Skip to NavigationSkip to content

Sangamo secures FDA’s Fast Track Designation for haemophilia candidate

Published on 17/05/17 at 09:23am

Sangamo Therapeutics has announced that the FDA has granted its cDNA gene therapy candidate for haemophilia A Fast Track Designation. The candidate, SB-525, is set to enter clinical trials, at Phase 1/2 level, this year with the results expected by late 2017, early 2018.

SB-525 uses a recombinant adeno-associated virus to deliver a human Factor VIII cDNA construct and proprietary, synthetic liver-specific promoter to the nucleus of liver cells with a single infusion. The therapy is designed as a single treatment strategy intended to provide continuous, therapeutic expression of Factor VIII protein.

Early studies, in non-human primates, have investors excited that this particular treatment could become best-in-class for haemophilia A.

The news comes only a week after Sangamo and Pfizer had entered into a potential $545 million deal. Pfizer was attracted to the licensing partnership as a result of Sangamo’s four lead products, one of which includes SB-525. The deal involved $70 million in cash up-front, with a further $475 million possible in milestone payments.

The deal also meant that should SB-525 prove successful in clinical trials, Pfizer will take over the work of developing the candidate beyond Phase 1/2, as well as being responsible for manufacturing and commercialisation activities.

"With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program," said Dr. Sandy Macrae, Sangamo's Chief Executive Officer, on the announcement of the partnership. "We believe Pfizer's end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases."

Ben Hargreaves

Mission Statement
Pharmafile.com is a leading portal for the pharmaceutical industry, providing industry professionals with pharma news, pharma events, pharma service company listings and pharma jobs,
Site content is produced by our editorial team exclusively for Pharmafile.com and our industry newspaper Pharmafocus. Service company profiles and listings are taken from our pharmaceutical industry directory, Pharmafile, and presented in a unique Find and Compare format to ensure the most relevant matches