Can you introduce Sana Biotechnology and explain how the company came to be?
The foundation of the company is built on the tremendous advances over the last decade or so in our ability to modulate and control genes and gene expression as well as to build cells from essentially from scratch, which combined, we call engineering cells. Nearly every disease is caused by damage to or missing cells, and although it is possible to repair and replace cells in a lab setting, we, as a community, have not yet reached the goal of curing numerous devastating diseases with these technologies. Our ambition as a company is to address the most important challenges in engineering cells as medicines, with the goal of making broadly accessible curative medicines a reality for a host of diseases. We have brought together leaders from both industry and academia to help define the most tractable of these challenges, identify the technologies to address them, and execute on making medicines that deliver on our ambitions for patients.
What is Sana’s unique contribution to the field?
Sana's purpose is to change the possibilities for patients through the power of engineered cells.
We are tackling two of the most fundamental challenges in making that vision a reality. In order to have the greatest positive impact for patients, we need to be able to engineer cells both inside and outside of the body. Scientists can modify cells and control genes in many ways in a petri dish. The real challenge is getting these technologies to the cells inside the body so that they can cut, paste, and rewrite the genome or control gene expression in a manner that corrects what has gone wrong in disease. Additionally, we want to be able to replace cells that are severely damaged or missing. Science has figured out how to harness the power of stem cells to essentially build cells from scratch, and now the key to making cell therapy work is figuring out how to conceal these therapeutic cells from the immune system so that it does not reject them as foreign.Sana was thus founded on two technologies: the delivery of genetic material, called the fusogen platform, and the internal hiding of cells from immune rejection, called the hypoimmune platform. Fortunately, we have made significant strides in both areas.
Can you talk about Sana's pipeline and the company's most advanced targets?
Our most advanced product candidate is SC291, a hypoimmune-modified CD19-targeted allogeneic CAR T for patients with B cell malignancies including certain lymphomas and leukemias. Autologous CAR T cell therapies, where the product is made from the patient’s own immune cells, has proven to be transformative in some cases, but difficult to deliver at scale. Allogeneic CAR T cell therapies, which are made from healthy donor cells, have solved some of these scale and logistical challenges, but to date have not had the same degree of long-term patient benefit. Our program is designed to hold onto the scale advantages of allogeneic CAR T cells while also addressing their major limitation – immune rejection of these cells. In history, the only cells in the medical field that have been successfully transplanted without immunosuppression are red blood cells. We have taken insight from this observation as well as the maternal fetal interface to design our system. We have successfully transplanted a broad range of cells using our technology in preclinical studies, and we are initiating a Phase 1 clinical study with initial data expected later in 2023. If we can solve the challenge of immune rejection, we see a direct path to making medicines with curative intent for patients across a host of different types of blood cancers, and over time, a broader range of medicines.
We are also advancing a therapy using pancreatic islet cells for the treatment of Type 1 diabetes, a disease in which the patient’s immune system attacks and kills the cells that make insulin. Leveraging our hypoimmune technology, we modify genetically these islet cells to hide them from the patient’s immune system. Our goal is to replace these missing pancreatic islet cells in a patient so that they can have normal blood glucose without the need for any insulin treatment, freeing the patient from the intense planning and monitoring of blood sugars and insulin injections – essentially curing the diabetes. We expect to begin our first study this year in type 1 diabetics that will give us insight into whether we can hide these cells, without any immunosuppression, from autoimmune and transplant rejection. If all goes according to plan, we will be able to share data later this year from this study.
We believe the ability to hide cells from the immune system will give us the opportunity to not only to treat certain cancers and type 1 diabetes, but also a number of other serious diseases ranging from lupus to multiple sclerosis to Huntington’s disease. Turning great science into medicines is never a direct or easy path, so unexpected challenges along the way are inevitable. However, if this technology works as we hope, we are optimistic about its potential to help patients across a number of diseases.
What has proved the most challenging in Sana’s journey so far?
Biology is always complex and filled with unexpected learnings. Understanding biology, what goes wrong in disease, and how to safely intervene with medicines that can be manufactured at a scale that allows for broad access is far and away the most challenging aspect of the journey. The second most challenging aspect is attracting the right talent and ensuring we create a culture to allow people to thrive. The right culture is one of fearless truth seeking, curiosity, and resilience. The third challenge is ensuring we have the capital, in any market, to leverage our people and biology insights into important medicines for patients. Making medicines is expensive, and we need investors that share our vision for the future.
How does Sana attract and retain talent?
People want to work where they can have the most impact, and we start with clarity of purpose and alignment around our mission. Second, people matter, as great people attract other great people, meaning that every hire has a ripple effect on who joins our company. Third, we work hard to create a culture where people thrive, meaning that they accomplish more than they ever thought possible. Ultimately, most people want to work where they can have the greatest impact and grow personally.
What are Sana’s key objectives for the next three to five years?
Our long-term aspirations are to be able to control or modify any gene in the body, to replace any cell that is damaged or missing, and to markedly improve access to cellular and gene-based medicines. We recognize that these outcomes are aspirational and not readily achievable, and also that the closer we come to reaching them the bigger our impact will be for patients. In the next 3-5 years, we expect to have some key data readouts that will inform us, patients, physicians, and investors of our progress across our key platforms and our first set of drug candidates. One of our platforms could allow us to create an off- the-shelf CAR T cell that can target multiple cancers at a scale that is not currently possible today. Of course, drug development is filled with risks, but with platform success, we see a relatively straightforward path to success across multiple drugs targeting multiple cancers. Success with this technology gives an opportunity to change the treatment of type 1 diabetes, lupus, and a number of other diseases. Success with our delivery platform offers the opportunity to change the treatment of certain cancers, sickle anemia, and other diseases. There is little doubt we will learn a lot over this time frame and will be having a very different conversation about our future in 3-5 years.
