Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.
Temporal lobe epilepsy (TLE) is the most common form of focal epilepsy characterized by recurrent seizures originating in the hippocampus and with sprouting of mossy fiber axons that contribute to new recurrent synaptic excitability in the dentate gyrus (DG).
The Charcot-Marie-Tooth Association (CMTA) has participated in a seed extension round for Armatus Bio Inc., an emerging biotechnology company that is advancing a unique gene therapy clinical candidate to target Charcot-Marie-Tooth (CMT) type 1A.
Solid Biosciences Inc. has received FDA clearance of its IND application for SGT-003, the company’s next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD). The planned first-in-human phase I/II trial will enroll pediatric patients with DMD to receive SGT-003 as a one-time intravenous infusion.
Ray Therapeutics Inc. has been awarded a $4 million grant by the California Institute for Regenerative Medicine (CIRM) to help advance development of the company’s optogenetics technology platform and support progression of RTX-021 for the treatment of geographic atrophy, the advanced form of age-related macular degeneration.
Takara Bio Inc. has announced the development of a novel adeno-associated virus (AAV) vector, Sonuaav, which exhibits high gene transfer efficiency into inner ear tissues, with a collaborator at Juntendo University School of Medicine.
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.