AVR-RD-01

Generic Name
AVR-RD-01
Brand Names
-
Drug Type
Biotech
Chemical Formula
-
CAS Number
-
Unique Ingredient Identifier
-
Background

AVR-RD-01 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence. AVR-RD-01 is being investigated for Fabry Disease.

Associated Conditions
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Associated Therapies
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Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Phase 1
Terminated
Conditions
Interventions
First Posted Date
2018-03-06
Last Posted Date
2024-01-05
Lead Sponsor
AVROBIO
Target Recruit Count
15
Registration Number
NCT03454893
Locations
🇧🇷

Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande Do Sul, Brazil

🇺🇸

Hackensack University Medical Center, Hackensack, New Jersey, United States

🇺🇸

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States

and more 2 locations
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