Developing Genetically Modified Muscle Progenitor Cells
In the world of stem cells, there are a number of well-recognized market participants, including Cellular Dynamics International (CDI), Ocata Therapeutics, Mesoblast, and others. These companies receive a great deal of attention from the stem cell community.
At the same time, there are occasionally brilliant companies that quietly operate under the radar screen, building themselves up one intelligent and methodical step at a time.
A company which precisely fits this description is Stem Cell Medicine Ltd. (www.stemcell-medicine.com), a novel regenerative medicine company headquartered in Jerusalem, Israel.
Founded in 2010, Stem Cell Medicine’s mission is to “develop and commercialize cellular therapies in combination with pharmaceutical products to be used for tissue repair and for the treatment of inflammatory, immunological and neurological disorders.”
Advancing Cellular Therapies in Combination with Pharmaceutical Products
Stem Cell Medicine Ltd. was exceedingly fortunate that founder investments provided the funds necessary for immediate and expansive infrastructure build-out.
Research faculty planning and construction occurred over a multi-year period, with the majority of the activity occurring between 2010 and 2012. Since that time, the company has shifted its focus toward in-house development of cellular therapies in combination with pharmaceutical products.
Most start-up and tech companies go through the opposite process. They often start operations within an incubator or “business park,” then proceed with investor fundraising, move onto product development, and finally (if cash flow allows), invest into company infrastructure and facilities.
Because of its unusual up-front investment into infrastructure and research facilities, Stem Cell Medicine has an enviable strategic edge within the marketplace, allowing it to plan first and act second.
The “Founding Father” of Stem Cell Medicine, Ehud Marom
Stem Cell Medicine was founded by Ehud Marom, a stem cell pioneer whose professional career has centered on building innovative companies structured around a strong technical framework. To most, Mr. Marom is best known for his involvement as CEO of Gamida Cell, where he directed the development of Gamida Cell’s flagship product from the pre-clinical stage through the beginning of Phase II trials.
He is also known for his involvement as VP of two divisions at Teva Pharmaceuticals Ltd., where he led the development and market launch of Copaxone. He was also the CEO of Makhteshim Ltd. He now serves as the Chairman of Stem Cell Medicine and Pharma Two B and is CEO and Chairman of Mapi Pharma.
While Mr. Marom’s vision has driven prolific research, collaboration, and partnering activity within Stem Cell Medicine Ltd. from 2010 to present, arguably Marom’s best skill is his ability to assemble a world class team.
A key team member driving the scientific innovation within the company is Dr. Frida Grynspan, Vice President of Research and Development and Site Manager for Stem Cell Medicine. Educated at the University of Illinois and Harvard University, Dr. Grynspan previously worked at two other stem cell powerhouses, Pluristem Therapeutics and Gamida Cell. She has also held positions at other leading companies within the regenerative medicine sector, including Collplant and Inteligene.
Interview with Dr. Frida Grynspan, VP of R&D and Site Manager for Stem Cell Medicine Ltd.
I had the distinct honor of interviewing Dr. Grynspan about a newly announced partnership that will align Stem Cell Medicine (SCM) with Ramot at Tel Aviv University Ltd. (Ramot) and Yeda Research and Development Company Ltd. (YEDA) to support the development of stem cell-based treatments for neuronal diseases.
Specifically, the groups have entered into a license and research funding agreement in which Stem Cell Medicine will fund research at Tel Aviv University led by Professor Dani Offen, supported by Professor David Yaffe of the Weizman Institute of Science. YEDA Research and Development Company is the commercial arm of the Weizman Institute of Science. As such, it functions to support technology transfer from the institute into the private marketplace.
Dr. Grynspan described the collaboration between Professors Offen and Yaffe as a “powerful synergy,” providing greater innovation than either researcher would be likely to produce independently.
The partnership, funded by Stem Cell Medicine, is intended to generate potential commercial products. Stem Cell Medicine will “hold the exclusive worldwide license for the development and commercialization of the resulting technology and therapeutic product pipeline.”
Specifically, the partnership will center around the development of allogeneic (“from another human”) progenitor muscle cells that are genetically-modified to secrete four neurotrophic factors: BDNF, GDNF, VEGF, and IGF-1.
Neurotrophic factors are family of proteins that play a critical role in regulating growth and survival of developing neurons, as well as maintaining adult neurons in a healthy state. Importantly, a growing body of research has validated that neurotrophic factors can facilitate the growth and development of neurons within both the central (CNS) and peripheral nervous systems (PNS).
Furthermore, during in vitro (“laboratory”) testing and in vivo (“animal testing”), neurotrophic factors have been able to rescue damaged neurons, making neurotrophic factors of substantial interest to Dr. Grynspan and her team as a therapeutic agent.
However, one of the key issues to date has been identifying which neurotrophic factors to utilize, in what ratios, and in what combinations. This is what makes this specific research collaboration so interesting. Stem Cell Medicine and its research partners, Ramot and YEDA, have identified that “neuroprotective effects occur when the neurotrophic factors are given together.”
For this reason, genetically-modified progenitor muscle cells are required to produce the four key neurotrophic factors of interest: BDNF, GDNF, VEGF, and IGF-1. When secreted in sufficient quantities, these neurotrophic factors can interact to influence regenerative and survival conditions within their local environment, thereby potentially influencing the growth and proliferative potential of nearby cells.
According to Dr. Grynspan, “This is an interesting platform technology for us, because we can enter into neuromuscular diseases.” She further elaborated by saying, “In a way, this platform technology will create ‘super cells,’ cells that have the capacity to affect their environment. When viewed in this manner, it is a natural progression of technology.”
According to Dr. Grynspan, “Stem Cell Medicine’s ex vivo process of creating the genetically modified muscle progenitor cells also gives them a very strong safety profile. Stem Cell Medicine is uniquely suited to the task of commercializing this promising technology platform, because the company has “state-of-the-art R&D laboratories and modern GMP manufacturing production rooms that, together with well-equipped analytical laboratories, provide an optimal environment for the development of products from inception to the clinic.”
Indeed, few stem cell companies have the facilities and infrastructure to take a product from conception, through development, clinical testing, and finally, into production.
Dr. Grynspan described Stem Cell Medicine’s facilities as very unique, adding that the intent is to “establish proof-of-concept, move the applications of the technology into the clinical testing, and finally, pass them through the regulatory pathways for approval.”
In addition to local Israeli markets, the company will consider other favorable markets as well for product approvals, including the European Union (EU) and Japan.
Finally, Stem Cell Medicine’s business plan calls for the engagement in collaboration with other companies and research institutions, which further aligns this collaboration with the company’s business strategy. The company plans to begin a financing round in 2016/2017.
When asked about the advantages or disadvantages of being located in Israel, Dr. Grynspan reflected, “Israel is small country with active research universities that are very close together. Everything is condensed within the country, making for a very collaborative environment.”
To learn more about Stem Cell Medicine, visit the company at www.stemcell-medicine.com. For more information about collaborations and partnerships, please contact Dr. Grynspan.
For information on the partnership between Stem Cell Medicine, Ramot, and Yeda, view the press release “Stem Cell Medicine Signs License and Research Agreement with Ramot and Yeda to Develop Stem Cell-Based Treatments for Neuronal Disease.”