Our Science

We are developing platforms that can repair and control genes in cells or replace any cell in the body.

We work with leading academic laboratories and industry partners on research and development related to our technologies.

Scalable manufacturing with a focus on its cost and working to align with key stakeholders will enable broader patient access to our therapies.

Overview

Scientists have made tremendous progress over the past decade in understanding how to modulate genes and use them now to make transformative medicines. The first wave of these transformative medicines has recently launched or is on pace to reach patients soon. However, the field is still in its infancy.

Assembling the right technologies is key to opening up therapeutic opportunities ranging from rare diseases caused by a single genetic mutation to acquired diseases that affect millions.

In our work, three aspirations guide us:

Developing the ability to repair any cell in the body
Developing the ability to replace any cell in the body
Developing the technologies and processes to enable access to our therapies

ex vivo

Imagine a world in which it is possible to provide engineered allogeneic CAR T cells to cure patients with B-cell malignancies and autoimmune diseases, to replace pancreatic islet cells in patients with type 1 diabetes, or to repair damaged neurons with glial progenitor cells in patients with multiple sclerosis.

Differentiating stem cells into clinically needed cell types has the potential to transform the way we treat a host of diseases. Cell replacement therapy offers the opportunity to turn this vision into a reality.

The key challenges in the field are making the appropriate cells at scale and then having them engraft, function, and persist. Making impactful medicines in this field requires success in all of these components.

Engraft = developing the right delivery system, understanding the underlying microenvironment, and ensuring cells have the right features to succeed
Function = understand and reproducibly manufacture the exact cell needed
Persist = overcome immune rejection and cell death

We view the most challenging barrier to be overcoming immune rejection from transplanting a ‘non-self’ cell. Sana has assembled a team of leaders in this area, and successful development of this capability will make cellular therapy products widely accessible.

in vivo

Imagine a world in which it is possible to cure genetically inherited diseases and modify genes to create better therapeutics for acquired diseases. Sana’s in vivo Cell Engineering platform aims to provide solutions for patients that current gene therapies cannot address. Success will require creative science, experienced product and clinical development strategies, and aggregating important technologies.

Successful in vivo Cell Engineering relies on three core components – delivery, gene modification, and execution.

Delivery – the ability to deliver any payload to any cell in a specific, predictable, and repeatable way. Success in each of these inputs unlocks the ability to make meaningfully improved medicines. Sana’s early focus is centered on developing its novel technology and building internal expertise in this area.
Gene Modification – the ability to gene-edit, base-edit, gene-insert, and control gene expression each has the potential to impact multiple diseases. Sana is focused on building internal expertise in this area and partnering important technologies with others in the field.
Execution – the ability to manufacture a consistent and scaled product, conduct smart clinical trials, and work with all stakeholders is essential for bringing medicines to patients.