Sangamo Therapeutics and Pfizer have begun dosing patients in a Phase I/II clinical trial (Alta) of investigational gene therapy SB-525 to treat patients with a rare genetic blood disease known as haemophilia A.

SB-525 is being developed to carry a recombinant adeno-associated virus (rAAV) vector with a Factor VIII gene construct driven by a synthetic and liver-specific promoter.

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Designed to assess the safety and tolerability of SB-525, the open-label, adaptive, dose-ranging Phase I/II Alta trial will include approximately 20 adults with severe haemophilia A. 

Sangamo Therapeutics chief executive officer Dr Sandy Macrae said: "We are excited to announce the first patient infusion in this initial clinical trial of SB-525.

“Gene therapy holds promise as a potential one-time treatment to control haemophilia A and prevent spontaneous bleeding.”

"Gene therapy holds promise as a potential one-time treatment to control haemophilia A and prevent spontaneous bleeding."

An ongoing global collaboration and licence agreement between Sangamo and Pfizer includes development and commercialisation of gene therapy programmes, including SB-525, for haemophilia A.

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Pfizer rare disease chief scientific officer Greg LaRosa said: "By combining Sangamo's scientific and technical expertise across multiple genomic platforms, with Pfizer's global reach, resources and expertise, we are advancing the promise of gene therapy with the hope of making a meaningful impact for people living with haemophilia A."

SB-525 investigational gene therapy has received orphan drug and fast-track designations from the US Food and Drug Organisation (FDA) and orphan medicinal product designation from the European Medicines Agency (EMA).

In addition to Pfizer, Sangamo has partnered with Bioverativ to develop therapies for haemoglobinopathies such as sickle cell disease, as well as Shire International for Huntington's disease.