Catalyst to ‘industrialize’ stem cell tech

Fate Therapeutics and Stemgent establish consortium to offer stem cell advances to the research market

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LA JOLLA, Calif.—Fate Therapeutics and Stemgent Inc. have formed a consortium, dubbed Catalyst, that will allow a select number of scientists to have access to the induced pluripotent stem (iPS) cells, as well as the knowledge surrounding them, that has been amassed by the partners.

According to Scott Wolchko, CFO of Fate Therapeutics, the work of Fate's and Stemgent's scientific founders—including Dr. Sheng Ding's breakthrough protein-based reprogramming technology—will be made available on an exclusive basis to Catalyst members for drug discovery. Ding, an associate professor of The Scripps Research Institute and scientific founder of both Fate Therapeutics and Stemgent, is the first to report the creation of iPS cells using cell-penetrating proteins, a technique that effectively eliminates any risk of genetic modification.

Catalyst will also include pioneering discoveries by Dr. Rudolf Jaenisch, a founding member of the Whitehead Institute, scientific founder of Fate Therapeutics and scientific advisory board member of Stemgent, who recently produced the first Parkinson's disease patient-derived iPS cells free of viral reprogramming factors.

Wolchko says the mission of Catalyst is to continue to develop and "industrialize" iPS technology and to provide these technologies for drug discovery to its members. 

"This includes sourcing cells from diverse genetic backgrounds; reprogramming these cells in a minimally-invasive, efficient, scalable manner without genetic modification; developing SOPs to create differentiated cells that are quantitated and characterized; and supplying high quality, commercial-grade iPS cell materials in large quantities with consistency and reliability," he says.

In exchange for providing funding to participate in the consortium, chosen companies and academics get access to cutting-edge cells and drug discovery tools that they can use to find and test new drugs.

"And they'll have access to technologies that other people, or companies, will not have," adds Ian Ratcliffe, Stemgent's CEO.

Ratliffe points out that the creation of Catalyst represents the first time many of these technologies will be made available to industry.

Paul Grayson, Fate Therapeutics' president and CEO, notes that Catalyst is the sole industry opportunity to access the most advanced iPS cell technology platform.

"The Catalyst program represents a unique business collaboration amongst its participants to accelerate the translation of iPS cell technology into the discovery and development of safer and more effective therapeutics," he says.

Wolchko says iPS cells are considered to be of great potential for toxicity testing, disease research and primary drug screening. 

"The ability to create and study normal versus disease cell types, model diseases and their progression and identify underlying disease mechanisms represents a very powerful platform to discover new drug candidates as well as to develop safer medicines," he says.  "Under Catalyst, these pre-competitive drug discovery technologies will be advanced using 'pooled' dollars from its members, thereby expanding the scope of development and deliverables beyond what would otherwise be possible with a single participant. In this fashion, Members will be able to leverage their R&D funding and realize a multiplier return on the investment for the iPS cell platform technology."

iPS cells are made by "reprogramming" fully differentiated mature cells, such as adult skin cells, to become pluripotent or stem-cell like and do not rely on the use of stem cells derived from embryos. Because iPS cells can differentiate into any cell type, iPS cells are considered of great importance to medicine because they can be used to model human diseases, discover and test drug candidates and develop personalized cell therapies. Previously, iPS cell generation required the delivery of genetic material through viruses or plasmids, which carry risks from insertion and are time-consuming, inefficient methods.

Any improvement of the cells, or development of scientific tools using them, that one company might make will benefit the other members of the consortium, adds
Grayson.

"Catalyst's technology—creating and differentiating iPS cells into distinct cell types—will uncover the essential epigenetic and gene expression profiles associated with the derivation of specific cell lineages," says Grayson. "These same technologies will prove critical in our discovery and development of conventional pharmaceuticals to guide cell fate for therapeutic benefit."

In addition to funding, Stemgent and Fate will reap additional benefits for the efforts they sow.

Stemgent, which makes tools for drug discovery, will gain insight from potential industry clients into the kinds of products that the company needs to develop, Ratcliffe said.

Meanwhile, Fate will have validation of its stem cells from several corporate partners, and input from experts with different specialties on the specifics of how the cells develop. Companies that are interested in becoming Catalyst members may contact Fate to discuss the opportunity.

"Fate Therapeutics and Stemgent are committed to advancing the best iPS technology and to provide Members the necessary intellectual property, know-how, tools and support to establish—within each member's own R&D department—the next paradigm of drug discovery for the development of novel, safer and more effective medicines for patients," adds Wolchko.


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