Atopic dermatitis (AD) is a chronic and highly prevalent skin disorder with multifactorial pathogenesis that includes mast cell (MC) activation as one of the main players.
Almac Discovery Ltd. has nominated a new preclinical candidate molecule, ALM-401, a first-in-class bispecific antibody-drug conjugate (ADC) targeting EGFR/ROR1. It is being developed for the treatment of refractory lung cancer characterized by dual expression of ROR1 and EGFR.
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from glutamic acid (E) coding for M-AAT to a lysine (K) coding for Z-AAT.
Avirmax Biopharma Inc. has received IND approval from the FDA to initiate a phase I/IIa trial for its gene therapy treatment targeting wet age-related macular degeneration (AMD), including polypoidal choroidal vasculopathy (PCV).
Gyre Therapeutics Inc. has announced IND clearance for Gyre Pharmaceuticals’ F-230 tablets by China’s National Medical Products Administration (NMPA). F-230 is a selective endothelin receptor antagonist, for the treatment of pulmonary arterial hypertension (PAH).
Actym Therapeutics Inc. has obtained IND clearance from the FDA to begin a phase I trial of ACTM-838. The first-in-human study will enroll patients in the U.S. and Australia with advanced solid tumors who have failed prior lines of therapy and have no clinically beneficial treatment options.
Kapoose Creek Bio Corp. has announced the identification and advancement of two lead compounds with potential in neurology, specifically for indications in neurodegeneration and mental health. The compounds, KCB-100 and KCB-200, have shown potent neuroplastic and neuroprotective activity in gold-standard tests of primary neurons.
Several cancer types are treated with epidermal growth factor receptor (EGFR)-targeting agents (EGFR inhibitors), but this treatment is associated with dermal toxicity in up to 90% of cases, where 80% of cases have rash, among other issues. This skin toxicity is mainly driven by elevation of Staphylococcus aureus and the proinflammatory cytokine IL-36γ. Skin keratinocytes’ cutaneous immune defense is impaired by EGFR inhibitors.
Germline variants that did not affect gene function nevertheless affected multiple aspects of breast cancer risk, via their visibility to the immune system and its reactions. Perhaps most surprisingly, the same genetic constellations that were protective at the very earliest stage of breast cancer, stage 0 or ductal carcinoma in situ, were associated with worse outcomes once a tumor had become invasive.
Biosplice Therapeutics Inc. has disclosed dual-specificity tyrosine-(Y)-phosphorylation regulated kinase 1A (DYRK1A) inhibitors reported to be useful for the treatment of cancer, diabetes and Alzheimer’s disease.
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