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Gene Therapy Shows Promise in Treating Fabry Disease, Improves Kidney Function
Breaking News: Patients with Fabry disease are experiencing significant improvements in kidney function following a single dose of the gene therapy ST-920 (isaralgagene civaparvovec). The innovative treatment also demonstrates reductions in disease severity within a median of two years post-treatment.
The encouraging results come from top-line data of the Phase 1/2 STAAR trial (NCT04046224), as recently announced by Sangamo Therapeutics, the company spearheading the growth of ST-920.
Significant Clinical Benefits Observed
According To Nathalie Dubois-Stringfellow, Phd, Sangamo’s Chief Development Officer, the data highlights the potential of ST-920 to deliver considerable clinical advantages over existing standards of care. This offers a new way to address the underlying biology of Fabry disease. Sangamo expressed gratitude to the patients and investigators involved in the study and anticipates sharing the data with health authorities.
Based on these positive outcomes, Sangamo intends to seek accelerated approval from the U.S. Food And Drug Administration (FDA) for ST-920.
Accelerated approval allows promising therapies to reach the market sooner, pending further trials confirming their clinical benefits.
The FDA has indicated previously that the STAAR study data could support the accelerated approval of ST-920. Sangamo anticipates submitting its application by early 2026.
Understanding Fabry Disease And ST-920
Fabry disease stems from mutations in the *GLA* gene, crucial for producing the alpha-Gal A enzyme that breaks down specific fatty molecules. A deficiency in this enzyme causes a buildup of toxic fatty molecules in cells. The accumulation leads to organ damage and the manifestation of Fabry disease symptoms, with kidney damage being a common issue.
ST-920 works by delivering a healthy copy of the *GLA* gene directly to liver cells. This enables the production of a functional alpha-Gal A enzyme, which can effectively break down the harmful fatty molecules. The therapy involves a single infusion into the bloodstream.
STAAR Trial: Key Findings
the STAAR study involved 32 individuals with Fabry disease, including both men and women, some of whom had previously undergone enzyme replacement therapy (ERT). The median age of participants was 42 years. Most participants have been monitored for at least two years following ST-920 treatment, with some followed for over four years.
Kidney function was assessed using the estimated glomerular filtration rate (eGFR), a standard measure of kidney efficiency in filtering blood. EGFR is measured in milliliters per minute per 1.73 square meters (mL/min/1.73m2).
Results from STAAR showed that eGFR tended to increase by 1.965 mL/min/1.73m2/year in the year following ST-920 treatment. Among 19 patients followed for two years, eGFR showed an increase of 1.747 mL/min/1.73m2/year.
Did You Know? The eGFR is a crucial indicator of kidney health, with higher values generally indicating better function. A decline in eGFR frequently enough signals progressive kidney disease.
Contrast With Current Treatments
Sangamo reports that the improvements in eGFR observed with ST-920 stand in stark contrast to current Fabry disease treatments, where eGFR tends to decline over time.
For the patient who received the treatment earliest, higher levels of alpha-Gal A enzyme activity were observed for up to 4.5 years. All 18 patients initially on ERT were able to discontinue it, with stable blood levels of lyso-Gb3-a key marker of Fabry disease-and steady heart function.
In addition to enhanced eGFR, the STAAR study participants also showed improvements in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI), which measures overall disease severity, and in the gastrointestinal symptoms rating scale (GSRS), which assesses digestive symptoms. Scores on the short form-36 (SF-36), a measure of overall life quality, also showed positive trends, with some participants needing less pain medication and experiencing more normalized sweating.
“We are thrilled to see these compelling topline STAAR study results, including the positive mean annualized eGFR slope at both 52 and 104 weeks, alongside notable improvements in a range of secondary endpoints,” Dubois-Stringfellow said.
According To Sangamo, ST-920 was generally well-tolerated. The most common side effects were fever, COVID-19, the common cold, and headache, all managed with standard medical care.
Detailed findings from the STAAR trial are slated for presentation at an upcoming medical conference.
| Measure | Result |
|---|---|
| eGFR Increase (1 year) | 1.965 mL/min/1.73m2/year |
| eGFR Increase (2 years) | 1.747 mL/min/1.73m2/year |
| ERT Discontinuation | all 18 patients on ERT discontinued |
| Side Effects | Mostly mild; fever, COVID-19, cold, headache |
The Future Of Fabry Disease Treatment
The Development of ST-920 represents a significant step forward in Fabry disease treatment. Current treatments often involve enzyme replacement therapy, which requires regular infusions and manages symptoms rather than addressing the underlying genetic cause.
gene therapy offers the potential for a one-time treatment that could provide long-term benefits, potentially reducing the burden on patients and healthcare systems. As research progresses, gene therapies like ST-920 may become a cornerstone in managing genetic disorders.
Pro Tip: Stay informed about clinical trials and advancements in genetic research.Connecting with patient advocacy groups can provide valuable support and facts.
The Potential approval of ST-920 by the FDA could revolutionize the treatment landscape for Fabry disease patients, offering new hope for improved quality of life and long-term health outcomes.
Frequently Asked Questions About Fabry Disease And Gene Therapy
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