Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.

From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.

As the average cost of new drug R&D continues to skyrocket, the perception around using artificial intelligence (AI) as a tool to boost drug discovery is changing. “Developing new AI-based drugs is a difficult task, not only for Korea but also for countries with leading AI technology,” Hyeyun Jung, principal researcher of Korea Health Industry Development Institute’s Center for Health Industry Policy, told the audience at the Bio Korea meeting on May 9. “But there is a change in perception; [namely that] applying AI to new drug development is not an option but a necessity.”

Researchers from Sichuan Baili Pharmaceutical Co. Ltd. and Systimmune Inc. presented preclinical data for the novel CD33-targeting antibody-drug conjugate (ADC) being evaluated for the treatment of hematologic malignancies.

Recent findings discovered a mutation in the METTL23 gene, which encodes methyltransferase-like protein 23, in a pedigree of normal-tension glaucoma (NTG). The aim of researchers from the Institute for Vision Research, The University of Iowa Roy J and Lucille A Carver College of Medicine was to confirm an association of mutations in this gene with NTG.

“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”

Homerun success of Novo Nordisk A/S’ semaglutide, which recently became the U.S.’s biggest blockbuster drug, is serving as an “inflection point” for obesity therapeutics and fueling the drive for new and improved therapies, speakers said at Bio Korea 2024 on May 8.

α-Fetoprotein (AFP) is a tumor-associated antigen and an ideal target for T-cell receptor T-cell (TCR-T) therapy. While the safety of AFP-targeting TCR-T products has been previously demonstrated in early clinical trials, the efficacy of these cell therapies is still modest, warranting further research.