information: Treatment of the first patient
Announcement: treatment of the first patient
Company: Sangamo Therapeutics (USA - CA)
Product: SB-FIX - adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX)
- genome editing product/gene therapy. SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo's ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with zinc finger nucleases to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy approaches, which are non-integrating, and may "wash out" of the liver as cells divide and turn over.
- SB-FIX has received Orphan Drug and Fast Track designations from the FDA.
Disease: hemophilia B
area: Rare diseases - Genetic diseases - Hematological diseases
Country: UK, USA
- This open-label clinical trial is designed to assess the safety, tolerability and preliminary efficacy of SB-FIX in adults with severe hemophilia B. The study is currently screening subjects in the United States at hospitals in Washington D.C., Duarte, Detroit, Indianapolis, Dallas, and in the United Kingdom in Glasgow, Birmingham and London.
- • On December 17, 2018, Sangamo Therapeutics announced treatment of the first patient in the Phase 1/2 clinical trial evaluating SB-FIX for patients with hemophilia B.
- • On February 28, 2018, Sangamo Therapeutics announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom has granted the Clinical Trial Authorisation (CTA) for enrollment of subjects into the ongoing Phase 1/2 clinical trial evaluating SB-FIX, a zinc finger nuclease (ZFN)-mediated in vivo genome editing treatment for hemophilia B. The CTA allows for initiation of Europe's first in vivo genome editing study.
- The CTA permits evaluation of SB-FIX in both adults and adolescents. Once preliminary safety and efficacy have been demonstrated in the ongoing SB-FIX Phase 1/2 clinical trial in adults (18 years or older), Sangamo may then begin enrolling adolescents (12 – 17 years of age) into the study. Clinical sites in the United States have been initiated and are screening adult subjects.
- Sangamo expects to initiate sites in the U.K. later this year for the SB-FIX Phase 1/2 clinical trial and to file additional CTAs for its SB-318 and SB-913 in vivo genome editing treatments for Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively.