Cited by 143RNA Interference and Gene DeliveryBarrier Structure and Function StudiesDrug Transport and Resistance Mechanisms

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Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

Barker, Scarlett J., Thayer, Mai B., Kim, Chaeyoung, Tatarakis, David, Simon, Matthew J., Dial, Rebekah, Nilewski, Lizanne, Wells, Robert C., Zhou, Yinhan, Afetian, Megan, Akkapeddi, Padma, Chappell, Alfred, Chew, Kylie S., Chow, Johann, Clemens, Allisa, Discenza, Claire B., Dugas, Jason C., Dwyer, Chrissa, Earr, Timothy, Ha, Connie, Ho, Yvonne S., Huynh, David, Lozano, Edwin I., Jayaraman, Srini, Kwan, Wanda, Mahon, Cathal, Pizzo, Michelle, Robles-Colmenares, Yaneth, Roche, Elysia, Sanders, Laura, Stergioulis, Alexander, Tong, Raymond, Tran, Hai, Zuchero, Y., Estrada, Anthony A., Gadkar, Kapil, Koth, Christopher M. M., Sanchez, Pascal E., Thorne, Robert G., Watts, Ryan J., Sandmann, Thomas, Kane, Lesley A., Rigo, Frank, Dennis, Mark S., Lewcock, Joseph W., DeVos, Sarah L.·2024·Science Translational Medicine

DOI 10.1126/scitranslmed.adi2245

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Abstract

Antisense oligonucleotides (ASOs) are promising therapeutics for treating various neurological disorders. However, ASOs are unable to readily cross the mammalian blood-brain barrier (BBB) and therefore need to be delivered intrathecally to the central nervous system (CNS). Here, we engineered a human transferrin receptor 1 (TfR1) binding molecule, the oligonucleotide transport vehicle (OTV), to transport a tool ASO across the BBB in human TfR knockin (TfR mu/hu KI) mice and nonhuman primates. Intravenous injection and systemic delivery of OTV to TfR mu/hu KI mice resulted in sustained knockdown of the ASO target RNA, Malat1 , across multiple mouse CNS regions and cell types, including endothelial cells, neurons, astrocytes, microglia, and oligodendrocytes. In addition, systemic delivery of OTV enabled Malat1 RNA knockdown in mouse quadriceps and cardiac muscles, which are difficult to target with oligonucleotides alone. Systemically delivered OTV enabled a more uniform ASO biodistribution profile in the CNS of TfR mu/hu KI mice and greater knockdown of Malat1 RNA compared with a bivalent, high-affinity TfR antibody. In cynomolgus macaques, an OTV directed against MALAT1 displayed robust ASO delivery to the primate CNS and enabled more uniform biodistribution and RNA target knockdown compared with intrathecal dosing of the same unconjugated ASO. Our data support systemically delivered OTV as a potential platform for delivering therapeutic ASOs across the BBB.

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Authors: Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M., Akkapeddi, P., Chappell, A., Chew, K. S., Chow, J., Clemens, A., Discenza, C. B., Dugas, J. C., Dwyer, C., Earr, T., Ha, C., Ho, Y. S., Huynh, D., Lozano, E. I., Jayaraman, S., Kwan, W., Mahon, C., Pizzo, M., Robles-Colmenares, Y., Roche, E., Sanders, L., Stergioulis, A., Tong, R., Tran, H., Zuchero, Y., Estrada, A. A., Gadkar, K., Koth, C. M. M., Sanchez, P. E., Thorne, R. G., Watts, R. J., Sandmann, T., Kane, L. A., Rigo, F., Dennis, M. S., Lewcock, J. W., & DeVos, S. L.
Affiliations: Denali Therapeutics (United States) (US)
Ionis Pharmaceuticals (United States) (US)
Denali Therapeutics Inc., South San Francisco, CA, USA.
Corresponding author: Barker, Scarlett J.
Thayer, Mai B.
Kim, Chaeyoung
Tatarakis, David
ORCID: Barker, Scarlett J.
Thayer, Mai B.
Kim, Chaeyoung
Tatarakis, David
Simon, Matthew J.
Dial, Rebekah
Nilewski, Lizanne
Wells, Robert C.
Afetian, Megan
Akkapeddi, Padma
Chappell, Alfred
Chew, Kylie S.
Chow, Johann
Discenza, Claire B.
Dugas, Jason C.
Dwyer, Chrissa
Earr, Timothy
Ha, Connie
Jayaraman, Srini
Mahon, Cathal
Pizzo, Michelle
Robles-Colmenares, Yaneth
Sanders, Laura
Stergioulis, Alexander
Tong, Raymond
Tran, Hai
Zuchero, Y.
Estrada, Anthony A.
Gadkar, Kapil
Koth, Christopher M. M.
Sanchez, Pascal E.
Thorne, Robert G.
Watts, Ryan J.
Sandmann, Thomas
Kane, Lesley A.
Rigo, Frank
Dennis, Mark S.
Lewcock, Joseph W.
DeVos, Sarah L.
Venue: Science Translational Medicine
Publisher: American Association for the Advancement of Science (AAAS)
Published: 2024-08-14
Volume: 16
Issue: 760
Pages: eadi2245-eadi2245
ISSN: 1946-6234 (ISSN-L)
1946-6242
DOI: 10.1126/scitranslmed.adi2245
OpenAlex: W4401596729
Cited by: 142
References: 70
Citations by year: 2026: 65 · 2025: 68 · 2024: 8
FWCI: 19.19
Type: journal article
Language: en
Open access: closed
Locations: Science Translational Medicine (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
Health Sciences › Medicine › Oncology › Drug Transport and Resistance Mechanisms
Keywords: antisense oligonucleotides, blood-brain barrier, transferrin receptor 1, MALAT1, brain drug delivery, systemic drug delivery, cynomolgus macaques, RNA knockdown, intrathecal drug delivery, tissue distribution
MeSH terms: Animals, Biological Transport, Blood-Brain Barrier, Humans, Macaca fascicularis, Receptors, Transferrin, Tissue Distribution, Oligonucleotides, Antisense, Mice, Gene Knockdown Techniques, RNA, Long Noncoding

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Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

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