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AVTX-801
AVTX-801 is a gene therapy that aims to treat certain genetic disorders by replacing or repairing faulty genes.
AVTX-801 is a gene therapy that aims to treat certain genetic disorders by replacing or repairing faulty genes. Used for Treatment of Methylmalonic acidemia.
At a glance
| Generic name | AVTX-801 |
|---|---|
| Also known as | D-Galactose |
| Sponsor | Eva Morava-Kozicz |
| Drug class | Gene therapy |
| Modality | Small molecule |
| Therapeutic area | Genetics |
| Phase | Phase 2 |
Mechanism of action
AVTX-801 uses a viral vector to deliver a healthy copy of the gene to the patient's cells, potentially correcting the underlying genetic defect. This approach has shown promise in treating a range of genetic diseases, including those caused by mutations in the gene responsible for the disorder being targeted by AVTX-801.
Approved indications
- Treatment of Methylmalonic acidemia
Common side effects
- Unknown
Key clinical trials
- Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801) (PHASE2)
- AVTX-801 D-galactose Supplementation in SLC35A2-CDG (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 |