This web site is part of the Business Intelligence Division of Informa PLC
This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.
Drug Names(s): AVR02, AVR 02
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.
Additional information available to subscribers only: