Product Description
APB-102 is a gene therapy candidate being evaluated for the potential treatment of SOD1 ALS. APB-102 is a recombinant AAVrh10 vector that expresses an anti-SOD1 artificial microRNA. The microRNA binds to SOD1 mRNA thereby reducing production of the mutant protein in patients with this form of the disease. Reducing mutant SOD1 protein levels may improve survival and function of motor neurons and potentially provide a therapeutic benefit to people with SOD1-linked ALS. APB-102 has received Orphan Drug and Fast Track designation from the U.S. Food and Drug Administration. (Sourced from: https://apic-bio.com/apic-bio-receives-fda-fast-track-designation-for-apb-102-for-the-treatment-of-patients-with-sod1-als/)
Mechanisms of Action: Gene Therapy,SOD1
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: Fast Track - Amyotrophic Lateral Sclerosis *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: UniQure N.V.
Company Location: AMSTERDAM P7 1105 BP
Company CEO: Matthew Kapusta
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Sweden, United States
Active Clinical Trial Count: 2
Highest Development Phases
Phase 2: Amyotrophic Lateral Sclerosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| AMT-162-001 | P2 |
Recruiting |
Amyotrophic Lateral Sclerosis |
2031-03-31 |
|
| SOD1-ALS | P2 |
Recruiting |
Amyotrophic Lateral Sclerosis |
2026-09-30 |
50% |