Beam Therapeutics Inc. · NASDAQ:BEAM

Beam Therapeutics — European Respiratory Society (ERS) Congress 2026

9 slides from Beam Therapeutics's conference presentation, September 2026

  1. Slide 1: First-in-human in vivo gene editing for severe alpha-1 antitrypsin deficiency

    First-in-human in vivo gene editing for severe alpha-1 antitrypsin deficiency (AATD): initial data from the Phase 1/2 study of BEAM-302 J ohn R Hur s t , E m i l y FA v a n ’ t Wo u t , T h o m a s A s h d o w n , M a t t h e w C o n ro n , N . G e r r y M c E l v a n e y, J e ff re y G a r re t t ,

  2. Slide 2: Conflict of interest disclosure

    Conflict of interest disclosure ü I have the following real or perceived conflicts of interest that relate to this presentation: Affiliation / Financial interest Commercial company Grants/research support: AstraZeneca Honoraria or consultation fees: AstraZeneca, Boehringer Ingelheim, Chiesi, GSK, Sa

  3. Slide 3: Potential for BEAM-302: a one-time treatment that restores gene function

    INTRODUCTION: Potential for BEAM-302 – a one-time treatment that restores gene function to address the spectrum of AATD GOALS OF BEAM-302 AATD BACKGROUND TREATMENT3 • Liver produces M-AAT for Progressive lung disease the first time due to:2 • Low and poorly functioning • Significantly reduces Z- sys

  4. Slide 4: BEAM-302 clinical study enrolled patients with both AATD-associated lung and liver disease

    METHODS: BEAM-302 clinical study enrolled patients with both AATD- associated lung and liver disease • Select key endpoints: PART A: – Rates of TEAEs – Blood levels of total AAT, Z-AAT and M-AAT, AATD-associated lung disease and functional AAT – HNE activity in blood – Z-polymer levels in blood 15 m

  5. Slide 5: Initial safety data suggest BEAM-302 safety profile is consistent with LNP-based therapies

    RESULTS: Initial safety data suggest that BEAM-302 safety profile is consistent with that of LNP-based therapies Part A (N=21) Part B (N=8) 15 mg 30 mg 60 mg 75 mg 30 mg 60mg n (%) (N=3) (N=3) (N=6) (N=9) (N=3) (N=5) Any TEAEs 3 (100) 3 (100) 6 (100) 8 (88.9) 3 (100) 5 (100) Related to BEAM-302 2 (6

  6. Slide 6: A single dose of BEAM-302 led to sustained increases in total AAT levels

    RESULTS: A single dose of BEAM-302 (60 mg) led to sustained increases in total AAT levels to above the 11 μM protective threshold Part A 30 mg 18 Part B 30 mg Part A 60 16 mg Part A: 60 mg Part B: 60 mg Total AAT (μM) Part B 60 (N=6) (N=5) 14 mg Baseline,† mean (SEM) 5.0 (0.3) 4.7 (0.5) 11 μM Blood

  7. Slide 7: BEAM-302 achieved sustained circulating M-AAT levels >90%

    RESULTS: BEAM-302 (60 mg) treatment achieved sustained circulating M-AAT levels >90%, with AAT expression under normal physiological control BEAM-302 resulted in normal AAT regulation BEAM-302 restored predominant production of the M-AAT isoform during inflammation, while maintaining a beneficial M/

  8. Slide 8: BEAM-302 offers the potential for a one-time therapy

    CONCLUSIONS: BEAM-302 offers the potential for a one-time therapy to correct the mutant Z allele in the SERPINA1 gene and restore AAT function Correct DNA mutation – Safety profile is consistent Durable restoration Please see the root cause of disease with LNP-based therapies of AAT function accompa

  9. Slide 9: Acknowledgments

    Acknowledgments Thank you to the study participants, their families, US UK and their caregivers for their participation, and the Boston University and Boston University Hospital Southampton study investigators, study sites, Alpha-1 Medical Center, Boston, NHS Foundation Trust, Foundation, and patien

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