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Received — 26 March 2026 ⏭ Nature Medicine

Nature Medicine
<i>LRRK2</i>-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial Omar S. Mabrouk · Ben Tichler · H. Moore Arnold · Eva C. Thoma · Sara M. Alexanian · Jingxian Chen · Tzu-Ying Liu · Beth Hirschhorn · Roy Llorens Arenas · John W. Annand · Unnati Kapadnis · Alan A. Shomo · Kelly E. Glajch · Kyle Ferber · Yuka Moroishi · Julie Czerkowicz · Jennifer Inra · Ronald B. Postuma · Tanya Gurevich · Pablo Mir · Huw R. Morris · Jason Aldred · Matthew A. Brodsky · Aaron Ellenbogen · Danielle Larson · Christopher M. Tolleson · Andrew Siderowf · Charalampos Tzoulis · Ernest Balaguer · Maria J. Marti · Hien T. Zhao · Holly B. Kordasiewicz · Roger Lane · Warren D. Hirst · Stephanie Fradette · Danielle L. Graham
Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04262-4The first-in-human clinical trial of the LRRK2-targeting antisense oligonucleotide BIIB094 in Parkinson’s disease demonstrates that the treatment is well tolerated and produces dose-dependent reductions in cerebrospinal fluid levels of LRRK2 and phosphorylated Rab10, indicating successful target engagement.
24 March 2026 at 08:00

<i>LRRK2</i>-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

By: Omar S. Mabrouk · Ben Tichler · H. Moore Arnold · Eva C. Thoma · Sara M. Alexanian · Jingxian Chen · Tzu-Ying Liu · Beth Hirschhorn · Roy Llorens Arenas · John W. Annand · Unnati Kapadnis · Alan A. Shomo · Kelly E. Glajch · Kyle Ferber · Yuka Moroishi · Julie Czerkowicz · Jennifer Inra · Ronald B. Postuma · Tanya Gurevich · Pablo Mir · Huw R. Morris · Jason Aldred · Matthew A. Brodsky · Aaron Ellenbogen · Danielle Larson · Christopher M. Tolleson · Andrew Siderowf · Charalampos Tzoulis · Ernest Balaguer · Maria J. Marti · Hien T. Zhao · Holly B. Kordasiewicz · Roger Lane · Warren D. Hirst · Stephanie Fradette · Danielle L. Graham

24 March 2026 at 08:00

Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04262-4

The first-in-human clinical trial of the LRRK2-targeting antisense oligonucleotide BIIB094 in Parkinson’s disease demonstrates that the treatment is well tolerated and produces dose-dependent reductions in cerebrospinal fluid levels of LRRK2 and phosphorylated Rab10, indicating successful target engagement.