The FDA has awarded orphan drug designation to Juvena Therapeutics Inc.’s JUV-161, an...

Getting revolutionary genetic medicines to the right place is the reason Genedit Inc. was created, and CEO Kunwoo Lee pared down the mission in one word. “Delivery, delivery, delivery,” Lee told BioWorld in describing the goal of Genedit’s Nanogalaxy platform that is focused on delivering genetic medicines into select tissue. Privately held Genedit will work with Genentech to find and develop hydrophilic nanoparticles for creating nucleic acid-based drugs to treat autoimmune diseases. Genedit is getting $15 million up front in a new deal that could bring in up to $629 million in near-term, preclinical and clinical development, commercial and net sales milestones.

The U.S. FDA issued a complete response letter to Shin Nippon Biomedical Laboratories Ltd.’s U.S. subsidiary, Satsuma Pharmaceuticals Inc., for its NDA for dihydroergotamine nasal powder (STS-101) for acute treatment of migraine, with or without aura, in adults. Shin Nippon acquired Satsuma for $220 million in April 2023 and gained rights to STS-101.

Dana-Farber Cancer Institute Inc. has identified proteolysis targeting chimera (PROTAC) compounds comprising a Von Hippel-Lindau disease tumor suppressor (VHL) ligase binding moiety covalently bonded to a histone deacetylase (HDAC) targeting moiety through a linker.

Axonis Therapeutics Inc. has disclosed dual specificity mitogen-activated protein kinase kinase (MAP2K7; MKK7) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, spinal cord injury, multiple sclerosis, neurodegeneration, lysosomal storage, Parkinson’s disease, Huntington’s disease and Alzheimer’s disease, among others.