12th Feb 2020 15:35
(Alliance News) - Syncona Ltd on Wednesday said its investee Freeline Therapeutics has revealed "promising" data from its gene therapy studies for the treatment of Fabry and Gaucher diseases.
Syncona holds a 79% stake in Freeline.
Fabry disease is a rare genetic disease that affects many parts of the body including the skin, eyes, gastrointestinal system, kidney, heart, brain, and nervous system. Gaucher is an inherited genetic disorder that results in the build-up of certain fatty substances in organs such as the spleen and liver, causing the affected organs to enlarge, thereby affecting their function.
Syncona said that preliminary data from the FLT190 study programme for Fabry showed that Freeline's adeno-associated viral gene therapy can deliver sustained levels of the alpha-galactosidase A enzyme from a single treatment. It added that a dose-escalating Phase 1/2 study in adult males with the disease provided encouragement that higher levels of the enzymes could be achieved through dose-escalation.
The alpha-galactosidase enzyme can help digest sugars and fats. When it's missing, complex sugars and fats can easily build up within the gut and throughout the body. The enzyme helps the body break carbohydrates, depriving gas production fuel and toxic bacteria.
Data from the dose escalation study showed a three to four-fold increase in enzyme activity was achieved by week four, and sustained through the data cut-off at week 20.
The biotechnology investment company also said that pre-clinical data in Gaucher disease suggests that the adeno-associated viral gene therapy has therapeutic potential. Data suggests a single injection of its treatment would result in sustained steady levels of fully active beta-glucocerebrosidase enzymes in the bloodstream.
Beta-glucocerebrosidase is an enzyme that helps break down a large molecule called glucocerebroside into glucose and simpler fat molecules.
"As of the data cut-off date, there have been no infusion-related adverse events. Two adverse events were reported in the study. The patient had elevations in ALT levels and levels returned to normal range within 3 weeks following immune suppression. A grade 2 myocarditis was observed but repeat MRI scans showed no evidence of changes compared to base line and it resolved without specific intervention," said Derralynn Hughes, clinical director of haematology & oncology at the Royal Free Hospital London.
Syncona shares were trading 0.4% higher in London on Friday afternoon at 236.50 pence each.
By Ife Taiwo; [email protected]
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