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Actio, a precision medicine startup, launches with $55M and a mouse lab partnership

BioPharma Drive: Drug Pricing

The two-year-old San Diego biotech is allied with The Jackson Laboratory to engineer better mouse models of the rare diseases it plans to target.

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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease 142
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David Liu receives Breakthrough Prize in Life Sciences

Broad Institute

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

Science 138
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Scientists reveal cellular changes unique to early Alzheimer’s disease

Broad Institute

By Allessandra DiCorato September 28, 2023 Credit: Strittmatter Laboratory, Yale University Amyloid beta clusters (red) builds up among neurons (green) in a mouse model of Alzheimer’s disease. Neurons are thought to produce the plaque-forming protein called amyloid beta, and the researchers found evidence for this in their data.

Disease 144
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Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

Therapies 116
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Drug Discovery Today publishes editorial on new approaches to the discovery of novel agents against 2019-nCoV (COVID - 19)

Drug Discovery Today

Drug Discovery Today has today published a pre print of an editorial by Yongtao Duan, Hai-Liang Zhu and Chongchen Zhou of Henan Provincial Key Laboratory of Children’s Genetics and Metabolic Diseases, Children’s Hospital Affiliated to Zhengzhou University, Zhengzhou, 450018, China

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Discovery and development of COVID?19 vaccine from laboratory to clinic

Chemical Biology and Drug Design

SARS-CoV-2 is responsible for the coronavirus disease of 2019 (COVID-19) which is one of the most widespread and powerful infections affecting human lungs. WHO proclaimed the outbreak of the Ebola virus disease (EVD), in 2014 that killed hundreds of people in West Africa.

Vaccine 100