Delivery of Macromolecular Therapeutics: Tackling a Billion Year Old Barrier

December 11th, 2019|Categories: Past Webinars|

Presenter: Steven Dowdy (UCSD School of Medicine) Date: December 17, 2019 Description: All macromolecular therapeutics, including ASOs, siRNAs, peptides, proteins, CRISPR, mRNA and non-viral DNA vectors, are taken up into cells by endocytosis. However, <1% to none of the endocytosed therapeutic cargo escapes into the cytoplasm ...

Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.

November 30th, 2019|Categories: Ago/siRNA/saRNA, Clinical Development, Hot Papers|

GonzĂ¡lez-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, ConceiĂ§Ă£o IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. J Neurol. 2019 Nov 14. PubMed ...

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