Sangamo to Present Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data

Sangamo Therapeutics (NASDAQ:SGMO) has announced that data from its hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy will be featured on a poster at the Annual Meeting of the American Society of Hematology (ASH).

As quoted in the press release:

Gene Therapy

• Abstract #2060: “Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A”
  Presenter: Barbara Konkle, M.D., Bloodworks Northwest, Professor of Medicine at University of Washington
  December 7^th, 2019, 5:30-7:30pm Eastern Time

The SB-525 poster will show updated Alta study data including durability of Factor VIII (FVIII) levels, bleeding rate, factor usage, and safety, for all five patients in the high dose cohort of 3e13 vg/kg, with approximately 4 months to 11 months of follow-up after treatment with SB-525.

As of the abstract submission date, four patients in the 3e13 vg/kg cohort achieved FVIII levels within the normal range with no bleeding events reported up to 24 weeks post-administration. These patients did not require FVIII replacement therapy following the initial prophylactic period of up to approximately 3 weeks post-SB-525 administration. The fifth patient in the 3e13 vg/kg cohort had only recently undergone treatment with SB-525 at the time of the abstract submission. As previously reported, one patient had treatment-related serious adverse events (SAEs) of hypotension and fever, which occurred approximately 6 hours after completion of the vector infusion and resolved with treatment within 24 hours, with no loss of FVIII expression. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.

Click here to read the full press release.