Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.
The knockout of Col4a3 in mice is used as a model of Alport syndrome. Knockout mice develop glomerular disease associated with neutral lipid accumulation in the kidney and progressive renal failure, which can be treated with lipid-lowering agents.
The histone acetyltransferase KAT2A, and its paralog KAT2B have been identified as key drivers of tumor cell plasticity in small-cell lung cancer (SCLC). Researchers from Auron Therapeutics Inc. aimed to evaluate the novel KAT2A/B heterobifunctional protein degrader, AUTX-703, in models of SCLC.
While there is tremendous enthusiasm for GLP-1 drugs for use in obesity, and 80% of the U.S. population is eligible to use the therapies, tremendous obstacles continue to block their access. The blockages include high prices that consumers currently cannot afford coupled with employer health plans that don’t offer the new treatments. A panel discussing the future of GLP-1s at the BioFuture 2024 conference in New York said the next five years will see enormous changes in the way these drugs are prescribed by physicians and used by patients.
Semaglutide, the glucagon-like peptide 1 receptor agonist from Novo Nordisk A/S, which has seemingly improved every disease it’s been tested on, was a focus at Kidney Week 2024, where researchers presented data from multiple clinical studies in patients with kidney diseases.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
Moma Therapeutics Inc. has reported preclinical results on MOMA-313, a potent and selective DNA polymerase θ (POLθ) inhibitor under development for the treatment of homologous recombinant (HR)-deficient cancers.
The development of covalent KRAS G12C inhibitors has represented a significant advance for non-small-cell lung cancer treatment, but other mutations such as KRAS G13C/D still lack effective treatments.
Researchers at the Stony Brook University Renaissance School of Medicine performed a screening in search of Kruppel-like factor 15 (KLF15) agonists for treating proteinuric injuries in the kidney.
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