Fate Therapeutics Announces FDA Clearance of Landmark IND for FT500 iPSC-derived, Off-the-Shelf NK Cell Cancer Immunotherapy

Company to Initiate First-ever U.S. Clinical Investigation of iPSC-derived Cell Product

Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has allowed its Investigational New Drug (IND) Application for FT500, the Company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line. The clinical trial of FT500 is expected to be the first-ever clinical investigation in the U.S. of an iPSC-derived cell product.

“The clearance by the FDA of our FT500 IND is a significant milestone and marks the beginning of an exciting new era for the clinical development of cell products,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “Clonal master iPSC lines are a renewable cell source that can uniquely produce cell products which are uniformly engineered and well characterized, can be mass produced in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. This revolutionary paradigm overcomes significant challenges that limit both patient- and donor-derived cell therapy, where heterogeneous populations of primary cells are repeatedly sourced, engineered, expanded and characterized on a batch-by-batch basis resulting in cell therapies with substantial variability in quality, consistency and potency.”

The Company plans to initiate first-in-human clinical testing of FT500 in combination with checkpoint inhibitor therapy for the treatment of advanced solid tumors. This study is expected to evaluate the safety and tolerability of multiple doses of FT500, in multiple dosing cycles with nivolumab, pembrolizumab or atezolizumab, in subjects that have progressed or failed on checkpoint inhibitor therapy.


Press Release here

Also recent news from Fate on their partnership with Japan for CAR-T Cell Cancer Immunotherapies based on Fate's iPSC technology here.

Further, Fate licenses new route to master iPS cell lines:

Fate PR announcement here

Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders. Its first-in-class cell therapy products undergoing clinical development today utilize healthy donor cells, which we modify ex vivo using pharmacologic modulators, such as small molecules, to improve the cells’ biological properties and therapeutic function. They're also pioneering a revolutionary approach to cell therapy – we use renewable master induced pluripotent stem cell (iPSC) lines generated from our proprietary iPSC product platform to derive cell therapy products that can be delivered off-the-shelf for the treatment of a large number of patients.

Fate's cells of interest are the cells of the immune system. They're cell therapy product pipeline is comprised of immuno-oncology programs, including off-the-shelf NK- and T-cell products derived from master iPSC lines, and immuno-regulatory programs, including products to prevent life-threatening complications in patients undergoing hematopoietic cell transplantation and to promote immune tolerance in patients with autoimmune disease.

Fate's cell therapy product pipeline is comprised of first-in-class cellular immunotherapies for cancer and immune disorders. Their programs reflect our dedication and commitment to pioneering ground breaking science to address severe, life-threatening diseases where the unmet need is significant and the treatment options are limited. Source & for further info: www.fatetherapeutics.com

iPSC Platform: Platform for Induction and Maintenance of Transgene-free hiPSCs Resembling Ground State Pluripotent Stem Cells
NK cancer paper: Human iPSC-Derived Natural Killer Cells Engineered with Chimeric Antigen Receptors Enhance Anti-tumor Activity

To provide an overview of the work Fate is undertaking, I've added below an interview with one of the leading scientists behind Fate's push forward using iPSC derived NK cells for cancer.

Natural killer cells as effective as, less toxic than T cells

Natural killer cells engineered with chimeric antigen receptors demonstrated comparable efficacy as T cells but appeared less toxic, according to study results.

The findings could be significant, given the advantages that natural killer cells offer.

For example, engineered natural killer cells could be delivered safely in an off-the-shelf manner, and research so far suggests natural killer cells do not trigger the same potentially severe toxicities — such as organ damage, neurotoxicity or death — that have been associated with use of re-engineered T-cells.

The results — obtained from studies in mice — have laid the groundwork for a planned trial in humans.

“One of the main challenges of immunotherapy has been the clinical manufacture of modified cells,” Dan S. Kaufman, MD, PhD, professor of medicine in the division of regenerative medicine and director of cell therapy at UC San Diego School of Medicine, as well as a HemOnc Today Editorial Board Member, said in a press release. “We have shown that we can engineer [human-induced pluripotent stem cells] and create chimeric antigen receptor-expressing natural killer cells to better target refractory cancers that have resisted other treatments.”

HemOnc Today spoke with Kaufman about the research his team has conducted into re-engineering natural killer cells with chimeric antigen receptors, the advantages to using natural killer cells instead of T cells, the potential implications if this approach is proven effective and safe, the malignancies for which this approach may offer the greatest benefit, and the next steps in research.

Question: Can you explain the rationale for this approach?

Answer: Natural killer cells are a population of lymphocyte immune cells that are separate from T cells. They are known to be a part of the immune system and are known to kill tumor cells and virally infected cells. Clinical studies with natural killer cells have been conducted for more than 15 years, and they have shown that allogeneic natural killer cells have been effective for treating certain cancer types — primarily leukemia, and specifically acute myeloid leukemia.

For the full interview from HemOnc Today please link here