OSAKA UNIVERSITY

2024 saw significant medical advancements, including stem cell therapy restoring vision, artificial ovaries for infertility, CAR T-cell therapy for brain cancer, bridge RNA for gene editing, gene therapy restoring hearing, and Xanomeline-trospium for schizophrenia treatment.

Recent medical breakthroughs include CRISPR therapy for sickle cell anemia, stem cell treatment restoring vision, artificial ovaries for fertility, CAR T-cell therapy for brain cancer, and gene therapy enabling a deaf child to hear.

A randomized, double-blind trial on L-arginine's efficacy and safety in treating Spinocerebellar ataxia type 6 (SCA6) showed a mild reduction in ataxia symptoms but lacked statistical significance. Future Phase 3 trials are needed to confirm efficacy and safety.

Yun Zhong, PhD, joins Wedbush Securities as SVP, Equity Research, Biotechnology, initiating coverage on ASND, BMRN, KROS, MLTX, PASG, PTGX, RZLT, and VYGR.

PDBj and ToMMo launch a portal linking genetic variant data from the Tohoku Medical Megabank Project with protein sequence and structural information, simplifying the visualization of variants on protein 3D structures and aiming to evolve into a comprehensive platform for precision medicine.

FT825 / ONO-8250, a CAR T-cell product targeting HER2, showed favorable safety and efficacy in Phase 1 solid tumor study, with no dose-limiting toxicities observed. Preclinical data highlighted its cancer-selective recognition profile, targeting HER2 variants uniquely expressed on tumor cells.

Research team uses corneal tissue from human-induced pluripotent stem cells (iPS cells) to restore vision in limbal stem cell deficiency patients, bypassing traditional corneal transplant limitations.

FT825 / ONO-8250, a CAR T-cell product targeting HER2, showed favorable safety and efficacy in Phase 1 solid tumor study, with no dose-limiting toxicities observed. Preclinical data highlighted the cancer-selective recognition profile of its novel HER2 antigen binding domain, derived from H2CasMab-2, which preferentially targets tumor cells.

Osaka University researchers developed a technology using single-molecule tracking to streamline drug discovery, screening new drugs 100 times faster. They validated the method with EGFR, identifying known and new EGFR-targeting drugs, suggesting potential for new drug mechanisms and repurposing.

Alpha Fusion secures ¥1.02 billion in Series B funding to accelerate Astatine-based therapeutics R&D and global supply chain, leveraging cyclotrons and halogen properties for cancer therapies.