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Drug Names(s): AVR02, AVR 02

Description: AVR-02 consists of hematopoietic stem cells engineered to express alpha galactosidase A. The Company’s approach is to genetically modify a patient’s own cells by adding a functional copy of the faulty gene. CD34+ hematopoetic stem cells are genetically modified to express the enzyme alpha-galactosidase A. The modified cells are then infused back into the patient via a one-time procedure. The objective is to deliver long-lasting or permanent, continuous elevation of endogenous enzyme thereby significantly improving patient outcomes and eliminating onerous lifetime biweekly intra-venous infusions of enzyme replacement therapy.

In Fabry's disease, deficiency of alpha-galactosidase A results in an accumulation of a toxic lipid called globotriaosylceramide or Gb3 in various organs of the body, including the nervous system, kidneys, and heart.

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