Intellia Therapeutics Inc. and Recode Therapeutics Inc. have established a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis.
Pompe disease is caused by a deficiency in the lysosomal enzyme acid α-glucosidase (GAA) that leads to accumulation of glycogen in the lysosomes, mainly seen in skeletal and cardiac muscles. Researchers from Duke University have developed a new murine model of Pompe disease, which recapitulates human infantile-onset disease. This model harbors the c.1826dupA mutation in the murine Gaa gene, which resembles the human GAA c.1826dupA (p.Y609*) mutation seen in infantile-onset Pompe disease.
Investigators from Tanta University and affiliated organizations have reported the discovery and preclinical characterization of novel carbonic anhydrase (CA) isoform II inhibitors as potential antiglaucoma agents.
Researchers from Zjengzhou University reported on the discovery and preclinical characterization of a novel PROTAC agent aimed at overcoming paclitaxel (Taxol) resistance in non-small-cell lung cancer treatment.
Lantern Pharma Inc. has generated a new class of highly specific and highly potent antibody-drug conjugates (ADCs) with a cryptophycin drug-payload, in collaboration with Bielefeld University.
It has been hypothesized that allogeneic, hypoimmune (HIP) iPSC-derived or donor-derived islet grafts could provide a new and safe therapeutic alternative for the treatment of type 1 diabetes mellitus (T1DM). Researchers from Sana Biotechnology Inc. and affiliated organizations have published preclinical data from studies further assessing this novel strategy in nonhuman primates (NHPs).
A vaccine based on messenger RNA (mRNA) technology against four surface proteins of the envelope and the inner membrane of the mpox virus (MPXV) demonstrated its efficacy in two animal models. Biontech SE scientists designed and tested two different combinations of antigens in mice and macaques for the two infective forms of the virus. One of them showed better results for its evaluation in clinical trials preventing the disease.