12 September 2016

Semma Therapeutics Announces $5 Million Grant from California Institute of Regenerative Medicine (CIRM) to Develop Novel Stem-Cell Derived Treatment for Diabetes

CAMBRIDGE, Mass., September 12 th , 2016 – Semma Therapeutics, a biopharmaceutical company developing cellular therapy for diabetes, announced today that it has accepted a $5 million grant from the California Institute of Regenerative Medicine (CIRM). The award provides funding to develop personalized cell therapy for the treatment of diabetes. Diabetic patients’ own cells will be collected, transformed first into stem cells and then differentiated into stem-cell derived pancreatic islets (SC-islets) to use as an investigational cell therapy for diabetes.

The worldwide diabetes epidemic takes a large toll on individual patients, families, and communities, and significantly impacts patient quality of life, increasing risk for serious health complications and early mortality. The transplantation of human cadaveric islet cells into patients with diabetes has proven to be an effective treatment for the disease, with a substantial number of patients achieving multi-year insulin independence. However, cadaveric islets are scarce and variable in quality, and patients must undergo lifelong immunosuppression to receive these allogeneic islets.

“The grant from CIRM funds a program designed to overcome these challenges,” said Robert Millman, co-founder and CEO of Semma. Using technology developed in the lab of Dr. Douglas Melton of Harvard University and licensed and further developed by Semma, unlimited supplies of pancreatic islets can be created from pluripotent stem cells. “Using this technology, the challenges of supply are overcome. By deriving these stem cells from a patient’s own blood or skin, the challenges of allogeneic immuno- rejection are also circumvented,” added Millman.

The program begins with patient evaluation and selection at UCLA. Peter Butler, M.D., Director of the Hillblom Islet Research Center, UCLA, is responsible for the clinical evaluation and selection of patients for participation in this program. His team will also collaborate with Semma to evaluate the differentiation of each patient’s stem cells into SC-islet cells. “I am thrilled to be a part of this program that will leverage the scientific, manufacturing, regulatory and clinical expertise of the Semma team, as well as scientists and clinicians at leading academic medical facilities in California,” said Butler.

At Cedars-Sinai in Los Angeles, high-quality pluripotent stem cells will be derived from each patient’s blood and then analyzed. This work will be performed at the Induced Pluripotent Stem Cell Core Facility, directed by Dhruv Sareen, PhD, at the Cedars-Sinai Board of Governors Regenerative Medicine Institute. Following this process, the stem cells will be transferred to City of Hope (COH), a premier site for manufacturing of stem cell derived products for Phase 1/2 clinical trials. The goal of this program is to generate suitable clinical grade differentiated SC-islet cells and to establish a path leading to the transplantation of these cells back into patients in a clinical trial. ​

About Semma Therapeutics
Semma Therapeutics was founded to develop transformative therapies for Type 1 diabetes patients. Work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing islet cells in the laboratory. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of- the-art device to provide a true replacement for the missing islet cells in a diabetic patient without immunosuppression. Semma Therapeutics is working to bring new therapeutic options to the clinic and ​ improve the lives of patients with diabetes. The company is headquartered in Cambridge, MA.

About the Larry L. Hillblom Islet Research Center at UCLA
The Larry L. Hillblom Islet Research Center at UCLA (LHIRC) was opened in November 2004 and was the first research center built specifically to study islet biology. LHIRC and was made possible by a grant from the Larry Hillblom Foundation. Its mission is to provide excellent training for young scientists from around the world, and in so doing, to understand the causes of islet cell destruction leading to diabetes and to understand how islet cells can be replaced. The overriding aim of LHIRC at UCLA is to contribute towards and provide leadership in the worldwide efforts that eventually will lead to the prevention and cure of diabetes.

About City of Hope
City of Hope is an independent research and treatment center for cancer, diabetes and other life- threatening diseases. Designated as one of only 45 comprehensive cancer centers, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research and treatment protocols that advance care throughout the world. City of Hope is located in Duarte, California, just northeast of Los Angeles, with community clinics throughout Southern California. It is ranked as one of "America's Best Hospitals" in cancer by U.S. News & World Report. Founded in 1913, City of Hope is a pioneer in the fields of bone marrow transplantation, diabetes and numerous breakthrough cancer drugs based on technology developed at the institution. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

About Cedars-Sinai
Cedars-Sinai is a leader in providing high-quality healthcare encompassing primary care, specialized medicine and research. Since 1902, Cedars-Sinai has evolved to meet the needs of one of the most diverse regions in the nation, setting standards in quality and innovative patient care, research, teaching and community service. Today, Cedars-Sinai is known for its national leadership in transforming healthcare for the benefit of patients. Cedars-Sinai impacts the future of healthcare by developing new approaches to treatment and educating tomorrow’s health professionals. Additionally, Cedars-Sinai demonstrates a commitment to the community through programs that improve the health of its most vulnerable residents. For more information, visit www.cedars-sinai.edu ​

About CIRM
CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding