Pioneering Genetic Cures: Sangamo

Genome Editing, Gene Therapy, Cell Therapy & Gene Regulation

Accelerating toward the future of new genomic therapies – Since the first mapping of the human genome was completed 18 years ago, the progress of sequencing technology has accelerated exponentially. Every day Sangamo is learning more about the function and interplay of its genes, how the team makes it stand out, and how they contribute to human health and disease. The therapeutic potential of the genome is being unlocked.

Over that same time, Sangamo has forged the way toward the future of genomic therapies. The firm’s scientists were the first to edit human cells and the first to conduct clinical trials with gene edited T cells. Recently, it initiated the first ever in vivo genome editing clinical trials. The company has continued to perfect its zinc finger nuclease technology, which today sets the standards for genomic therapies along the three critical dimensions of specificity, precision, and efficiency.

Along the way, it has become experts across a range of genomic therapies, including genome editing, gene regulation, gene therapy, and cell therapy. With this technological range and the firm’s development and manufacturing experience, Sangamo is uniquely prepared to deliver the new genomic therapies that will change the practice of medicine.

Technologies for Genomic Medicines

New genomic therapies are being developed by Sangamo and others which may address the underlying mistakes in DNA that lead to some genetic diseases. The company is also unlocking the potential for precise genetic cures ever since it was founded in 1995. Sangamo has been a leader in genomic therapy research and development. With its experience developing gene editing technologies, it has also become experts in gene therapy, cell therapy, and gene regulation technology. This range of approaches allows it to connect the underlying biology of the disease to the appropriate technology.

Delivery and Manufacturing

The firm currently uses both adeno associated virus (AAV) and mRNA to deliver its therapeutics and continue to develop and refine these platforms as well as its manufacturing processes for therapeutic cell production. Using the molecular biology and AAV expertise it is also developing gene therapy approaches for key indications.

The ZFN 2.0

Innovation and excellence are coded in Sangamo’s DNA as its scientists continuously strive to improve its technology and capabilities. Currently, it is working on next-generation ZFN constructs that are up to 20-fold more active than current constructs. This new and improved ZFN 2.0 platform has the potential to enable the development of more potent genomic therapies for a broad range of monogenic diseases.

Therapeutic Programs under Development

Sangamo’s strategy is to retain and develop product candidates by itself in certain therapeutic areas that meet key criteria including having a breakthrough potential for unmet needs; where development and commercialization are financially and operationally feasible; and where it sees synergies across its product candidates and therapeutic approaches.

The organization is also focusing its proprietary pipeline on research and development of product candidates for inherited metabolic diseases, central nervous system diseases, and immunology.

Collaboration is critical to maximizing the value of its platform technology and has brought important funding and expertise to the research. The firm will continue to collaborate, especially in therapeutic areas that are highly competitive or that require special disease area expertise or resources.

The Man Showing the Right Path

Sandy Macrae (M.B., Ch.B., Ph.D.), President and CEO:

Sandy was the member of the Board of Directors since June 2016. He has twenty years of experience in the pharmaceutical industry most recently serving as the Global Medical Officer of Takeda Pharmaceuticals, from 2012 to March 2016, where he established and led the Global Medical Office, which encompasses medical affairs, regulatory affairs, pharmacovigilance, outcomes research and epidemiology, quantitative sciences and knowledge and informatics. From 2001 to 2012, Dr. Macrae held roles of increasing responsibility at GlaxoSmithKline, including Senior Vice President, Emerging Markets Research and Development (R&D), from 2009 to 2012. In that position, he provided expertise and resources to create a first-of-its-kind group to expand GSK’s global reach by providing R&D strategies, clinical development, and regulatory resources to enter emerging markets and Asia-Pacific. From 2007 to 2008, he was Vice President, Business Development. In that position, he was responsible for scientific assessment and business development project leadership for the neurology, psychiatry, cardiovascular and metabolic therapeutic areas. Earlier in his career, he worked for SmithKline Beecham, where he was responsible for clinical development in the therapeutic areas of neurology and gastroenterology.

Dr. Macrae received his B.S. in pharmacology and his M.B., Ch.B. with honors from Glasgow University. He is a member of the Royal College of Physicians. Dr. Macrae also earned his Ph.D. in molecular genomics at King’s College, Cambridge.

“We are committed to translating ground-breaking science into genomic therapies that transform patients’ lives.”