Compound class:
Antibody
Comment: Nivisnebart is an anti-sortilin 1 (SORT1) monoclonal antibody. It was developed for potential to treat neurodegenerative diseases. SORT1 down-regulation is proposed as a mechanism to elevate progranulin (PGRN) levels for therapeutic benefit in frontotemporal dementia patients with granulin precursor (GRN) mutations [1]. This mechanistic approach is also considered relevant to more prevalent neurodegenerative diseases including Alzheimer's and Parkinson's diseases. Nivisnebart is potentially the INN for Alector's second anti-SORT1 clinical candidate AL101 (a.k.a. GSK4527226), that is in phase 2 clinical trial.
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References |
1. Miyakawa S, Sakuma H, Warude D, Asanuma S, Arimura N, Yoshihara T, Tavares D, Hata A, Ida K, Hori Y et al.. (2020)
Anti-sortilin1 Antibody Up-Regulates Progranulin via Sortilin1 Down-Regulation. Front Neurosci, 14: 586107. [PMID:33384578] |