Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding iduronate-2-sulfatase (I2S). I2S is a lysosomal enzyme responsible for the cleavage of glycosaminoglycans (GAGs), and its deficiency results in accumulation of GAGs leading to a multisystemic disorder.

Current therapies for chronic obstructive pulmonary disease (COPD) alleviate symptoms, but do not deal with disease progression. Respiratory mucus is primarily made up of mucins, of which mucin 5AC (MUC5AC) is the most predominant in COPD. It was hypothesized that mitochondria may control the expression of MUC5AC in COPD airway epithelium by modulating intracellular reactive oxygen species (ROS) levels.

Head and neck squamous cell carcinoma (HNSCC) accounts for high number of new diagnoses each year. Current management is based on surgery followed by radiotherapy or chemotherapy, where treatment-induced fibrosis is a common complication associated with therapy. Since fibrosis is mostly driven by dysregulation of the Rho-ROCK signaling axis, researchers from Australia have investigated the potential of modulating ROCK2 with the small-molecule inhibitor RX-10616 for its potential antifibrotic effect combined with radiotherapy in patient-derived murine models of HNSCC.

Alchemedicine Inc. and Asahi Kasei Pharma Corp. have entered into an exclusive license agreement on lead HiSAP (high solubility and permeability) compounds owned by Alchemedicine.

Beactica Therapeutics AB, together with researchers at KU Leuven, has been awarded a €2.5 million (US$3.0 million) grant by the European Innovation Council (EIC) to advance BEA-17, a precision immune therapy for glioblastoma.

Teijin Pharma Ltd. has entered into a joint research agreement with Aska Pharmaceutical Co. Ltd. to jointly develop novel small-molecule drug candidates for gynecological diseases.