Grace Science LLC has received FDA clearance of its IND application for GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 deficiency.
IKZF2 encodes the critical zinc fingers 2 and 3, which are responsible for the DNA binding activity of Helios. Researchers from the University of British Columbia and affiliated organizations have identified new de novo dominant negative variants of the IKZF2 gene.
Abbisko Therapeutics Co. Ltd. has received FDA clearance of its IND application for ABSK-012, a highly selective small-molecule inhibitor of fibroblast growth factor receptor 4 (FGFR4), for advanced solid tumors.
Acetylenic lipids extracted from plants or marine sponges are known to have relevant cytotoxicity against tumoral cells. Previous work has shown that structural modifications to lipidic alkynylcarbinol (LAC) underpin specific changes in its cytotoxic activity.
Kynexis BV has launched with €57 million in series A financing with the aim of using its experience in psychiatry, neurology, and drug discovery and development to advance therapeutics for brain diseases.
Propanc Biopharma Inc. has announced that PRP enhances the sensitivity of resistant pancreatic tumor cells to standard chemotherapy treatment and alters the tumor microenvironment by decreasing the fibrotic tissue and its malignancy.
Gate Bioscience Inc., a new(ish) company with a new class of drugs in the works, emerged from stealth mode and disclosed a $60 million series A financing led by Versant Ventures and A16z Bio + Health. Arch Venture Partners and GV took part in the financing as well.