Gene editing
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.
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.
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