Gene editing
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
When hiring job candidates to work on cell and gene therapies, companies look for more than just technical skills. Talent acquisition executives from Bristol Myers Squibb and Intellia Therapeutics offer an inside look at what they want in an employee.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
Analysts appear optimistic for Intellia’s gene editor nex-z, which showed a greater serum TTR reduction than Alnylam’s Amvuttra.
BioSpace takes a look at the headlines across a heavy earnings week for biotechs, checking in on Allogene, Rapport and more.
BEAM-101 seems to be competitive with approved sickle cell treatments, William Blair analysts said in a note to investors, but a patient death underscores the need for less-toxic preconditioning treatments.
Jefferies analyst Maury Raycroft said in a note to investors that Thursday’s mid-stage readout pointed to the “unprecedented” complete response rate of Intellia’s investigational in vivo gene editing therapy in the disorder.
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
Stephen Majors from the Alliance for Regenerative Medicine, which hosts the conference, spoke with BioSpace about what the more than 2,000 attendees can expect to learn next week in Phoenix about the pressing issues confronting the industry.
In an effort to expand its cash runway beyond 12 months, Prime Medicine has signed a deal with Bristol Myers Squibb worth a potential $3.5 billion, while also streamlining its pipeline to trim costs.
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