Combining Sarepta Therapeutics Inc.’s gene editing technology and Genedit Inc.’s Nanogalaxy platform to treat neuromuscular disorders shows promising potential, the companies reported. A year into the research collaboration, Genedit’s polymer nanoparticles have demonstrated the ability to deliver therapeutic cargo to specific muscle tissue following system administration of targeted genetic medicines.
Edigene Inc. has extended a long-running partnership with an academic lab at the Chinese Academy of Medical Sciences and Peking Union Medical College with the goal of co-developing hematopoietic stem cell regenerative therapies and technology.
Edigene Inc. has extended a long-running partnership with an academic lab at the Chinese Academy of Medical Sciences and Peking Union Medical College with the goal of co-developing hematopoietic stem cell regenerative therapies and technology for the treatment of inherited blood disorders, as well as the discovery of biomarkers to improve quality control of stem cell production.
A raft of potentially high-value drug development collaborations, most for gene and RNA therapies, led the first day of the 40th Annual J.P. Morgan Healthcare Conference Monday. Pfizer Inc. enlisted Beam Therapeutic Inc. to advance in vivo base editing programs for up to $1.35 billion, while Bayer AG tapped Mammoth Biosciences Inc.’s in vivo CRISPR systems expertise in a potential $1 billion-plus deal. Selecta Biosciences Inc. inked a new $1.1 billion partnership with Ginkgo Bioworks Inc. to develop next-generation gene therapy capsids, while Acadia Pharmaceuticals Inc. agreed to pay Stoke Therapeutics Inc. as much as $967 million to develop RNA-based medicines. Work on new mRNA vaccines and therapies is also proceeding, with both Pfizer and Biontech SE announcing new collaborations in the space.
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.
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.
After signing a potential $1.2 billion cell therapy deal with Vertex Pharmaceuticals Inc. in August, Arbor Biotechnologies Inc. has raised $215 million in series B financing to advance next-generation precision gene editing therapeutics.
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.
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).
Prime Medicine Inc. (PM) likely has “a lock on prime editing technology for therapeutic uses,” CEO Keith Gottesdiener told BioWorld, though research labs are continuing to refine the approach. Cambridge, Mass.-based PM has $315 million in the bank that will help advance the platform, which behaves like a DNA word processor to search and replace disease-causing genetic sequences at their exact location in the genome.
