Currently Viewing:
Newsroom
Currently Reading
Suspension of a Rural Syringe Service Program Increased Risks of HIV and HCV Acquisition
May 23, 2019 – Wallace Stephens
LifeScan Finds Partner to Move Into CGM Market
May 23, 2019 – Mary Caffrey
Lenalidomide Beats Standard of Care in Smoldering MM
May 23, 2019 – Samantha DiGrande
What We're Reading: Addressing Maternal Care Disparities; Eggs and Stroke Risk; CBD for Opioid Addiction
May 23, 2019 – AJMC Staff
High-Deductible Insurance Plans Create Barriers to COPD Care
May 22, 2019 – Wallace Stephens
Patients With Ovarian Cancer Could Benefit From More Genetic Testing
May 22, 2019 – Laura Joszt
What We're Reading: Vermont AG Sues Sackler Family; Half-Price Insulin; Asthma Rates Fall in LA
May 22, 2019 – AJMC Staff
Increasing Use of Primary Care May Lower Rates of Respiratory Failure
May 22, 2019 – Wallace Stephens
African American COPD Patients Underutilize Pulmonary Rehabilitation
May 21, 2019 – Wallace Stephens

Gene Therapy for Fabry Disease to Advance to Phase 2 Clinical Trials

Kelly Davio
Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).
Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).

FD is caused by absent or deficient activity of the lysosomal enzyme alpha-galactosidase A (a-Gal A) (encoded by the GLA gene), and causes buildup of globotriaosylceramide in cells, which can result in irreversible organ damage. Avrobio’s proposed treatment, AVR-RD-01, is created by extracting and isolating CD34+ stem cells from patients with FD, then transducing them with lentiviral vector that carries a normal GLA gene. The resulting product is infused back into the patient in an out-patient setting in a single dose.

The company will use the newly raised funds to initiate a phase 2 trial of AVR-RD-01 based on promising initial results of a phase 1 clinical trial in 1 patient with FD; the data, presented at the 59th Annual Meeting of the Japanese Society for Inherited Metabolic Diseases, showed that the patient had plasma a-Gal A activity near zero at the study’s initiation. Within 45 days of receiving AVR-RD-01, the patient’s plasma a-Gal A activity increased into the normal range for individuals without FD. The 6-month assessment showed that the patient’s plasma levels remained in the normal range. No related serious adverse events (AEs) were reported during the initial 6-month study period.

If eventually approved, AVR-RD-01 could revolutionize the treatment of FD; currently, the standard of care for the disease is enzyme replacement therapy that involves lifelong, biweekly infusions of the high-cost drug Fabrazyme (agalsidase beta), which carries a price tag of over $300,000 per patient per year. In addition to its expense, Fabrazyme was also subject to a global shortage in 2009 after a manufacturing process introduced viral contamination into the drug supply, and some patients were forced to undergo reduced-dose therapies that resulted in increased rates of serious AEs.

In addition to AVR-RD-01, Avrobio hopes to develop 3 more gene therapies targeting other lysosomal storage disorders: Gaucher disease, and cystinosis (for both of which Avrobio hopes to initiate clinical development by mid-2019), as well as Pompe disease.

 
Copyright AJMC 2006-2018 Clinical Care Targeted Communications Group, LLC. All Rights Reserved.
x
Welcome the the new and improved AJMC.com, the premier managed market network. Tell us about yourself so that we can serve you better.
Sign Up