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ABO-101
ABO-101 is a gene therapy that delivers a functional copy of the FANCC gene to correct Fanconi anemia complementation group C deficiency.
ABO-101 is a gene therapy that delivers a functional copy of the FANCC gene to correct Fanconi anemia complementation group C deficiency. Used for Fanconi anemia complementation group C (FA-C).
At a glance
| Generic name | ABO-101 |
|---|---|
| Sponsor | Abeona Therapeutics, Inc |
| Drug class | Gene therapy |
| Target | FANCC gene |
| Modality | Biologic |
| Therapeutic area | Hematology/Oncology |
| Phase | Phase 2 |
Mechanism of action
ABO-101 uses an adeno-associated virus (AAV) vector to deliver the wild-type FANCC gene into patient hematopoietic stem cells, restoring the function of the Fanconi anemia complementation complex. This correction allows bone marrow cells to repair DNA damage more effectively and reduces the risk of bone marrow failure and malignant transformation associated with Fanconi anemia type C.
Approved indications
- Fanconi anemia complementation group C (FA-C)
Common side effects
- Infusion-related reactions
- Transient cytopenias
- Liver enzyme elevation
Key clinical trials
- Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine) (PHASE1, PHASE2)
- A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ABO-101 CI brief — competitive landscape report
- ABO-101 updates RSS · CI watch RSS
- Abeona Therapeutics, Inc portfolio CI