Researchers from Eli Lilly & Co. reported preclinical data on LY-3954068, a MAPT small-interference RNA (siRNA) strategy in AD models. The therapy is designed to reduce tau protein synthesis by inhibiting the translation of MAPT mRNA, with the goal of effectively lowering all forms of tau.
Biohaven Pharmaceuticals Inc. has presented preclinical data on their novel bispecific degrader BHV-1310 for the potential treatment of autoimmune disorders. To date, about 10% of the global population is affected by autoimmune diseases. IgG-lowering agents have suboptimal pharmacology and pharmacodynamic effects and have safety and tolerability challenges.
Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies (DLB) and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder.
Remegen Co. Ltd. emerged as a surprise challenger in the generalized myasthenia gravis space, unveiling positive phase III data of its China-approved lupus drug, telitacicept (RCT-18; Tai’ai), in the rare autoimmune neuromuscular disorder at the 2025 American Academy of Neurology conference.
Tryptamine Therapeutics Ltd. is gearing up to enter the clinic with lead compound TRP-8803, an intravenous-infused psilocybin therapy, in patients with binge eating disorder in conjunction with psychotherapy.
After dropping development in December of its lead program, Spruce Biosciences Inc. has found new life by acquiring a BLA-ready enzyme replacement therapy for the rare genetic neurodegenerative disease Sanfilippo syndrome type B. If approved, the therapy, tralesinidase alfa, could bring Spruce a priority review voucher.
Gilgamesh Pharmaceuticals Inc. has identified phenalkylamine compounds acting as 5-HT2A receptor agonists reported to be useful for the treatment of psychiatric disorders.
Astrogen Co. Ltd. has synthesized prodrugs of 4-hydroxy atomoxetine reported to be useful for the treatment of aphasia, apraxia, attention deficit hyperactivity disorder, cognitive disorders, Alzheimer’s disease, Parkinson’s disease and ischemic stroke.
Researchers from Tridem Bioscience GmbH & Co. KG provided details on the development of TRB-001, an anti-α-synuclein construct consisting of a B-cell epitope derived from human α-synuclein (SNCA), conjugated to CRM197 and C-type lectin (CLEC) β-glucan.
1st Biotherapeutics Inc. has joined The Michael J. Fox Foundation for Parkinson’s Research (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE) program.
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