A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease


The proposed study uses a recombinant AAV2/6 vector encoding the cDNA for human α-Gal A (ST-920). The α-Gal A produced by this cDNA has an identical amino acid sequence to the native enzyme, and also to Fabrazyme® (agalsidase beta or equivalent), a clinically approved recombinant protein product. The ST-920 construct encodes a liver-specific promoter, the human α-1-antitrypsin (hAAT) promoter and includes liver-specific regulatory elements. In addition, rAAV2/6 exhibits liver tropism thus providing the potential for long-term hepatic production of α-Gal A in Fabry disease subjects. Studies of ST-920 in a Fabry disease mouse model administered rAAV2/6 encoding hGLA cDNA by intravenous (IV) injection show generation of therapeutic circulating levels of α-Gal A. The one-time treatment with ST-920 minimizes the risk of infusion--related reactions. The goal of ST-920 is to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3.

I'm interested

18 Years and over
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• ≥ 18 years of age
• Documented diagnosis of Fabry disease
• One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma
Exclusion Criteria:

• Known to be unresponsive to ERT
• Neutralizing antibodies to AAV2/6
• Currently receiving migalastat (Galafold™)
• eGFR ≤ 40 ml/min/1.73m2
• New York Heart Association Class III or higher
• Active infection with hepatitis A, B or C, HIV or TB
• History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert's syndrome
• Elevated circulating serum AFP
• Recent or recurrent hypersensitivity response to ERT within previous 6 months
• Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months (topical treatment and inhaled allowed).
• Contraindication to use of corticosteroids
• History of malignancy except for non-melanoma skin cancer
• Recent history of alcohol or substance abuse
• Participation in prior investigational interventional drug or medical device study within previous 3 months
• Prior treatment with a gene therapy product
• Known hypersensitivity to components of ST-920 formulation
• Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection

Biological: ST-920

Fabry Disease

Sangamo, Rare, Lysosomal Storage Disease, Gene Therapy

Brenda Diethelm-Okita -
Chester Whitley, MD, PhD
Phase 1/Phase 2
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