Gene editing
In a Phase Ib trial, VERVE-102 showed it could lower bad cholesterol across doses, setting up an opt-in decision for Eli Lilly and a showdown with Novartis.
Already reeling from years of market chaos, the announced departure of CBER chief Peter Marks sent a ripple across biopharma markets.
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
BEAM-302 “has set the bar for efficacy in this space,” William Blair analysts wrote in an investor note on Monday.
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.
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