AVR-RD-02
- Generic Name
- AVR-RD-02
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
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- CAS Number
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- Unique Ingredient Identifier
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- 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 deoxyribonucleic acid (cDNA) that, upon its integration into human genome, encodes for functional human glucocerebrosidase (GCase). AVR-RD-02 is being investigated in Gaucher Disease.
- Associated Conditions
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- Associated Therapies
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No trials found
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