Clinical Trials

Date: 2017-10-12

Type of information: Presentation of results at a congress

phase: 1-2

Announcement: presentation of results t the 59th Annual Meeting of Japanese Society for Inherited Metaboli Diseases

Company: Avrobio (USA - MA)

Product: AVR-RD-01

Action mechanism:

• gene therapy. AVR-RD-01 is a lentiviral gene therapy being investigated as a single-dose therapy with durable and life-long potential for patients with Fabry disease. AVR-RD-01 uses a state-of-the-art lentiviral vector system that is an efficient and proven gene transfer system for the stable addition of genes to the patient’s stem cells. In patients with Fabry disease, CD34+ stem cells are extracted and isolated, and then transduced with lentiviral vector carrying a normal GLA gene to create AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient in an out-patient setting with the goal of restoring normal GLA gene expression such that normally active ?-galactosidase A enzyme is produced by the patient’s own body. 

Disease: Fabry disease

Therapeutic area: Rare diseases - Genetic diseases - Lysosomal diseases

Country: Canada

Trial details: (NCT02800070)

Latest news:

• • On October 12, 2017 , Avrobio announced initial six-month clinical data from the first patient treated with AVR-RD-01, its lentiviral gene therapy for Fabry disease. Fabry disease is caused by absent or deficient alpha-galactosidase A (a-Gal A) activity, a lysosomal enzyme encoded by the GLA gene.
• At the initiation of the study, the patient with Fabry disease had plasma a-Gal A activity near zero. Within 45 days of receiving AVR-RD-01, the patient’s plasma a-Gal A activity increased into the normal range for individuals without Fabry. The six-month assessment demonstrated continued plasma a-Gal A activity within the normal range for individuals without Fabry. These data are from an academic Phase 1 trial being conducted in partnership with Avrobio by the FACTs team in Canada. The data were presented at the 59th Annual Meeting of Japanese Society for Inherited Metabolic Diseases, October 12-14, 2017, in Kawagoe, Japan.
• Summary of the data presented at the meeting include:
• Patient #1, a male subject with Fabry disease who received a single dose of AVR-RD-01, an ex vivo lentiviral gene therapy manufactured utilizing the patient’s stem cells which are genetically modified to express normally active a-Gal A.Within 45 days of receiving a single dose of AVR-RD-01, the Fabry patient’s plasma a-Gal A activity increased into the normal range for individuals without Fabry. At the 6-month evaluation, the plasma enzyme activity remains increased within the normal range for individuals without Fabry.
• The patient received a minimal intensity conditioning regimen prior to receiving AVR-RD-01 gene therapy. This conditioning regimen allowed for the AVR-RD-01 infusion to be performed on an out-patient basis.
• No study related serious adverse events were reported during this initial 6-month study period.

   

Is general: Yes