Sangamo and Astellas Announce Capsid License Agreement to Deliver Genomic Medicine for Neurological Diseases

Zinc finger technology

Sangamo’s zinc finger platform uniquely confers several advantages for genomic medicines, including versatility, specificity and clinical utility.

Overview

Zinc Finger Proteins (ZFPs or ZFs) are human proteins which naturally regulate the genome through sequence-specific interactions with DNA and regulatory proteins.

Sangamo’s ZF technologies are composed of a DNA-binding domain and a functional domain (e.g., nuclease, transcription factor).

ZF technologies are highly versatile and customizable. We can append a variety of different functional domains, allowing us to adapt and optimize the functionality based upon the specific disease of interest.

ZF Versatility

ZF technologies also offer unique high-resolution targeting that allows for genome-wide coverage.

Using Sangamo’s proprietary library of thousands of zinc fingers, we can design hundreds of ZF arrays to target a desired genomic location. ZF arrays can then be fine-tuned to allow for greater activity and specificity through modifications to the DNA-protein interface. This allows us to design the optimal ZF protein for each potential therapeutic use.

In addition, ZF are compact proteins, which not only allows greater genome accessibility, but also makes them highly compatible with viral vectors, greatly improving our ability for cellular delivery.

For example, ZF-repressors are small enough for multiplexed packaging into viral vectors and can achieve high levels of gene repression, which may be suitable for potential therapeutic applications.

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Furthermore, Sangamo scientists recently developed a compact base editor system that can be targeted with high precision and specificity using ZFs.

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Using our highly versatile zinc finger platform, we are designing and developing genomic medicines to transform the lives of patients suffering from severe diseases.

For more information on our ZF technology, watch the short video below.

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