A bioRxiv preprint reports that small “molecular editing” changes converted LLC0424, a dual NSD2/GSPT1 degrader, into two selective compounds: fluorine substitution produced the NSD2-directed PROTAC 424-ND, while methylene insertion produced the GSPT1 molecular glue…
Four approvals, a first-ever in-vivo CRISPR filing accepted with Priority Review, two high-profile pivotal misses, and roughly $1.6 billion of confirmed private financing — a week where mechanism mattered less than manufacturing, endpoint choice…
FDA approved Isembyld (apitegromab-mstn) for adults and children two years and older with spinal muscular atrophy who are already receiving an SMN2-targeted treatment. The approval establishes the first U.S. therapy for SMA directed at…
FDA approved Joenja, leniolisib, for children aged four through eleven years with activated phosphoinositide 3-kinase delta syndrome who weigh at least 27 kilograms. The 40-milligram and 50-milligram twice-daily doses make Joenja the first FDA-approved…
Immutep reported that its continuing review of the discontinued TACTI-004 Phase III trial has identified structural differences between eftilagimod alfa manufactured in 200-liter and 2,000-liter processes, including a subtle N-glycan difference. Earlier Phase I
Eloxx Pharmaceuticals dosed the first two participants in EXACT, a randomized, placebo-controlled, delayed-start Phase 2b study of exaluren in nonsense-mutation Alport syndrome. The global study plans to enroll 24 patients with premature stop mutations…
The U.S. Food and Drug Administration extended its review of zanzalintinib plus atezolizumab for previously treated metastatic colorectal cancer by three months, moving the Prescription Drug User Fee Act action date from December 3,…
GenScript Biotech conditionally placed 77.126 million new shares at HK$30.50 each, targeting net proceeds of approximately HK$2.327 billion. About 70% is intended for artificial-intelligence-driven drug-discovery capacity and infrastructure, 20% for related research, digital-workflow integration…
John P. Sharp resigned as Skye Bioscience’s chief financial officer and principal financial and accounting officer effective September 8. The board appointed president and chief executive officer Punit Dhillon as principal financial and accounting…
Exegenesis Bio and Modalis Therapeutics entered a research collaboration and license agreement giving Modalis access to EMC181, an engineered muscle-tropic, liver-detargeted adeno-associated virus capsid, for MDL-201. The program combines that capsid with Modalis’s CRISPR-GNDM…
