Fate Therapeutics gets US patent for class of small molecule modulators of Rho-associated kinase

The newly issued patent claims a class of small molecule inhibitors of Rho-associated kinase (ROCK) that are crucial to the therapeutic application of human induced pluripotent stem cells (hiPSCs).

Modulators belonging to the patented class have been shown to be necessary for the high-throughput derivation of transgene-free hiPSCs, and for the maintenance, survival and genomic stability of hiPSCs in culture. Fate Therapeutics holds an exclusive license from The Scripps Research Institute (TSRI) to the patent in all commercial fields.

Fate Therapeutics COO and CFO Scott Wolchko noted that the company believes the class of ROCK inhibitors covered by these issued claims is a key component to enable the clinical manufacture of hiPSCs for therapeutic applications.

"These newly issued claims complement the hiPSC-related cell compositions previously patented under our Whitehead Institute for Biomedical Research intellectual property portfolio, which we believe are foundational to hiPSC generation.

"Our growing patent estate covering small molecule-enhanced reprogramming and pluripotency maintenance and expansion is a reflection of the breadth of our proprietary platform for developing iPSC-based regenerative therapeutics," Wolchko added.

Thiazovivin, one specific ROCK inhibitor covered within the patented class, was first discovered by Dr Sheng Ding, a scientific founder of Fate Therapeutics. Under a research collaboration between TSRI and Fate Therapeutics, Dr Ding and his team first demonstrated that Thiazovivin, in combination with other small molecules, increases the reprogramming efficiency of human fibroblasts into hiPSCs by 200-fold as compared to non-chemically enhanced methods of hiPSC generation (Lin, T., et al, Nature Methods 6, 805 – 808 (2009)).

Additional work by both Dr Ding and Fate Therapeutics further differentiated the ability of Thiazovivin and its related class of molecules, as compared to other ROCK inhibitors, to significantly enhance hiPSC survival and homogeneity.

Thiazovivin, as well as compositions and cell culture media comprising Thiazovivin, are patented under U.S. Patent No. 8,044,201 entitled Stem Cell Cultures, to which Fate Therapeutics holds an exclusive license from TSRI in all commercial fields.

In March 2014, Fate scientists published an article in the journal Stem Cell Reports describing its proprietary stem cell modulation platform for developing hiPSC-based regenerative therapeutics. The company’s therapeutic platform consists of stage-specific cell culture systems containing combinations of small molecule modulators including ROCK inhibitors, and enables the rapid, parallel derivation of hiPSC clones and their subsequent expansion and survival as transgene-free, single cells in culture.

Fate Therapeutics is currently researching therapeutic applications of hiPSC-derived myogenic progenitor cells and hematopoietic cells.