First patient dosed in Fabry Disease gene therapy trial – PharmaTimes

Freeline has announced the dosing of the first patient in its MARVEL1 study, a multi-centre Phase I/II clinical trial of its liver-directed AAV gene therapy for Fabry Disease.

The study, which is the first clinical-stage adeno-associated virus (AAV) gene therapy study globally for Fabry Disease, leverages Freelines proprietary gene therapy platform, including its novel capsid, which has already shown clinical benefit for Haemophilia B patients.

The study aimed to deliver a replacement copy of the missing gene to the liver, which will then produce continuous high levels of GLA, offering the potential for therapy with a single treatment.

The initiation of the MARVEL1 study and dosing of the first patient is a significant milestone for Freeline, said Chris Hollowood, executive chairman of Freeline.

Continuous high expression of alpha GLA holds the potential for better treatment outcomes than is seen with ERT, the current standard of care. We believe we can access high expression at relatively low doses. With two programmes in the clinic on a common proprietary gene therapy platform, Freeline are building a leading systemic gene therapy company using next-generation AAV technology. These innovative gene therapies have the potential to change patients lives.

Fabry disease is a type of lysosomal storage disorder in which certain fatty molecules are not properly metabolised. Patients have a genetic mutation which leads to a deficiency of -galactosidase A enzyme (GLA) resulting in an accumulation of lipids, such as globotriaosylceramide (Gb3) and globotriaosylsphingosine (LysoGb3), throughout the body. This can cause highly debilitating progressive multi-organ disease.

It is estimated that Fabry Disease affects one in every 40,000 people. It is currently treated by enzyme replacement therapy (ERT), which requires regular and expensive infusions.

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First patient dosed in Fabry Disease gene therapy trial - PharmaTimes

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