BEAM-302 / Beam Therap - LARVOL DELTA

Late Breaking Abstract - 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 (ERS 2026) - "fAAT increased in a dose-responsive manner as measured by anti-neutrophil elastase capacity. BEAM–302, the first-in-human in vivo therapy to correct a disease-causing mutation, was well tolerated in single-dose cohorts and enabled durable restoration of SERPINA1 gene function at doses ≥60mg."

First-in-human • Late-breaking abstract • P1/2 data • Preclinical • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Infectious Disease • Pulmonary Disease • Respiratory Diseases • ELANE • SERPINA1

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (clinicaltrials.gov) - P1/2 | N=106 | Recruiting | Sponsor: Beam Therapeutics Inc. | Trial completion date: Aug 2027 ➔ May 2030 | Trial primary completion date: Jun 2027 ➔ May 2028

Trial completion date • Trial primary completion date • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) (GlobeNewswire) - "Beam Therapeutics Inc...announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD)...Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, previously reported in March, established clinical proof of concept as a potential treatment for AATD and in vivo base editing...Beam has initiated dosing in the fourth cohort of Part A, evaluating 75 mg of BEAM-302, and expects to report updated data at a medical conference in the second half of 2025."

Orphan drug • P1/2 data • Alpha-1 Antitrypsin Deficiency

Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights (GlobeNewswire) - "Beam plans to continue the dose-escalation portion of Part A of the ongoing BEAM-302 Phase 1/2 clinical trial and expects to report further data at a medical conference in the second half of 2025. The company plans to dose the first patient in Part B of the ongoing BEAM-302 Phase 1/2 clinical trial, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. The company plans to continue dosing in the Phase 1/2 clinical trial of BEAM-301 in GSDIa."

P1/2 data • Trial status • Alpha-1 Antitrypsin Deficiency • Metabolic Disorders

Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation (GlobeNewswire) - P1/2 | N=106 | NCT06389877 | Sponsor: Beam Therapeutics Inc. | "Beam Therapeutics Inc....today announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, establishing clinical proof-of-concept as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing. Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable dose-dependent correction of the disease-causing mutation....Beam plans to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 trial, including enrolling and dosing a fourth dose cohort, and expects to report further data at a medical conference in the second half of 2025. In addition, the company plans to dose the first patient in Part B, which will include AATD patients with mild to moderate liver disease, in the second half of 2025."

P1/2 data • Trial status • Alpha-1 Antitrypsin Deficiency

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (clinicaltrials.gov) - P1/2 | N=106 | Recruiting | Sponsor: Beam Therapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

BEAM‐302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha‐1 Antitrypsin Deficiency (ESGCT 2023) - "The PiZ mutation is an ideal target for correction to wildtype (PiM) by an adenine base editor (ABE) which converts an A to G in genomic DNA...Furthermore, repeat administration of BEAM-302 resulted in further increases in editing rates. Taken together these data support the hypothesis that base-editing by BEAM-302 has the potential to mitigate both the liver and lung pathology of AATD."

Preclinical • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases • ELANE • SERPINA1

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (clinicaltrials.gov) - P1/2 | N=106 | Not yet recruiting | Sponsor: Beam Therapeutics Inc.

New P1/2 trial • Alpha-1 Antitrypsin Deficiency • Genetic Disorders • Hepatology • Pulmonary Disease • Respiratory Diseases

Beam Therapeutics Reports Pipeline Updates and Second Quarter 2023 Financial Results (GlobeNewswire) - "Beam Therapeutics Inc...today reported second quarter 2023 financial results and provided an update on its clinical and pipeline progress...The first patient has consented and is expected to be dosed in the third quarter of 2023...The company plans to share updates on these efforts by year-end 2023...Submit a regulatory application for authorization to initiate clinical trials for BEAM-302 in the first quarter of 2024...Cash, cash equivalents and marketable securities were $1.1 billion as of June 30, 2023, as compared to $1.1 billion as of December 31, 2022....Beam expects that its cash, cash equivalents and marketable securities as of June 30, 2023, will enable the company to fund its anticipated operating expenses and capital expenditure requirements at least into 2025."

Commercial • IND • Trial status • Acute Lymphocytic Leukemia • Alpha-1 Antitrypsin Deficiency • Hematological Malignancies • Leukemia • Metabolic Disorders • Oncology • T Acute Lymphoblastic Leukemia

Beam Therapeutics to Present First Data Highlighting Base Editing Program for Alpha-1 Antitrypsin Deficiency at 23rd ASGCT Annual Meeting (GlobeNewswire, Beam Therapeutics) - "CAMBRIDGE, Mass., April 28, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced preclinical data showcasing the potential of its novel base editing approach for the treatment of alpha-1 antitrypsin deficiency (Alpha-1) liver and lung diseases. The data will be presented in oral and poster sessions during the 23rd American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be hosted virtually May 12-15, 2020."

New molecule • Preclinical • Alpha-1 Antitrypsin Deficiency • Gastroenterology