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AB-1002
AB-1002 is a gene therapy that aims to treat certain genetic disorders by delivering a healthy copy of the gene.
AB-1002 is a gene therapy that aims to treat certain genetic disorders by delivering a healthy copy of the gene. Used for Treatment of Duchenne muscular dystrophy.
At a glance
| Generic name | AB-1002 |
|---|---|
| Also known as | Gene Therapy, NAN-101 |
| Sponsor | AskBio Inc |
| Drug class | Gene therapy |
| Modality | Small molecule |
| Therapeutic area | Genetic disorders |
| Phase | Phase 2 |
Mechanism of action
AB-1002 uses a viral vector to deliver the healthy gene to the target cells, replacing the faulty gene and potentially restoring normal function. This approach has shown promise in treating genetic diseases caused by mutations in a single gene.
Approved indications
- Treatment of Duchenne muscular dystrophy
Common side effects
- Muscle weakness
Key clinical trials
- Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (PHASE2)
- AB-1002 in Patients With Class III Heart Failure (PHASE1)
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:
- AB-1002 CI brief — competitive landscape report
- AB-1002 updates RSS · CI watch RSS
- AskBio Inc portfolio CI