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Sangamo Provides Clinical Development Update Including Early Phase 1/2 Beta Thalassemia Gene-edited Cell Therapy Data
BRISBANE, Calif., April 2, 2019 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, today announced recent progress across
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"BRISBANE, Calif., April 2, 2019 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, today announced recent progress across its clinical portfolio, including early data for ST-400, an ex vivo gene-edited beta thalassemia cell therapy developed in partnership with Sanofi, and next steps for its in vivo genome editing programs. Additionally, in separate announcements, Sangamo today also provided an update on its AAV manufacturing capacity and capabilities, and in a joint press release with partner Pfizer, reported interim data from the SB-525 hemophilia A gene therapy program. These updates will be discussed during a conference call and webcast scheduled for 8:00 a.m. Eastern Time this morning (details below).\n\n \n\"Over the last three years, we've built a solid foundation to support Sangamo's clinical organization and we believe this morning's updates demonstrate how the various components of our clinical development activities are coming together,\" said Sandy Macrae, Sangamo's Chief Executive Officer. \"We are leveraging our technical, clinical, and manufacturing expertise along with our partnerships to create a genomic medicine company with an advancing pipeline of clinical development programs in gene therapy, ex vivo gene-edited cell therapy and in vivo genome editing.\"\nST-400 ex vivo gene-edited cell therapy for beta thalassemia: ST-400 is an autologous cell therapy that involves gene editing of a patient's own hematopoietic stem cells (HSCs) using non-viral delivery of zinc finger nuclease (ZFN) technology. The first patient treated with ST-400 in the Phase 1/2 THALES study has the most severe form of transfusion-dependent beta thalassemia (β0/β0). For the two years prior to treatment in the study, this patient received packed red blood cell (PRBC) transfusions every other week. \nDuring the ST-400 infusion, the patient experienced a serious adverse event, a transient allergic reaction considered related to the cryoprotectant present in the product. Thereafter, the post-transplant clinical course was routine. The patient demonstrated neutrophil and platelet recovery, within two and four weeks of infusion, respectively, indicating that ST-400 successfully reconstituted hematopoiesis following conditioning. Indels (small insertions or deletions generated at the targeted DNA sequence) hav...