Promising data from Sangamo Therapeutics‘ research programs, including Alzheimer’s disease, were recently announced.
The results were presented during the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, D.C.
Sangamo’s work in Alzheimer’s revealed a significant reduction in the production of tau, a key protein in several neurodegenerative diseases when aggregated, using the company’s proprietary gene-regulation technology called zinc finger protein transcription factor (ZFP-TF).
The reduction of tau levels has been associated with a reversal of pathology in Alzheimer’s, including a decrease in protein tangles in the brain and neuronal protection.
Animal models showed significant reductions in tau levels across all regions of the brain, including areas involved in learning and memory, such as the cortex, hippocampus, and striatum.
In addition, data from mice with Alzheimer’s suggest that a single administration of ZFP-TFs significantly reduced neuronal dystrophies (degenerative alterations in neurons characteristic of Alzheimer’s). The effects were shown to be long-lasting and specific, as tau was the only suppressed gene.
Sangamo also announced it will advance its Fabry disease program toward human clinical development.
And, the company presented preclinical data from its programs in metabolic disorders mucopolysaccharidosis (MPS) Type I and II.
Sangamo recently initiated two Phase 1/2 clinical trials evaluating genome editing products SB-318 and SB-913, as well as ZFN-mediated in vivo treatments for MPS I and MPS II, respectively. Results are expected in late 2017 or early 2018.
In developments in Sangamo’s molecular biology approaches, cell therapy experiments demonstrated the potential to edit the genome of human B-cells, a type of immune cell.
The data demonstrate the possibility to genetically modify B-cells and limit their natural ability to produce large amounts of antibodies to generate protein production reservoirs. This innovative approach could be relevant for multiple indications, including immune disorders and cancer immunotherapies.
“Such range of expertise allows us to be selective as we pair technology platforms with therapeutic applications, and compels us to make strategic choices about our product candidates,” Sandy Macrae, PhD, Sangamo’s CEO, said in a press release.
To develop some of its products, the company plans to establish collaborations, as in the case of gene regulation for Alzheimer’s, its gene therapy for hemophilia A with Pfizer, and in its oncology program.