Approval Probability
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BEAM-302 · 1 trial · 1 indication
Numbers and percentages of patients reporting a given AE
Absolute Levels of AAT over time
| Arm | Type | Description |
|---|---|---|
| BEAM-302 Drug Product | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BEAM-302 | DRUG | BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD |
Part A: Inclusion Criteria: * Males or females 18 - 70 years of age inclusive at the time of consent. * Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing). * Blood total AAT level \<11 μM or equivalent protein in mg/dL. * Patients receiving augmentation therapy i...
BEAM-302 is an investigational small molecule being developed for the treatment of Alpha 1-Antitrypsin Deficiency (AATD), a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug has received Orphan Drug and RMAT designations from the FDA.
BEAM-302 is being developed by Beam Therapeutics Inc., a biotechnology company publicly traded under the ticker symbol BEAM. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of BEAM-302 in adult patients with Alpha 1-Antitrypsin Deficiency.
BEAM-302 is currently in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 1 trial is actively recruiting participants and is designed to evaluate the safety and efficacy of BEAM-302 in adult patients with Alpha 1-Antitrypsin Deficiency.
BEAM-302 is being studied in a Phase 1 clinical trial with the identifier NCT06389877. This trial is titled 'A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)' and is currently recruiting participants in the United States, Australia, Ireland, Netherlands, New Zealand, and the United Kingdom.
BEAM-302 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development. The FDA has granted BEAM-302 Orphan Drug and RMAT (Regenerative Medicine Advanced Therapy) designations, which are intended to expedite the development and review of promising therapies for serious conditions.