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BEAM-302 · 1 trial · 1 indication
| 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: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Blood total AAT level \<11 μM. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC \<70% at screening (Ph 1 o...
BEAM-302 is an investigational therapy being developed for Alpha 1-Antitrypsin Deficiency, a rare genetic disease. It is designed to target the SERPINA1 PiZ mutation that causes the condition. It has not been approved for any use and remains in clinical development.
BEAM-302 targets the SERPINA1 PiZ mutation, the genetic variant responsible for Alpha 1-Antitrypsin Deficiency. By acting on this specific mutation, the therapy aims to address the underlying cause of the disease rather than only managing symptoms. It is a small molecule therapeutic in Phase 1 testing.
BEAM-302 is being developed by Beam Therapeutics Inc., a biotechnology company that trades on the Nasdaq under the ticker BEAM. The company is running the clinical program for this asset in Alpha 1-Antitrypsin Deficiency.
BEAM-302 is in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The FDA has granted it Orphan Drug and Regenerative Medicine Advanced Therapy designations, which support its development path in Alpha 1-Antitrypsin Deficiency.
BEAM-302 is being studied in one active trial, NCT06389877, titled a study to evaluate the safety and efficacy of BEAM-302 in adult patients with Alpha-1 Antitrypsin Deficiency. The trial is currently recruiting up to 192 participants aged 18 and older across the United States, Australia, Ireland, the Netherlands, New Zealand, and the United Kingdom.