Gene editing
A single 1-mg/kg dose of Eli Lilly’s base editor can lower LDL cholesterol levels by 62% in patients with a heritable form of hypercholesterolemia. The pharma acquired the asset last year in the $1.3 billion Verve Therapeutics buy.
In this episode of Denatured, you’ll be hearing from Miguel Forte, president of the International Society for Cell and Gene Therapy (ISCT), and Jon Ellis, co-founder & CEO of Trenchant Bios, speaking live from the ISCT annual meeting. We dive into mesenchymal stem cells and induced pluripotent stem cells, exploring the science behind them, the manufacturing challenges, and the potential for scalable, engineered next-generation therapies.
Weight loss giant Eli Lilly has penned a DNA editing deal with Profluent Bio, continuing its dealmaking spree as well as its commitment to artificial intelligence.
With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for hereditary angioedema.
As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.
The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.
Beam is now in the strongest position of all companies advancing a genetic therapy for alpha-1 antitrypsin deficiency, according to analysts at William Blair.
Pfizer will be responsible for all global clinical and regulatory activities for Beam Therapeutics’ liver-directed gene editor.
Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows.
A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.
PRESS RELEASES