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: Europe
Company CEO: Matthew Kapusta
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Sweden, United States
Active Clinical Trial Count: 2
Recent & Upcoming Milestones
- Clinical Outcomes Expected - UniQure N.V. announced they will present P1 Amyotrophic Lateral Sclerosis results in 1H26 for APB-102
Highest Development Phases
Phase 2: Amyotrophic Lateral Sclerosis
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT06100276 |
SOD1-ALS | P2 |
Active, not recruiting |
Amyotrophic Lateral Sclerosis |
2026-09-30 |
12% |
2025-10-23 |
Primary Endpoints|Study Completion Date|Treatments|Trial Status |
2024-517244-57-00 |
AMT-162-001 | P2 |
Recruiting |
Amyotrophic Lateral Sclerosis |
2031-03-31 |
2025-05-02 |
Treatments |