First Patient Receives Higher Dose of FLT190, Freeline’s Gene Therapy
The first patient has been dosed with the second, higher dose of FLT190, Freeline Therapeutics’ investigational gene therapy for Fabry disease that’s being studied in its Phase 1/2 MARVEL-1 clinical trial.
Mutations in GLA, the gene that provides instructions for producing Gal A, are the underlying cause of Fabry disease. A faulty Gal A enzyme results in fatty substances globotriaosylceramide (Gb3) and Lyso-Gb3 building up to toxic levels, which can damage the heart, kidney, skin, and central nervous system — the brain and spinal cord.
FLT190 consists of a modified and harmless adeno-associated virus that carries a healthy version of the GLA gene to liver cells, thus ensuring functional Gal A enzyme is produced.
This one-time treatment is administered directly into the bloodstream and is expected to eliminate the need for standard ERT or chaperone therapy. ERT delivers a healthy version of an enzyme to patients, while chaperone therapy involves small molecules binding to faulty enzymes, improving their function.
“We believe FLT190 has the potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment, and we are excited to have initiated this [group] in MARVEL-1,” Pamela Foulds, MD, Freeline’s chief medical officer, said in a press release.
The dose-finding MARVEL-1 trial (NCT04040049) is assessing the safety and effectiveness of FLT190 in up to 15 men with classic Fabry disease, which is associated with total or near-total absence of working Gal A. The trial is recruiting at five sites across Europe and more are expected to open by year’s end.
In the study’s dose-escalation first phase, patients who were previously treated with ERT or chaperone therapy will receive ascending doses of FLT190 to determine the safest and most effective dose.
FLT190 will be evaluated in the trial’s second part with patients who have not received prior treatment.
Patients will be monitored for a nine-month period after which they may choose to enter a long-term extension study for at least five years (MARVEL-2).
https://fabrydiseasenews.com/news/first-fabry-patient-receives-higher-flt190-gene-therapy-dose/
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