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.
DUBLIN – Bacteriophage, like other viruses, can be viewed as complex molecular machines with two essential functions: infection and replication. The first involves the efficient injection into the host cell of their genomic payload; the second involves hijacking the host’s nucleic acid replication machinery to generate the functional and structural proteins needed to give rise to progeny viruses.
CEO John Leonard said Intellia Therapeutics Inc. plans “to share information on a cohort-by-cohort basis, so we get a consistent readout” and, as the year goes on, longer-term follow-up findings will emerge from the phase I trial with the company’s lead in vivo genome editing candidate, NTLA-2001.
After cutting a deal worth up to $1.1 billion with Crispr Therapeutics AG, it took Vertex Pharmaceuticals Inc. only two days to come up with another collaboration that could top the $1 billion mark, this time with Obsidian Therapeutics Inc.
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.
DUBLIN – Cellectis SA is picking up $15 million worth of equity in Cytovia Therapeutics Inc. and could earn as much as $760 million in development, regulatory and sales milestones from a deal involving up to five gene-edited allogeneic natural killer (NK) cell or chimeric antigen receptor (CAR-NK) cell therapies employing its Talen (transcription activator-like effector nuclease) gene editing technology.
Sekar Kathiresan is building Verve Therapeutics Inc. around the concept of a one-and-done treatment for cardiovascular disease because only half of patients are disciplined enough to take a statin every day. “That’s a huge issue for durable cholesterol lowering after a heart attack,” Verve’s CEO and co-founder told BioWorld.
Tessera Therapeutics Inc., a Cambridge, Mass.-based company working to "write therapeutic instructions into the genome," has raised $230 million in series B financing to back its development of potential cures and treatments for cardiovascular, oncological, neurodegenerative and infectious diseases.
