AVR-RD-02
- Generic Name
- AVR-RD-02
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- -
- Unique Ingredient Identifier
- -
- Background
AVR-RD-02 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized, complementary...
- Associated Conditions
- -
- Associated Therapies
- -
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
- First Posted Date
- 2019-10-30
- Last Posted Date
- 2024-01-18
- Lead Sponsor
- AVROBIO
- Target Recruit Count
- 8
- Registration Number
- NCT04145037
- Locations
- 🇺🇸
University of Iowa, Iowa City, Iowa, United States
🇺🇸UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇨🇦University Health Network, Toronto, Ontario, Canada
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