Headquarters: Richmond, California, USA
Ticker: SGMO (NASDAQ)
Founded: 1995
Website: sangamo.com
Sangamo Therapeutics is a clinical-stage biopharmaceutical company focused on gene therapy, gene editing, and cell therapy for patients with serious genetic diseases, neurological conditions, and other high unmet needs[1]. The company has developed a proprietary zinc finger protein (ZFP) technology platform that enables precise genome editing and regulation of gene expression[2].
Sangamo's zinc finger platform represents one of the earliest gene editing technologies, predating CRISPR-Cas9 by over a decade. The company has leveraged this expertise to build a diversified pipeline spanning neurology, oncology, and immunology.
| Drug | Indication | Mechanism | Phase |
|---|---|---|---|
| ST-501 | Parkinson's disease | ZFP-TF targeting tau | Preclinical |
| ST-502 | Parkinson's disease | Alpha-synuclein modulation | Preclinical |
| TX-200 | CAR-Treg | Autoimmune | Phase 1 |
ST-501 is Sangamo's lead neurological program, using zinc finger protein transcription factors (ZFP-TFs) to regulate the expression of the MAPT gene encoding tau protein. This approach aims to:
ST-502 targets the SNCA gene encoding alpha-synuclein, the protein that forms Lewy bodies in Parkinson's disease. The ZFP-TF approach allows:
| Drug | Indication | Mechanism | Phase |
|---|---|---|---|
| KITE-361 | Prostate cancer | CAR-T (PSMA) | Phase 1 |
| GLPG | Various | Multiple candidates | Discovery |
| Drug | Indication | Mechanism | Phase |
|---|---|---|---|
| TX-200 | Autoimmune | CAR-Treg | Phase 1 |
Sangamo's platform is based on zinc finger proteins (ZFPs), which are transcription factors that can be engineered to bind to specific DNA sequences. Key features include:
ZFP-TFs are engineered transcription factors that can either activate or repress gene expression without cutting DNA. This approach offers:
For permanent gene editing, Sangamo uses ZFP nucleases that create double-strand breaks at precise genomic locations:
Sangamo has developed a proprietary manufacturing platform for:
Sangamo entered a strategic partnership with Biogen in 2020 for neurological disease programs worth up to $2.7 billion[3]. The collaboration focuses on:
Sangamo has advanced multiple programs through clinical trials:
| Metric | Value |
|---|---|
| Market Cap | ~$200M (2024) |
| Cash Position | ~$150M (Q3 2024) |
| R&D Budget | ~$100M annually |
| Employees | ~500 |
Competition: CRISPR and other gene editing technologies have gained significant ground
Clinical Delays: Some programs have experienced clinical holds or delays
Funding Constraints: Market capitalization has limited resources for large trials
Partnership Dependence: Heavy reliance on partner funding for clinical development
Zinc Finger Proteins
Zinc finger protein technology for gene therapy. Molecular Therapy. 2020. ↩︎