Beam Therapeutics’ first clinical data in the rare disease alpha-1 antitrypsin deficiency “set a bar for efficacy in the space,” according to one analyst. But the ongoing market slide poses challenges for biotech companies aiming to raise money by selling stock.
The death wasn’t due to Beam Therapeutics’ sickle cell disease therapy, BEAM-101, but was instead attributed to the preconditioning treatment needed before the genetic medicine is infused. A different Beam program in preclinical development could avoid the need for toxic preconditioning; data from both programs will be presented during the upcoming American Society of Hematology annual meeting.
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Eli Lilly is acquiring Beam Therapeutics’ opt-in rights to three Verve Therapeutics gene-editing therapies for cardiovascular conditions. The deal comes four months after the pharmaceutical giant began a partnership on a preclinical Verve gene-editing therapy for a different target.
Sickle cell disease is among the Beam Therapeutics programs spared, and here the company needs to show differentiation from genetic medicines under FDA review from Vertex Pharmaceuticals and Bluebird Bio. Meanwhile, Beam aims to find partners for its research in allogeneic cancer cell therapies and hepatitis B.
Beam Therapeutics’ application to begin human testing of a cell therapy made with base-editing technology is now under an FDA clinical hold. In other clinical hold news, the FDA lifted a hold on a Celyad Oncology cell therapy clinical trial that was halted in March.
As the annual J.P. Morgan HealthCare Conference kicked off Monday, two pharma giants unveiled deals that give them access to in-vivo gene editing technologies. Pfizer is teaming up with Beam Therapeutics to develop new base-editing therapies while Bayer hopes to develop new medicines with the in-vivo CRISPR editing tools of Mammoth Biosciences.
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Fresh off its first FDA approval, Apellis Pharmaceuticals is teaming up with Beam Therapeutics, a partnership that will use Beam’s base-editing technology to develop new therapies for complement system disorders. Apellis has committed to pay Beam $75 million to kick off the alliance.
Base-editing biotech Beam Therapeutics engineered an all-stock buyout of Guide Therapeutics, a startup that finds, in vivo, the lipid nanoparticles that can deliver genetic medicines. Beam said GuideTx’s tech helps it address tissue types throughout the body, not just the liver.
The companies will explore the use of MGTA-117, which is currently in preclinical IND-enabling studies as a conditioning regimen for patients receiving gene therapy designed to be less toxic than the current standard of chemotherapy or radiation.
The company was founded to develop base editing, a form of gene editing that alters genes at the single-letter level rather than using the "cutting" technique typical of CRISPR/Cas9. Beam had filed to go public in September, aiming at raising $100 million.
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Beam formed the agreement with Prime Medicine, which was founded by one of its own co-founders based on research at The Broad Institute of Harvard and MIT.
The company, officially launched in 2018 after operating in stealth mode for a year, is developing technologies to enable genome editing at the single-letter level, as opposed to the "cutting" of DNA and RNA normally used in CRISPR/Cas9.
The company, partnered with Beam Therapeutics, Verily Life Sciences and academic institutions, plans to use gene editing to tackle coronary artery disease. However, experience with gene therapy indicates cost could be a hurdle.
The company is developing means using CRISPR/Cas9 to edit genomes at the single-letter level, as opposed to the DNA- and RNA-cutting method usually employed.
FDA slaps clinical hold on Crispr Therapeutics and Vertex Pharmaceuticals' upcoming gene-editing trial.