Beam Therapeutics, a pioneer in the gene editing field of base editing, has unveiled early data from its first in vivo candidate, BEAM-302, which it hopes to take into the clinic next year.
Beam Picks Crowded AAT Field To Prove Its In Vivo Gene Editing
Base Editing Could Trump Existing Approaches
With its first US study for a base-edited CAR-T therapy now back on track, Beam Therapeutics is gearing up to begin studies of its first in vivo candidate, BEAM-302, next year in AAT.

More from Alimentary/Metabolic
The company plans a tiered sales approach as it targets endocrinologists for hypothalamic obesity, which is more widely diagnosed than its current indication for Bardet-Biedl syndrome.
Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
Novo’s semaglutide has shown its benefits as an injectable in patients with peripheral arterial disease and as an oral agent in cardiovascular outcomes, but stronger results are expected soon from Lilly’s tirzepatide.
The obesity market leader has unveiled its second deal in days, paying $75m upfront for a potential first-in-class ACSL5 inhibitor, while the deal also provides some respite for Lexicon.
More from Therapy Areas
The pact could be worth more than $2.6bn and adds to growing big pharma investment in the technology.
Following the near-total dominance of IgG antibodies, the UK group is backing the potential of IgA and IgE-based therapies to transform cancer treatment with the purchase of US-headquartered TigaTx.
By allowing it to enter the brain more easily, trontinemab’s brain shuttle brings more patients to ‘amyloid zero’ levels faster, and with fewer brain swelling side effects.