Autosomal dominant polycystic kidney disease (PKD) is a chronic and debilitating condition affecting over 12 million patients worldwide. PKD arises from mutations in the polycystin-1 (PC1) or PC2 genes. PKD is characterized by the formation of fluid-filled cysts in the kidneys, leading to inflammation, fibrosis and organ damage, ultimately resulting in renal failure.

Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.

NLRP3 inflammasome is a key component of the innate immune system. As a major mediator of inflammation, NLRP3 plays a central role in the pathogenesis of neurodegenerative and autoinflammatory conditions.

Dewpoint Therapeutics Inc. has closed its series D financing to advance DPTX-3186, a first-in-class condensate modulator (c-mod), into clinical trials by year-end and deliver early clinical proof of concept in gastric cancer by the end of next year.

Fibrobiologics Inc. has announced IND-enabling updates from its psoriasis research program demonstrating the potential of human dermal fibroblast (HDF) spheroids as a novel therapeutic approach for chronic-relapse psoriasis.

Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.