Gene editing
The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together.
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
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.
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