Tech Transfer Roundup: Arrakis Gets Penn IP Toward Small Molecule RNA Therapies
Canaan Partners-backed biotech will investigate the therapeutic potential of three-way junctions in folded RNA. UC Berkeley and Duke sign separate license agreements related to CRISPR gene-editing technology.
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CRISPR Therapeutics and Vertex initiated the first human study of CRISPR gene-editing technology with an industry sponsor, following an investigator-sponsored study initiated in China two years ago. These are not the first gene-editing trials, but they're important milestones for the CRISPR field.
Obsidian CEO Michael Gilman described the company's technology – which allows CAR-T cells and other therapies to be activated by available small molecule drugs – as being able to modulate the effective yet toxic therapies by dialing down, turning up or shutting off the treatments as needed.
It sparked at least one analyst to question how Sarepta came up with a price for FDA's first approved Duchenne muscular dystrophy treatment.