O.24Biodistribution of onasemnogene abeparvovec (AVXS-101) DNA, mRNA, and SMN protein in human tissue

Kaspar, B.; Thomsen, Gretchen M.; Hsieh, Ching Chyuan; Do, Janet; Solano, Steven M.; Chu, Binh T. T.; Barkho, Basam Z.; Fugère, Martin; Kaufmann, Peter; Foust, Kevin D.; Kaspar, Allan; L’Italien, James; Sproule, Douglas M.; Feltner, Douglas E.; Chung, Wendy K.; Burghes, Arthur; McGovern, Vicki L.; Hevner, Robert F.; Conces, Miriam; Mendell, Jerry R.

doi:10.1016/j.nmd.2019.06.307

Cited by 3 publications

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“…In contrast, a gene therapy drug, onasemnogene abeparvovec (brand name, Zolgensma), which is administered via the AAV vectors encoding the normal SMN1 gene, was approved by the FDA in 2019 [ 107 , 108 ]. Onasemnogene abeparvovec can be administered intravenously because the AAV9 vector enables delivery across the blood–brain barrier [ 109 , 110 ]. In general, AAV vector treatment is administered only once in a lifetime because this approach induces neutralizing antibodies against AAV; however, non-clinical data show persistent expression in neurons [ 111 ].…”

Section: Current Status Of Clinical Application Of Nucleic Acid Drugsmentioning

confidence: 99%

Nucleic Acid Drugs—Current Status, Issues, and Expectations for Exosomes

Yamada¹

2021

Cancers

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Nucleic acid drugs are being developed as novel therapeutic modalities. They have great potential to treat human diseases such as cancers, viral infections, and genetic disorders due to unique characteristics that make it possible to approach undruggable targets using classical small molecule or protein/antibody-based biologics. In this review, I describe the advantages, classification, and clinical status of nucleic acid therapeutics. To date, more than 10 products have been launched, and many products have been tested in clinics. To promote the use of nucleic acid therapeutics such as antibodies, several hurdles need to be surmounted. The most important issue is the delivery of nucleic acids and several other challenges have been reported. Recent advanced delivery platforms are lipid nanoparticles and ligand conjugation approaches. With the progress of exosome biology, exosomes are expected to contribute to the solution of various problems associated with nucleic acid drugs.

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