Harvard Researchers’ Immune-Silent Stem Cell Technology Licensed to Sana Biotechnology

Developed through Harvard’s Blavatnik Biomedical Accelerator,
innovations could lead to novel cell therapies to treat disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Protecting transplanted cells from immune attack is a serious challenge.
As with any organ transplant, cell lines derived from stem cells are
sometimes rejected. Innovations by Harvard University researchers are
now enabling a new company to develop a solution that may work in cell
therapies for any patient with any disease.

Harvard’s
Office of Technology Development (OTD) has executed a license
agreement with Sana Biotechnology, a recently launched company
co-founded by Harvard faculty that is focused on creating and delivering
engineered cells as medicines for patients. Harvard’s foundational
technology consists of methods for producing hypoimmunogenic stem cells
that can be differentiated into any cell type and then transplanted into
a patient without triggering immune rejection.

Sana was launched in January 2019 with operations in Seattle, WA;
Cambridge, MA; and South San Francisco, CA. The company’s founding
venture capital firms include ARCH Venture Partners, Flagship
Pioneering, and F-Prime Capital.

“We are excited to have placed this important technology with Sana, a
startup which has assembled a world-class team to advance the
development of allogeneic (off-the-shelf) cell therapies,” said Vivian
Berlin, Managing Director, Strategic Partnerships, in Harvard OTD. “The
company’s leadership, vision, and focus on scaled solutions give us
confidence that this groundbreaking technology can be developed and
deployed to the fullest extent possible for patients.”

The hypoimmunogenic stem cells are derived from human pluripotent stem
cells that have been genetically engineered to appear benign to,
effectively hiding from, the immune system. To achieve this outcome, the
inventors modified the cells’ genomes to reduce the activity of genes
that produce proteins that can provoke the transplant recipient’s immune
system, and to increase the activity of genes that produce tolerogenic
factors that signal “friend” not “foe.”

Sana intends to create hypoimmunogenic pluripotent stem cells and use
them as the starting material to develop novel cell therapies. Under
appropriate conditions, these engineered stem cells can be
differentiated, at significant scale, into various types of cells a
patient needs to replace missing or damaged cells in the body. It has
been shown in the field that stem cells can be turned into, for example,
cardiomyocytes (heart muscle), endothelial cells (blood vessels),
hepatocytes (liver cells), or pancreatic beta cells (cells that produce
insulin).

“Overcoming the immune-rejection barrier is essential for making stem
cell–derived therapies broadly accessible,” said Chad
Cowan, PhD, whose laboratory in Harvard’s Department
of Stem Cell and Regenerative Biology (SCRB) developed the licensed
innovations. “These inventions give us the tools to create treatments
and cures for a host of conditions that have few, if any, effective
treatments today.”

In addition to the Cowan Lab’s platform technology, which is exclusively
licensed to Sana within the field of therapeutics, the license agreement
grants Sana non-exclusive access to Harvard technology for performing
high-efficiency, ex vivo genome editing.

Cowan is a co-founder of Sana and the company’s Chief Scientific
Officer. Richard Mulligan, the Mallinckrodt Professor of Genetics, Emeritus,
at Harvard Medical School, is also a co-founder.

“Harvard’s research enterprise is a uniquely powerful engine for
biomedical discovery and startup formation,” said Isaac T. Kohlberg,
Harvard’s Chief Technology Development Officer and Senior Associate
Provost. “Through focused business development efforts, we’re fortunate
to have connected with a team of entrepreneurs and investors who share
our researchers’ vision to transform the state of medical care beyond
conventional pharmaceuticals. By licensing this cell therapy platform to
Sana, we’re leveraging the expertise and resources of a dedicated
business team to ensure Harvard’s innovations bring the greatest benefit
to patients in need.”

The Cowan Lab’s research was supported in part by the National
Institutes of Health (DK097768) and was developed further through
funding from Harvard’s Blavatnik
Biomedical Accelerator and the Harvard
Stem Cell Institute.

“The Harvard research team, with the Blavatnik Biomedical Accelerator,
has developed and validated this technology to an advanced stage, and we
are thrilled to have the opportunity to push it forward,” said Steve
Harr, MD, President and CEO of Sana. “Sana is focused on engineering
cells as medicines for patients, both reprogramming cells in the body to
fix function and making cells outside the body that physicians can
utilize to replace missing or damaged cells in the body. Harvard’s
technology is an important piece in making our vision a reality.”

Cowan, formerly a faculty member in Harvard SCRB, is currently Assistant
Professor of Medicine at Harvard-affiliated Beth Israel Deaconess
Medical Center. Prof. Cowan is also a faculty member of Harvard Stem
Cell Institute.

About Harvard Office of Technology Development

Harvard’s Office of Technology Development (OTD) promotes the public
good by fostering innovation and translating new inventions made at
Harvard University into useful products that are available and
beneficial to society. Our integrated approach to technology development
comprises sponsored research and corporate alliances, intellectual
property management, and technology commercialization through venture
creation and licensing. More than 70 startups have launched to
commercialize Harvard technologies in the past 5 years, collectively
raising more than $1.5 billion in equity financing. To further bridge
the academic-industry development gap, Harvard OTD manages the Blavatnik
Biomedical Accelerator and the Physical Sciences & Engineering
Accelerator. For more information, please visit http://otd.harvard.edu.

About the Blavatnik Biomedical Accelerator

The Blavatnik Biomedical Accelerator at Harvard University, established
in 2013 through a gift from the Blavatnik Family Foundation, guides
Harvard innovations toward translational impact by providing strategic,
monetary, and advisory support for well-defined research projects. Our
work is expressly aimed at hastening the pace of scientific progress,
developing a shared vision with partners in industry, and ensuring that
lifesaving technologies born at Harvard will become new products that
impact the world for the better. To date, the Blavatnik Biomedical
Accelerator has provided $20 million in direct support to 109 Harvard
research projects. Completed projects have resulted in numerous
corporate partnerships, licensing agreements, and startup companies,
generating more than $40 million in new research funding and more than
$30 million in commercialization revenue. Startups launched around
Blavatnik Biomedical Accelerator technologies have collectively raised
more than $500 million in investment. For more information, visit https://otd.harvard.edu/accelerators/blavatnik-biomedical-accelerator.

About the Harvard Stem Cell Institute

The Harvard Stem Cell Institute (HSCI) was founded in 2004 to pursue the
promise of stem cell science and regenerative medicine. Funded by
philanthropy, we are a network of 1,000+ Harvard-affiliated stem cell
scientists throughout the University, its eight affiliated hospitals,
and the biomedical industry who are dedicated to finding cures for
disease. Our research focuses on diseases of aging, the blood, cancer,
metabolism, skin, and the nervous system. Our goal is to bring stem
cell-based treatments to patients as quickly as possible. For more
information, visit http://hsci.harvard.edu.

Contacts

Harvard Office of Technology Development
Caroline Perry,
617-495-4157
[email protected]