A New Approach for Alzheimer’s

Theresa Brady
Theresa Brady
May 20, 2020

We highlight the technology and scientific advancements of BIO Digital participating companies and BIO member companies in our Biotechnology: Beyond Imagination series. Our next feature focuses on Sangamo Therapeutics and its work with gene therapy for a potential treatment for Alzheimer’s patients.

Beyond Imagination Member Spotlight: Sangamo Therapeutics

A pioneer in genomic medicine, Sangamo Therapeutics uses four types of technologies – gene therapy, cell therapy, in vivo genome editing and in vivo genome regulation – to focus on the genetic causes of disease, such as hemophilia, Fabry and Alzheimer’s.

The company recently announced a collaboration with Biogen to develop ST-501, a gene regulation therapy for Alzheimer’s disease and other tauopathies, using Sangamo’s zinc finger protein technology.

The hope is that this technology may one day slow or even halt the disease.


Innovative Technology

Sangamo and Biogen are developing a technology that uses a new gene regulation therapy—zinc finger protein-transcription factors—to target genes and reduce the buildup of toxic tau proteins in the brain. This buildup leads to cell death and tissue loss in Alzheimer’s patients. Slowing or eliminating the buildup may slow down the disease or perhaps provide a permanent treatment for a devastating condition.

The zinc finger platform is based on natural human proteins that recognize specific DNA sequences and enable precise, efficient and specific treatment. The technology can target almost any genome sequence, which may help develop treatments for challenging genetic conditions. And since it allows the complete editing of the target gene, it would result in almost no detectable effects beyond the target.  

Commenting on the technology’s potential, Sandy Macrae, CEO of Sangamo said,  “We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genomic level.”

Alzheimer’s is a debilitating disease – for the patient and their loved ones. BIO supports Sangamo’s work to go beyond symptom-based care by imagining a technology that may treat this disease in the future.

Registered attendees of BIO Digital can learn more about Sangamo Therapeutics by attending a session with Sangamo’s CEO Sandy Macrae: Genomic medicine and bioethics – Ushering in a new era of responsible healthcare. Attendees can also connect with companies like Sangamo through BIO’s One-on-One Partnering™ system, educational programming and company presentations.