Researchers from Huidagene Therapeutics Co. Ltd. have evaluated the effects of adenine base editing (ABE)-induced exon skipping of exon 50 in a humanized mouse model of Duchenne muscular dystrophy (DMD).
Betta Pharmaceuticals Co. Ltd. has described hypoxia-inducible factor-2α (HIF-2α; EPAS1) inhibitors reported to be useful for the treatment of Von Hippel-Lindau disease, cancer, autoimmune disease and inflammatory disorders.
Research at Humanwell Healthcare (Group) Co. Ltd. has led to the development of heterocyclic 15-hydroxyprostaglandin dehydrogenase (15-PGDH) inhibitors reported to be useful for the treatment of acute lung injury, diabetes, fibrosis, graft-vs.-host disease, heart failure, inflammatory bowel disease, alopecia and osteoporosis, among others.
Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.
Evaxion Biotech A/S has unveiled the technology behind its proprietary genetic adjuvant developed to enhance the effectiveness of DNA and mRNA vaccines for infectious diseases and cancer.
Benchsci (Scinapsis Analytics Inc.), has announced a CAD$95 million (US$70 million) series D funding round. The funds will be used to expand the company’s artificial intelligence (AI) drug discovery platform, Ascend by Benchsci, which enables scientists to discover biological connections, reduce trial and error experimentation, and uncover risks early.
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