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Gene Therapy Trial Report

Summary

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)


NCTID NCT06389877 (View at clinicaltrials.gov)
Description 🔄
Development Status Active
Indication Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term DOID:13372
Drug Product Name BEAM-302
Sponsor Beam Therapeutics Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 🔄 192 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SERPINA1 (p.Glu366Lys)
Therapeutic Modality Gene editing
Therapy Route in vivo
Mechanism of Action Mutation correction
Route of Administration Intravenous
Drug Product Type 🔄 Encapsulated mRNA
Target Tissue/Cell Liver
Gene Delivery System Type 🔄 Endocytosis
Non-Viral Vector Type LNP
Editor Type 🔄 ABE mRNA
Dose 1 🔄 Part A: 15mg (n=3); 30mg (n=3); 60mg (n=6); 75mg (n=9); 2X 60mg (n=3)
Dose 2 🔄 Part B: 30mg (n=3); 60mg (n=5)
Dose 3 🔄 Pivotal dose selected: 60mg

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2024-04-22
Completion Date 🔄 2030-05
Last Update 🔄 2026-10-05

Participation Criteria


Eligible Age 🔄 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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 only). * Evidence of emphysema on chest imaging. Key Exclusion Criteria: * BMI \>30. * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Significant liver disease as defined in the protocol. * Active hepatitis A, B or C infection Part B: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Clinical diagnosis of AATD-related liver disease along with evidence of METAVIR F1, F2, or F3 liver fibrosis. * A postbronchodilator FEV1 ≥40% (Phase 1) or ≥35% (Phase 2) of predicted at screening. * FibroScan ≤12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Previous diagnosis of liver cirrhosis or complications of cirrhosis. Part C: Key Inclusion Criteria: * Age 18-75 years. * Confirmed PiZZ AATD diagnosis. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥30% of predicted. * Evidence of emphysema chest imaging. * Liver stiffness \<12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * BMI \>40. * Known history of liver cirrhosis or complications of cirrhosis.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 12
Locations 🔄 New Zealand,Netherlands,United States,Ireland,United Kingdom,Australia

Regulatory Information


Has US IND True
FDA Designations 🔄 Orphan Drug Designation, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates Data from Parts A and B of Phase 1/2 trial released in March 2026, Beam intends to pursue an accelerated approval for BEAM-302

Resources/Links