Gene editing
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No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
The intellectual property landscape for newer gene-editing technologies, like that for CRISPR-Cas9, remains unclear and hard to navigate.
Tome Biosciences, a gene editing startup that launched in late 2023 with $213 million in funding, will eliminate 131 positions in November, according to a Worker Adjustment and Retraining Act notice.
Despite the promise of its technology and funding from big backers, Tome Biosciences is “operating at reduced capacity” and weighing its “strategic options” as the Massachusetts-based company faces an uncertain future.
Ultracompact CRISPR systems, which are in some cases one-third the size of Cas9, are being designed to be more specific and enable in vivo gene editing in difficult to reach tissues.
As the yearslong litigation over ownership of CRISPR gene editing continues, investors have forged ahead with funding the technology’s development by biopharma.
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.
Results from a Phase I study presented Friday at the ASGCT 2024 annual meeting showed that despite a good safety profile, Excision BioTherapeutics’ HIV gene editor failed to suppress viral activity.
The biotech touted its prime editing technology at ASGCT on Tuesday after receiving FDA clearance last week for a clinical study of a drug candidate based on the platform.
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