Lysine demethylase 6A (KDM6A) is a demethylase that plays a key role at regulating developmental gene expression signatures in several tissues, including neuronal cells. The KDM6A gene is located in chromosome X and pathogenic variants in this gene are tied to Kabuki syndrome type 2. Even though progress in understanding the functions of KDM6A has been made, its role in cochlear development and auditory function remains poorly understood.

Researchers from Vicero Inc. have developed a Vincobody platform, which allows for design of novel proprietary VHH antibody fragments that possess the efficacy of dual checkpoint blockade while mitigating the toxicity limitations of current monoclonal antibody therapies.

Previous research has demonstrated that activin receptor-like kinase 7 (ALK7) signaling suppresses lipolysis, resulting in increased adipocyte size and lipid content. Additionally, predicted loss of function variants in the ALK7 gene (ACVR1C) have been associated with reduced waist-to-hip ratio (WHR) adjusted for body mass index, protection from type 2 diabetes, as well as reduced risk of cardiovascular disease.

THB-335 is an oral inhibitor of mast/stem cell growth factor receptor Kit (KIT), which regulates the activation and migration of mast cells, making it a relevant therapeutic target for inflammatory and allergic processes.

Researchers from Revolo Biotherapeutics Ltd. recently presented preclinical data for IRL-201104, a clinical-stage immunomodulatory peptide currently in phase II development as an intravenous treatment of eosinophilic esophagitis.

At the recent AAAAI/WAO meeting, Blueprint Medicines Corp. presented the in vitro and in vivo characterization of BLU-808, a selective inhibitor of wild-type KIT, a transmembrane receptor tyrosine kinase involved in mast cell activation, proliferation and chemotaxis.

Researchers from Medical University of Vienna and affiliated organizations have presented findings from a study that aimed to assess the perilymph and tissue distribution of AC-102, a small and lipophilic intratympanically delivered pyridoindole derivative, in clinical development at Audiocure Pharma GmbH for the treatment of idiopathic sudden sensorineural hearing loss.