AVROBIO Announces Clinical Data Presentation and Multiple Events at WORLDSymposium™ 2020
February 04, 2020 at 07:00 am EST
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AVROBIO, Inc. announced that the company will provide clinical data updates for its investigational programs in Fabry disease and cystinosis, as well as present data from the first clinical use of its plato™ gene therapy platform, including an optimized vector, automated manufacturing system and personalized conditioning regimen. These data will be presented at the WORLDSymposium™, an annual scientific meeting dedicated to lysosomal disorders, Feb. 10-13, 2020, in Orlando, Fla. Analyst and investor event Feb. 10, 2020, from 7:00-9:00 p.m. ET: In addition to members of AVROBIO management, featured speakers will include leading researchers in the area of lysosomal disorders and gene therapy: Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical trial of AVR-RD-04 (CTNS-RD-04), an investigational gene therapy for cystinosis; associate professor of pediatrics at University of California, San Diego, School of Medicine; and chair of the American Society of Gene and Cell Therapy (ASGCT) Gene and Cell Therapy of Genetic and Metabolic Diseases Committee; Jeffrey A. Medin, Ph.D., principal investigator of the Fabry Disease Clinical Research and Therapeutics (FACTs) clinical study of AVR-RD-01, AVROBIO’s investigational gene therapy for Fabry disease; vice chair of research innovation and MACC Fund professor, pediatrics (hematology/oncology) at the Medical College of Wisconsin; and Mark Thomas, M.D., lead investigator for the company-sponsored Phase 2 clinical trial of AVR-RD-01 (FAB-201) for Fabry disease; nephrologist at the Department of Nephrology, Royal Perth Hospital; and clinical professor at the University of Western Australia Medical School. A live webcast of the presentation and accompanying slides will be available at 7:00 p.m. ET under “Events and Presentations” in the Investors section of the company’s website at avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days. Scientific presentation: “Gb3 substrate in endothelial cells of renal peritubular capillaries was reduced in a previously untreated classic Fabry disease male patient treated with AVR-RD-01 investigational lentiviral gene therapy” Birgitte Volck, M.D., Ph.D., president of research & development, AVROBIO, will present an update on the AVROBIO-sponsored Phase 2 trial of AVR-RD-01 in Fabry disease (FAB-201); Poster presentation: Feb. 12, 2020, at 4:30-6:30 p.m. ET; Contemporary Forum platform presentation: Feb. 13, 2020, at 1:45 p.m. ET.
AVROBIO, Inc. is a gene therapy company. The Company is focused on developing potentially curative hematopoietic stem cells (HSC) gene therapies to treat patients with rare diseases following a single dose treatment regimen. The gene therapies that the Company is engaged in developing employ HSCs that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The Company's development focus has been on a group of rare genetic diseases referred to as lysosomal disorders, primarily managed with enzyme replacement therapies (ERTs). Its pipeline is comprised of three HSC gene therapy programs, which include AVR-RD-02 for the treatment of Gaucher disease type 1 and type 3; AVR-RD-03 for the treatment of Pompe disease, and AVR-RD-01 for the treatment of Fabry disease.
AVROBIO, INC. 
