Fractyl Health Inc. has expanded an academic-industry scientific partnership focused on advancing research on the role of the gut and pancreas in metabolic disease.

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.

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.

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.

Researchers from Coave Therapeutics SA presented a novel conjugated AAV (coAAV)-GBA1 candidate for the treatment of patients suffering from PD related to GBA1 mutations.

Approximately 2% to 6% of familiar dilated cardiomyopathy (DCM) cases are caused by inherited mutations in the RBM20 gene, which encodes the RNA binding motif 20 (RBM20), a splicing factor that regulates the splicing of several targets involved in the regulation of sarcomeric structure and calcium handling in the myocardium.