Gene editing
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
A new executive order aims to smooth the path for getting U.S. manufacturing facilities up and running; HHS says it will require placebo-controlled trials for all vaccine approvals; tariff threats hit BioNTech; Novo Nordisk’s FDA application for an oral version of Wegovy is accepted; and more.
Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative to conventional CRISPR. Clinical results have so far been promising.
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.
PRESS RELEASES