Promis Neurosciences Inc. recently presented the rational design of an Alzheimer’s disease (AD) vaccine candidate maximizing the selective targeting of toxic amyloid-β oligomers.

Dravet syndrome (DS) is a rare and severe form of epilepsy that causes intellectual disability and motor deficits and can lead to premature death. A loss-of-function mutation in one copy of SCN1A, encoding the voltage-gated sodium channel (NaV)1.1 α subunit, is the most frequent alteration found in DS patients and has been linked with inhibitory neuron dysfunction. Despite the potential of gene therapies, AAV-mediated SCN1A gene replacement for DS has not been possible yet due to AAV genome size constraints.

Additional early-stage research and drug discovery news in brief, from: Eleva, NLS Pharmaceutics, Rein Therapeutics, Skye Bioscience.

Oral peptide delivery specialist Cyprumed GmbH is about to find out if the high bioavailability of its tablet formulations seen in animal models will translate across to humans, after signing a $493 million license and option agreement with Merck & Co. Inc.

Momentum Therapeutics Inc. has described receptor-interacting serine/threonine-protein kinase 3 (RIPK3) inhibitors reported to be useful for the treatment of systematic inflammatory response syndrome, autoimmune disease, cancer, metabolic diseases, neurodegeneration and pain.

Insmed Inc. has divulged cathepsin C (dipeptidyl peptidase I, DPP1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, asthma, metastatic cancer, chronic rhinosinusitis, heart failure, inflammatory bowel disease, psoriasis and thrombosis, among others.

Shanghai Pharmaceuticals Holding Co. Ltd. has identified C-C chemokine receptor type 4 (CCR4) antagonists reported to be useful for the treatment of asthma, allergic rhinitis, atopic dermatitis, cancer, systemic lupus erythematosus, rheumatoid arthritis and drug-induced rash.