According to a news release on Yahoo! Finance, clinical-stage biotechnology company Freeline Therapeutics Holdings plc (“Freeline”) shared that it had initiated dosing in the second cohort of the Phase 1/2 MARVEL-1 study. Within the study, researchers are evaluating FLT190 as a potential therapeutic option for Fabry disease. It will build upon data from the first cohort, which was previously dosed. Over the course of this study, up to four cohorts will be dosed to determine the ideal dosing level.
FLT190 is a liver directed investigational AAV gene therapy that is being developed for Fabry disease. Using a proprietary AAV3 capsid, FLT190 delivers a functional GLA copy into the body. This helps to promote α-Gal A to normal range, reducing the symptoms and impact of Fabry disease. Additionally, this treatment is designed to work with just one dose, making it an easy and minimally invasive option for patients. FLT190 has received Orphan Drug designation in both the European Union and the United States.
An Overview of Fabry Disease
Fabry disease is a rare inherited disorder of glycosphingolipid metabolism. It is considered to be a lysosomal storage disorder. Normally, GLA plays a role in producing an enzyme called α-galactosidase A (α-Gal A), which helps to metabolize a specific kind of fat. In Fabry disease, GLA mutations cause the body to produce either low levels of this enzyme, or enzymes that don’t function very well. The enzyme then begins to build up in cells throughout the body, causing a number of adverse health effects. Fabry disease can be classic (manifesting in childhood or teenage years) or late-onset (manifesting in mid-adulthood). Symptoms can (but do not always) include:
- A painful burning sensation in the hands or feet
- Pain that worsens during physical activity
- Abdominal pain
- Dizziness
- Swelling of the lower extremeties
- Proteinuria (high levels of protein in the urine)
- Decreased or absent sweat production
- Flu-like symptoms
- Heat or cold intolerance
- Chronic fatigue
- Nausea and vomiting
- Angiokeratomas
- Diarrhea or constipation
- Corneal dystrophy
- Anemia (low red blood cell count)
- Kidney failure
- Heart disease
The above is not an exhaustive list of potential symptoms.
