BEAM News & Events

Beam Therapeutics presented the recently reported clinical data from the BEAM-302 Phase 1/2 trial in alpha-1 antitrypsin deficiency at a symposium on translating scientific discovery in gene editing into clinical progress for patients with lung disease. "For BEAM-302, the data shared today build on the growing body of clinical evidence that supports the profound impact of treating AATD at the root cause of disease, the DNA mutation, with this one-time investigational therapy," said Amy Simon, chief medical officer of Beam. Based on feedback from the FDA, Beam intends to pursue an accelerated approval pathway for BEAM-302 and anticipates enrolling approximately 50 additional patients in an expansion of the ongoing open-label Phase 1/2 trial, expecting to initiate this pivotal cohort in the second half of 2026.

Dr. Marty Makary, the commissioner of the Food and Drug Administration, resigned on Tuesday, according to multiple media reports citing administration officials. Makary ultimately resigned over concerns about the administration's decision to authorize fruit-flavored e-cigarettes, which he opposed, four people familiar with the matter told The New York Times. Kyle Diamantas, the F.D.A.'s top food regulator, was named the acting commissioner, according to The Times and Politico, which was first to report of Makary's resignation. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks that could be impacted by the news include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).