Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D23CDU
|
|||
| Drug Name |
AVR-RD-02
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Gaucher disease [ICD-11: 5C56.0Y; ICD-9: 272.7] | Phase 2/3 | [1] | |
| Company |
AVROBIO
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal acid glucosylceramidase (GBA1) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT05815004) Guard3: An Open-label, Parallel-arm, Randomized, Controlled, Phase 2/Phase 3 Study Evaluating the Efficacy and Safety of Autologous HSC Gene Therapy, AVR-RD-02, Compared to ERT for Gaucher Disease Type 3 in Participants Aged 2 to 25. U.S.National Institutes of Health. | |||
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