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.
Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
Scribe Therapeutics Inc. raised $100 million in a series B round to continue its engineering-intensive approach to developing CRISPR-based therapies that employ custom-designed CasX enzymes.
Berkeley, Calif.-based Caribou Biosciences Inc. has raised $115 million in an oversubscribed series C financing co-led by Farallon Capital Management, PFM Health Sciences and Ridgeback Capital Investments. Proceeds from the round will be used to advance its CRISPR technology platform and pipeline of off-the-shelf genome-edited CAR T and CAR-NK cell cancer therapies, including CB-010, its lead CAR T program, now in a phase I trial for patients with relapsed/refractory B-cell non-Hodgkin lymphoma.
Excision Biotherapeutics Inc.’s CEO is specific when he talks about his company’s therapies and what they may achieve: a functional cure. “When you treat someone and they become cancer free, you can’t use the world ‘cured’ because the cancer may come back decades later,” Daniel Dornbusch told BioWorld. “But you can talk about a functional cure, meaning the cancer didn’t come back for a very long time. It’s functionally cured for maybe 10, 20 or 30 years."
