A Chinese patent covering methods and compositions for the use of CRISPR-Cas9 in eukaryotes has been granted to Dublin-based Ers Genomics Ltd., making what Michael Arciero, vice president of intellectual property and commercial development at the company, called “a strong but not surprising statement by China,” which has made the biotechnology industry one of its top national priorities.
Chroma Medicine Inc. emerged from stealth with $125 million in series A financing and high ambitions to rewrite the rules of genomic medicine by modulating the epigenetic status of target genes in order to switch expression on or off and thereby achieve therapeutic outcomes.
A multidisciplinary team of scientists led by Britt Adamson at Princeton University along with collaborators from the Massachusetts Institute of Technology, the genome editing company Editas Medicine, and University of California, San Francisco have developed a novel high-throughput screening tool, Repair-seq, to profile mutations at targeted DNA lesions.
Investigators at MIT have identified a protein capable of delivering its own mRNA to cells, and engineered that protein to deliver mRNA sequences of their choosing. In a mouse model, the team used their approach to deliver the mRNA for two different proteins.
Facing inevitable challenges ahead beyond peak performance for its wildly successful cystic fibrosis franchise, Vertex Pharmaceuticals Inc. is continuing to look to the future, announcing Aug. 24 it licensed rights to CRISPR gene-editing technology from Arbor Biotechnologies Inc. Co-founded by Feng Zhang and David Walt, Arbor's tech could figure into new Vertex cell therapies for diabetes, hemoglobinopathies and other diseases.
Investigators at MIT have identified a protein capable of delivering its own mRNA to cells, and engineered that protein to deliver mRNA sequences of their choosing.
Brain-wide genome editing via a single systemic dose of modified adeno-associated virus variants that cross the blood-brain barrier may represent a promising new approach for the development of disease-modifying treatments for familial Alzheimer's disease. This strategy could also be applicable to other central nervous system (CNS) disorders, according to a proof-of-concept (PoC) study led by researchers at The Hong Kong University of Science and Technology (HKUST).
Brain-wide genome editing via a single systemic dose of modified adeno-associated virus variants that cross the blood-brain barrier may represent a promising new approach for the development of disease-modifying treatments for familial Alzheimer's disease.
Sometimes, scientific progress comes from conceptual insights that arrive in a flash. More often, however, such progress arrives in a decidedly less glamorous, though no less important, manner – through the development of new technologies in what can be a very slow iterative cycle of getting a new method to work.
