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Zavegepant

Zavegepant (BHV-3500) is a calcitonin gene-related peptide (CGRP) receptor antagonist. … [L45505] CGRP is released from sensory nerves and acts as a strong vasodilator, and thanks to these properties, it is involved in pain pathways.

Zetomipzomib

Zetomipzomib is under investigation in clinical trial NCT04033926 (A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis).

Eplontersen

It was previously investigated as a potential treatment for hereditary transthyretin-mediated amyloidosis. … [A262985] As eplontersen targets both the WT and mutant TTR, it poses tremendous promises as a treatment.

Leniolisib

Leniolisib is a potent and selective inhibitor of phosphoinositide 3-kinase δ (PI3Kδ). … [L45758] APDS is a primary immunodeficiency caused by mutations in genes encoding the PI3Kδ, thereby increasing the activity of PI3Kδ, causing immune dysfunction, and elevating susceptibility to infections

Polyinosinic-polycytidylic acid

Ivarmacitinib

Ivarmacitinib (SHR0302) is a selective inhibitor of Janus kinase 1 (JAK1) under investigation for the treatment of various immuno-inflammatory conditions.

Tebipenem pivoxil

Tebipenem pivoxil is an orally administered prodrug of tebipenem, a carbapenem antibacterial that kills bacteria by binding essential penicillin-binding proteins and disrupting cell wall biosynthesis. … severe or resistant Gram-negative infections but had until now been available only intravenously, meaning patients with resistant complicated urinary tract infections generally required hospitalization, a

Lexanersen

Lexanersen is an antisense oligonucleotide under investigation for use as a disease-modifying therapy in Huntington's disease.

Lotilaner

Lotilaner is an ectoparasiticide that is a member of the isoxazoline family of compounds.

Zilganersen

Zilganersen is a glial fibrillary acidic protein (GFAP)-directed antisense oligonucleotide used to treat Alexander disease.