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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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Enhancing gene therapy with Circio

Drug Target Review

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

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Radioligand therapy pipeline with in-license for compounds targeting Fibroblast Activation Protein (FAP)

The Pharma Data

Broad expression of FAP demonstrated in tumors or in tumor stroma across many solid tumors 1 ,2 ,3. Novartis Oncology continues to reimagine cancer care through development of robust radioligand therapy portfolio. We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology.

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The Future of Drug Discovery: Tackling the Undruggable with New Biotechnologies

DrugBank

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

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Addressing increasingly resistant drugs by infectious agents

Drug Target Review

10 Reported cAMP as the second messenger signalling molecule conserved from bacteria to humans and modulates several biological processes, including protein expression, gene transcription, and cell development and differentiation. In this assay, active compounds are discovered under physiologically relevant conditions.

Drugs 111
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The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

Conversations in Drug Development Trends

Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness. Fortunately, personalized treatment plans help circumvent therapy resistance while optimizing response rate and improving quality of life.

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Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. ” This technology could be used to design protein therapeutics that can bind to, and “shut down,” harmful or misfolded proteins in living cells.