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Trial intelligence / Profile preview
Adaptive phase 1/2 study of AVR-RD-02 gene therapy in type 1 Gaucher disease
MOH_2021-05-03_009821Phase 2Not yet recruiting
- Sponsor
- Tectonic Therapeutic
- Start
- Dec 2020
- Modality
- Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of AVR-RD-02 in Gaucher disease type 1 (GD1) (2 arms), sponsored by Tectonic Therapeutic. Primary endpoint: Primary endpoints not specified. Planned start Dec 2020; status: not yet recruiting.
02
Drugs
AVR-RD-02
03
Conditions
Gaucher disease type 1 (GD1)
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