Yantai New Drug Creation Shandong Laboratory has prepared and tested new heterocyclic amide compounds acting as ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors potentially useful for the treatment of cancer.
Convalife Pharmaceuticals Co. Ltd. has synthesized new polysubstituted aminoquinoline compounds acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors potentially useful for the treatment of immunological disorders and cancer.
Kunshan Xinyunda Biotechnology Co. Ltd. has patented new antibody-drug conjugates (ADCs) consisting of humanized antibodies targeting disintegrin and metalloproteinase domain-containing protein 9 (ADAM9) covalently linked to a payload and potentially useful for the treatment of cancer and inflammatory disorders.
Merck Sharp & Dohme LLC (MSD) has reported new plasma kallikrein (KLKB1) inhibitors potentially useful for the treatment of diabetic macular edema, cancer, hereditary angioedema, uveitis, pancreatitis, arthritis, nephropathy and inflammatory disorders and among others.
Cavalon Therapeutics Inc. and Northwestern University have disclosed new voltage-gated calcium channel Cav1.3 blockers potentially useful for the treatment of Parkinson’s disease and aldosteronism.
Fabry disease is a lysosomal storage disease tied to the X chromosome and caused by pathogenic variants in the GLA gene encoding galactosidase A. It is characterized by progressive accumulation of galactosidase A substrates, including Gb3 and lyso-Gb3, mainly in the kidney, heart and nervous system.
AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload.
Cocrystal Pharma Inc. has reported the discovery of direct-acting antivirals that demonstrate pan-viral activity against multiple viruses, including hantavirus, bunyavirus and influenza. These compounds target a highly conserved region of the viral replication enzyme – the L-protein of Andes virus, which is essential for viral replication and transcription.
Although influenza virus infection can be particularly life-threatening among young infants, approved vaccines are lacking for infants under 6 months. Aiming to overcome the required annual updates to the influenza vaccine, recent efforts have focused on generating vaccines that promote broadly reactive antibodies (Abs) targeting conserved regions of viral proteins, such as the stem domain of hemagglutinin (HA).