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AAV9.SLC6A1 Gene Therapy
AAV9-mediated delivery of the SLC6A1 gene to the brain
AAV9-mediated delivery of the SLC6A1 gene to the brain Used for Treats Huntington's disease.
At a glance
| Generic name | AAV9.SLC6A1 Gene Therapy |
|---|---|
| Sponsor | Emily de los Reyes |
| Drug class | Gene therapy |
| Target | SLC6A1 |
| Modality | Biologic |
| Therapeutic area | Neurology |
| Phase | Phase 1 |
Mechanism of action
This gene therapy uses an adeno-associated virus 9 (AAV9) vector to deliver the SLC6A1 gene to the brain, where it is expected to restore normal function of the SLC6A1 protein.
Approved indications
- Treats Huntington's disease
Common side effects
Key clinical trials
- Gene Therapy for SLC6A1 Neurodevelopmental Disorder (PHASE1, PHASE2)
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:
- AAV9.SLC6A1 Gene Therapy CI brief — competitive landscape report
- AAV9.SLC6A1 Gene Therapy updates RSS · CI watch RSS
- Emily de los Reyes portfolio CI