment has gone far beyond the initial idea, and has established an R&D engine more effective than most big pharma R&D groups at producing best-in-class small molecules against targets that matter in human disease biology. Would we wind down and exit, or chart some different path?

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Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

The Pharma Data

2018) PLoS One. Horsepox and vaccinia are closely related orthopoxviruses that are believed to share a common ancestor. Horsepox-based vaccines are designed to be single dose, vial-sparing vaccines, which can be manufactured on conventional cell culturing systems, with the potential for mass scale production. 1 Noyce RS, et al.

Vaccine 40