AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells – Yahoo…

AgeX Therapeutics, Inc. (NYSE American: AGE) and Lineage Cell Therapeutics, Inc. (NYSE American and TASE LCTX), announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" covering what is commonly designated "induced Pluripotent Stem (iPS) cells. The issued claims include methods to manufacture pluripotent cells capable of becoming any cell in the body. The patent has an early priority date, having been filed before the first scientific publication of Shinya Yamanaka, for which he won the Nobel Prize for Physiology or Medicine in 2012.

"This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state," said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. "Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development." A video describing the significance of the patent in AgeXs product development is available on the AgeX website.

"The issuance of this patent highlights Lineages dominant position in the field of cell therapy," stated Brian M. Culley, CEO of Lineage. "Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success."

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been "reprogrammed" or "induced" to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

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For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

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Contacts

Media Contact for AgeX:Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells - Yahoo...

Patent Granted To Lineage & AgeX – Anti Aging News

Lineage Cell Therapeutics and AgeX Therapeutics have been awarded a United States Patent and Trademark Office patent for Methods Of Reprogramming Animal Somatic Cells.

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr Michael D West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Patent 10,501,723 covers induced pluripotent stem cells which includes methods to manufacture iPSs cells that are capable of becoming any cell within the body. This patent has an early priority date having been filed before the first scientific publication, and was assigned to Advanced Cell Technology of Marlborough, Massachusetts and licenced to Lineage as well as being sublicensed to Age X for defined fields of use.

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Patent Granted To Lineage & AgeX - Anti Aging News

Charles River Announces Strategic Partnership with Bit Bio, Increasing Portfolio of Translational Drug Discovery Technologies – BioSpace

Dec. 10, 2019 13:00 UTC

WILMINGTON, Mass.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) today announced that it has entered into an exclusive Discovery and Safety Services partnership with Bit Bio, a company that offers consistent and efficient reprogramming of human cells for use in research, drug discovery, and cell therapies.

By applying an engineering approach to synthetic and stem cell biology, Bit Bio has developed proprietary technologies for the efficient, consistent, and scalable reprogramming of induced pluripotent stem cells.

Cellular Reprogramming

Cellular reprogramming is the process by which human stem cells, given a precise set of genetic instructions, differentiate into a desired cell type. Current cellular reprogramming approaches are inefficient, with low cell yields, creating a gap for applications requiring high quality, consistent, and pure human cells.

To overcome this hurdle, Bit Bio has developed a gene engineering approach, opti-ox (optimised inducible over-expression). This platform, validated on both muscle and brain cells, enables precise, controllable stem cell reprogramming. According to Bit Bio, the process is more efficient and scalable than available technologies in transforming stem cells into desired cell types.

By combining the purity, scale, and speed of the opti-ox platform with deep learning algorithms, Bit Bio has the potential to accelerate the discovery and application of every single human cell type.

Partnering for Translational Drug Development

By partnering with Bit Bio, Charles River plans to offer clients access to an expanding suite of authentic human cells through their use in target discovery, validation and screening services. In drug discovery and safety, the use of high quality, authentic human cells at scale will enable the development of therapies with a higher chance of success in patients. Additionally, through the partnership, Charles River will contribute to the development and validation of novel cell lines.

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About Charles River

Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.

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Charles River Announces Strategic Partnership with Bit Bio, Increasing Portfolio of Translational Drug Discovery Technologies - BioSpace

Fate Therapeutics Presents its First Off-the-shelf, iPSC-derived CAR T-Cell Cancer Immunotherapy Program at ASH Annual Meeting – GlobeNewswire

FT819 Exhibits Enhanced Tumor Clearance In Vivo Compared to Primary CAR T Cells in Preclinical Leukemia Model

Master Engineered iPSC Line for FT819 Fully Characterized for Complete Elimination of TCR Expression and Integration of Novel 1XX CAR into TRAC Locus with No Evidence of Off-target Effects

Company Plans to Submit an IND Application for FT819 during 1H20

SAN DIEGO, Dec. 10, 2019 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced new in vivo preclinical data for FT819, its first off-the-shelf, iPSC-derived chimeric antigen receptor (CAR) T-cell product candidate, at the 61st American Society of Hematology (ASH) Meeting and Exposition in Orlando, Florida.

FT819 is derived from a clonal master engineered induced pluripotent stem cell (iPSC) line with complete elimination of T-cell receptor (TCR) expression and a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus. The cell product candidate is being developed under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D. The Company has now selected a single engineered iPSC clone, and generated and fully-characterized the master engineered iPSC bank for GMP production of FT819.

CAR T-cell therapy continues to deliver remarkable outcomes for patients with hematologic malignancies, and next-generation approaches are needed to enable broad and timely patient access and reduce the cost and complexity of therapy, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. With early evidence of clinical activity for our off-the-shelf, iPSC-derived NK cell programs, we are excited to lead in bringing next-generation CAR T-cell therapies to patients and plan to submit an IND for FT819 in the first half of 2020.

The Companys iPSC product platform unites stem cell biology and precision genetic engineering to create renewable master engineered iPSC lines that can be repeatedly used to mass produce cancer-fighting immune cells, replacing the high production costs, weeks of manufacturing time, and complex engineering processes required for current-generation CAR T-cell immunotherapies with an off-the-shelf product that has the potential to reach many more patients.

At ASH, scientists from the Company and MSK presented new in vivo preclinical data demonstrating that FT819 exhibits durable tumor control and extended survival. In a stringent xenograft model of disseminated lymphoblastic leukemia, FT819 demonstrated enhanced tumor clearance and control of leukemia as compared to primary CAR19 T cells. At Day 35 following administration, a bone marrow assessment showed that FT819 persisted and continued to demonstrate tumor clearance, whereas primary CAR T cells, while persisting, were not able to control tumor growth. Over the past twelve months, the collaboration team has worked to optimize its processes for making T cells from iPSCs, and has now shown the production of pure T-lymphocytes consisting of both CD8+ and CD4+ T cells having a global gene expression profile that is highly-similar to primary T cells based on a principal component analysis.

As proof-of-principle for the unique advantages arising from selecting a single engineered iPSC clone for the production of CAR T-cell therapy, the scientists assessed 747 clones after engineering a pool of cells using CRISPR. It was found that only about 2% of clones met the Companys standards for overall quality including containing both bi-allelic disruption of the TCR, proper insertion of the CAR into the TRAC locus without random transgene integrations, and no evidence of off-target genomic modifications or translocations. The Company selected the top-performing clone for generation of the master engineered iPSC bank for GMP production of FT819.

Fate Therapeutics has exclusively licensed from MSK foundational intellectual property covering the production and composition of iPSC-derived T cells. In August, the Company announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell, including an iPSC. The foundational patent, which expires in 2034, is owned by MSK and is licensed exclusively to Fate Therapeutics for all human therapeutic uses.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Companys cell product candidates, including FT819, its ongoing and planned clinical studies, and the expected clinical development plans for FT819. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company may cease or delay planned development and clinical trials of any of its product candidates for a variety of reasons (including any delay in enrolling patients in current and planned clinical trials, requirements that may be imposed by regulatory authorities on the conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, or the occurrence of any adverse events or other negative results that may be observed during development), the risk that results observed in preclinical studies of its product candidates, including FT819, may not be replicated in future clinical trials or studies, and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Presents its First Off-the-shelf, iPSC-derived CAR T-Cell Cancer Immunotherapy Program at ASH Annual Meeting - GlobeNewswire

Fate Therapeutics Announces New Preclinical Data for FT596 Off-the-Shelf, iPSC-derived CAR NK Cell Cancer Immunotherapy – Benzinga

FT596 as a Monotherapy Demonstrates Comparable Anti-tumor Activity to CAR19 T Cells In Vivo in Humanized Mouse Model of Lymphoma

Combination of FT596 with Rituximab Shows Durable Tumor Clearance In Vivo in Preclinical Lymphoma Model

Company Plans to Initiate Enrollment of First-in-human Clinical Trial of FT596 in Early 2020

SAN DIEGO, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced new in vivo preclinical data for FT596, its off-the-shelf, multi-antigen targeting natural killer (NK) cell product candidate derived from a clonal master engineered induced pluripotent stem cell (iPSC) line. The data were featured during the 61st American Society of Hematology (ASH) Meeting and Exposition as part of the organization's CAR-T and Beyond press program, which spotlighted promising next-generation cancer immunotherapies having the potential to overcome the key limitations of patient-specific chimeric antigen receptor (CAR) T-cell therapy.

"Current patient- and donor-specific CAR T-cell immunotherapies recognize only one antigen and fail to address the significant risk of relapse due to antigen escape. FT596 is ground-breaking in that it is designed to be available off-the-shelf for timely patient access and to promote deeper and more durable responses by targeting multiple tumor-associated antigens," said Bob Valamehr, Ph.D., Chief Development Officer of Fate Therapeutics. "Additionally, since FT596 is manufactured from a renewable master engineered iPSC line, the complexities of patient-by-patient genetic engineering and production are greatly reduced and, for the first time, we are able to mass produce multi-functional cellular immunotherapies in a uniform and cost-effective manner."

FT596 is the first cellular immunotherapy engineered with three active anti-tumor components to be cleared for clinical investigation by the FDA. In addition to a proprietary CAR targeting CD19, FT596 expresses a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor that has been modified to augment antibody-dependent cellular cytotoxicity, enabling coincident targeting of CD19 and additional tumor-associated antigens such as CD20. FT596 also expresses an interleukin-15 receptor fusion (IL-15RF), a potent cytokine complex that promotes survival, proliferation and trans-activation of NK cells and CD8 T cells without the need for systemic cytokine support. Together, these features of FT596 are intended to maximize potency and minimize toxicity in treated patients. The Company plans to initiate enrollment of a first-in-human clinical trial of FT596 in early 2020.

New preclinical data presented at ASH showed that FT596 administered as a monotherapy exhibited durable tumor clearance and extended survival in vivo similar to primary CAR T cells in a humanized mouse model of CD19+ lymphoma. Additionally, when combined with the anti-CD20 monoclonal antibody rituximab, FT596 showed enhanced killing of CD20+ lymphoma cells in vivo as compared to rituximab alone. These data confirm previously presented in vitro findings that demonstrate the unique multi-antigen targeting functionality of FT596, and the product candidate's potential to effectively overcome CD19 antigen escape.

The Company also announced that, in preparation for Phase 1 initiation, it had recently completed GMP production of FT596. In a single small-scale manufacturing campaign, the Company produced over 300 cryopreserved, infusion-ready doses of FT596 at a cost of approximately $2,500 per dose. The Company's iPSC product platform unites stem cell biology and precision genetic engineering to create renewable master engineered iPSC lines that can be repeatedly used to mass produce cancer-fighting immune cells, replacing the high production costs, weeks of manufacturing time, and complex manufacturing processes required for current-generation CAR T-cell immunotherapies with a lower-cost, easier-to-manufacture, well-characterized, off-the-shelf product that has the potential to reach many more patients.

FT596 is the third off-the-shelf, iPSC-derived NK cell product candidate from the Company's proprietary iPSC product platform cleared for clinical investigation by the FDA in the past twelve months. On Saturday, the Company announced that the first patient treated for acute myeloid leukemia in its Phase 1 clinical trial of FT516, an off-the-shelf, iPSC-derived NK cell cancer immunotherapy engineered to express hnCD16, showed no morphologic evidence of leukemia, chimerism of FT516 in the bone marrow, and hematopoietic recovery, including complete neutrophil recovery without growth factor support (>1,000 per L), after receiving three once-weekly doses of FT516 and IL-2 cytokine support.

About Fate Therapeutics' iPSC Product PlatformThe Company's proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company's first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company's platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics' iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.

About FT596FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology, which contains a NKG2D transmembrane domain, a 2B4 co-stimulatory domain and a CD3-zeta signaling domain, that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 Fc receptor that has been modified to augment antibody-dependent cellular cytotoxicity by preventing CD16 down-regulation and enhancing CD16 binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that promotes enhanced NK cell activity. The FDA has allowed investigation of FT596 in an open-label Phase 1 clinical trial as a monotherapy, in combination with rituximab for the treatment of advanced B-cell lymphoma, and in combination with obinutuzumab for the treatment of chronic lymphocytic leukemia. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and CD16 receptors, in combination with IL-15RF signaling, convey synergistic anti-tumor activity. Increased degranulation and cytokine release were observed upon dual receptor activation in lymphoma cancer cells as compared to activation of each receptor alone, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a mixed cellular composition cytotoxicity assay comprised of CD19+ and CD19- tumor cells, FT596 combined with CD20-directed monoclonal antibody therapy effectively eliminated the heterogeneous population of tumor cells, a result that was not observed with single-antigen targeted CAR19 T cells.

About FT516FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 Fc receptor, which has been modified to prevent its down-regulation and enhance its binding to tumor-targeting antibodies. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma (clinicaltrials.gov ID number NCT04023071). CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. In addition, ADCC is dependent on NK cells maintaining active levels of CD16 expression, and the expression of CD16 on NK cells has been shown to undergo considerable down-regulation in cancer patients, which can significantly inhibit anti-tumor activity.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company's immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Company's immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Company's NK cell product candidates, including FT596 and FT516, its ongoing and planned clinical studies, and the expected clinical development plans for FT596 and FT516. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company may cease or delay planned development and clinical trials of any of its product candidates for a variety of reasons (including any delay in enrolling patients in current and planned clinical trials, requirements that may be imposed by regulatory authorities on the conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Company's product candidates for clinical testing, or the occurrence of any adverse events or other negative results that may be observed during development), the risk that results observed in preclinical studies of its product candidates, including FT596 and FT516, may not be replicated in ongoing or future clinical trials or studies, and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company's periodic filings with the Securities and Exchange Commission, including but not limited to the Company's most recently filed periodic report, and from time to time in the Company's press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Announces New Preclinical Data for FT596 Off-the-Shelf, iPSC-derived CAR NK Cell Cancer Immunotherapy - Benzinga

Induced Pluripotent Stem Cells Market Assessed To Tell Apart High Growth By Implies In 2018 to 2026 – Wolf Mirror

The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met. Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition. This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification. This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation. A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strong CAGR of 7.0% from 2018 to 2026. In 2017, the market was worth US$ 1,254.0 Mn and is expected to reach US$ 2,299.5 Mn by the end of the forecast period in 2026.

Customization to be the Key Focus of Market Players

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products. The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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Induced Pluripotent Stem Cells Market Assessed To Tell Apart High Growth By Implies In 2018 to 2026 - Wolf Mirror

Kyoto University Seeking To Use Stem Cells On Knee Treatment – Anti Aging News

Kyoto University has asked the government of Japan for approval to conduct a human clinical trial involving transplanting cartilage made from induced pluripotent stem cells as an approach to treat damaged knee joints.

Led by Professor Noriyuki Tsumaki the research team will be culturing iPS cells to create cartilage tissue which will be used to transplant into knees; according to the University the team submitted the study plan to the health ministry on November 7, 2019 for review by its special panel.

Their treatment approach has already been tested in animal studies where it was found to be effective and shown to carry a low risk of rejection, cancerization, and fibrosis reaction, according to the Kyoto research team.

Their plan which includes its safety was approved in October by a University board, which is hoped to help treat patients with degenerated or damaged cartilage due to injuries or illness. Cartilage tissue covers joint bones and absorbs shock, joints are not able to move smoothly if the cartilage is damaged or if it turns fibrous with age.

The Kyoto team hopes that this treatment will be an improvement on current methods in which cartilage tissue is transplanted as it is hard to acquire tissues, and the transplanted tissues often tend to turn fibrous to help effectively treat damaged knee joints.

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Kyoto University Seeking To Use Stem Cells On Knee Treatment - Anti Aging News

Story of a 15-year old scientist: How it all began – EuroScientist

Children and young adults these days have awide range of possibilities how to spend their leisure time. Some of us like to watch movies, some of us enjoy playing aflute and some of us prefer to go laboratories, put on a white coat and carry out an experiment. Zuzana Hudov, a15-year old student from Slovakia, fell into the category of teenagers who preferred going to labs and make experiments. Even though this is just onestory it once may be astory of hundreds of young aspiring students.

As alittle child Zuzanawas not much different from all the other kids in the kindergarten. Shewas playful, energetic and endlessly curious about the world surrounding her. However, curiosity diverted from the behaviour of her classmates. Most of the kids liked to explore the outside world, yet Zuzanawas more fascinated about the things that could not be understood by just looking at them. Shewould spend hours and hours browsing through an old Encyclopedia, looking at the pictures of human body, homogenesis phases and even insect behaviour. Her restless mind caused her agreat deal of problems in kindergarten since sheoften refused to go to bed during the afternoon sleep-time and, often, she would even take abook from the small kindergarten library and read it, hidden under the duvet, while other kids were sleeping. When Zuzana was five, her mother realised that the kindergarten was noplace for her daugther, and therefore, with the approval of experts, Zuzana was admitted to primary school ayear earlier than regular . That was where the real journey started, says Zuzana.

The competitive atmosphere of school brought Zuzanas ambitions and her desire to thrive to life. Even though shewas more than ayear younger than her classmates, she always perfomed very well and was one of the best pupils in her class. Although Zuzanastill preferred to read books, shefelt aresponsibility for the community and mediated with teachers and pupils about potential improvements to make; therefore she was electedclass representative.

Until the age of eleven her hobbies were pretty general: reading, playing the piano and dancing. Zuzanas interests changed when she was in sixth grade, as at that time, biology and chemistry classes were added to school curriculum. Zuzana was fascinated by the two new subjects and used to spend several hours each day to read popular science books and magazines. At the age of twelve sheperformed her first study about human body mass index (BMI) and obesity and took part in a local competition.

Due to her ambition and success in and outside school, she got the chance to study at GBAS Suany, aprestigious Slovak bilingual grammar school, where she started at the age of thirteen, and joined classes with classmates 3 years older than her.

During the biology classes, especially on the topics of neuroscience and genetics, Zuzana realised that although her desire for more knowledge was being fulfilled, her ambitions to do her own investigations and actuallycontribute to science were not satisfied. While looking for opportunities to collaborate with reasearch labs, she discovered that in many Western countries it is not uncommon for youngstudents to doscientific internships. She started contacting research institutions in Slovakia, but never was selected because of her young age. Consequently she started to applyoutside her home country and was eventually accepted as a summer intern at Masaryk University in Brno, Czech Republic at the Department of Histology and Embryology.

The laboratories that gave Zuzana this opportunity was lead by Mgr. Da Bohaiakov, Ph.D. and focused on neurogenesis research. The project she worked on was the immunofluorescent analysis of in vitro neural rosette formation from induced pluripotent stem cells (iPSCs). The aim of her research was to analyse the markers of neural rosette formation, which is a2D in vitro model of human neurogenesis. During the neurulation phase of embryogenesis there are many things than may go wrong, which can lead to neural tube defects (NTDs), such as spina bifida. NTDs are very dangerous and cause alot of pain and suffering in an individuals life. However, these days we are not only unable to treat these disorders, but we are not even able to observe the neurulation process non-invasively. That is why scientists are trying to developwith the in vitro solutions, which neural rosettes might potentially be.

Zuzanas first laboratory internship marked some new beginnings in her life such as the her first successfull research project, and afirst scientific work that won the 3rd place at the national Stredokolsk Odborn innos (High School Scientific Activity). At the age of 15 shewas the youngest participant in history.

The success of her first project was the incentive for Zuzanato participate in more scientific activities; therefore during the following term she launched abiology club at her school, took part in an international DNA essay contest where she finished among the top ten participants and carried out more work in a laboratory and completed one more internship.

In spite of all her success Zuzana still feels the responsibility for her community and she thinks that if shecould achieve all of this, why not any other student in the world? This is the reason why she decided to publish her story

Iam certainly no better than any other child, she says, yet Iwas lucky, Ihad an idea and enough passion not to give up, even though the circumstances were against me.

Zuzana hopes that her story can motivate her fellow pupils and she urges parents to foster the curiosity and ideas of their children. Additionally she wants to send the message that is importnat to look outside your own surrounding and use the possibilities Europe of today gives to everyone.

By Zuzana Hudacova

Featured image credit: Zuzana Hudov

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Stemonix, Atomwise Team Up on Drug Discovery With MicroOrgans and AI – Xconomy

XconomySan Diego

Two venture-backed startups that have developed technologies intended to speed up the drug discovery and development process are combining those tools in a bid to achieve their goals together.

San Francisco-based Atomwise has developed deep learning techniques for use in structure-based small molecule drug discovery. Since 2012 it has raised more than $50 million to bolster the development and application of its tech, which has been used by pharmaceutical and agrochemical companies, as well as by universities and hospitals across 40 countries. It says its computational tools can quickly analyze billions of compounds and thereby speed up the process of identifying compounds that bind to disease-causing proteins.

Stemonix, which has offices in Maple Grove, MN, and in San Diego, has developed models for testing potential drugs from human induced pluripotent stem cells, creating what it calls living microtissues to mimic human organs, including the brain and heart. The startup reported raising a $14.4 million Series B earlier this year to speed the commercialization of its platforms. Its MicroOrgans technology provides 3D disease models that the company hopes will give researchers a more accurate indication of whether compounds are likely to work in humans.

This is especially difficult to determine when it comes to finding drugs that work on rare neurological diseases, which are tricky to replicate in animal models because of the complexity of the human brain, according to Stemonix.

Atomwise has struck a number of deals with biotech and pharma companies interested in its tech, including Atropos Therapeutics, Hansoh Pharma, and Eli Lilly (NYSE: LLY). Financial terms of this latest deal werent disclosed.

The companies plan to use Atomwises AI and Stemonixs human MicroBrain 3D disease model to target Rett syndrome, a severe neurological disorder caused by mutations in the MECP2 gene. The gene encodes a protein thats needed for the development of the nervous system and normal brain function. Babies with the condition, most often infant girls, typically develop normally for the first 6 to 18 months of life before symptoms kick in. Then, they experience a progressive loss of motor skills and speech.

Some medications are used to ameliorate their symptoms, but no FDA-approved treatment exists. A number of companies are in human testing with experimental drugs for the condition.

Those in late-stage testing include Newron Pharmaceuticals, which is developing an experimental drug called sarizotan that aims to reduce patients episodes of apnea, or breathing disturbances, and San Diegos Acadia Pharmaceuticals, which last year licensed trofinetide, an investigational treatment targeting the disease, from Australias Neuren Pharmaceuticals.

The UKs GW Pharmaceuticals (NASDAQ: GWPH) is studying non-psychoactive parts of cannabis, including cannabidiol and cannabidivarin, as potential treatments.

Novartis (NYSE: NVS) subsidiary AveXis is also advancing a gene therapy for Rett, but in August said that its advancement to human testing would be delayed so it could redo and add to preclinical studies. The decision was made following revelations that some of the animal data the company submitted to the FDA in support of onasemnogene abeparvovec (Zolgensma), the gene therapy it developed for babies with spinal muscular atrophy, had been manipulated. Novartis acquired AveXis in 2018.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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Channel in Nerve Cell May be Key in Unlocking Parkinson Disease Therapy – Pharmacy Times

Researchers at the University of Cologne have identified Cav2.3 channels as a new mechanism for the development of Parkinson disease which may be the beginning of a new targeted therapy. The findings were published in Nature Communications.1

Parkinson disease is the second most common neurodegenerative disease in which a specific population of dopamine-producing nerve cells in the mid-brain die off selectively. The resulting lack of dopamine then leads to symptoms such as resting tremors, muscle stiffness, and problems executing voluntary movement. It affects more than 6 million people worldwide and is strongly age-dependent.2

It has previously been found that at the cellular level, disturbances in the calcium-dependent signaling pathways are integral to the development of Parkinson disease. Calcium plays a key role in many cellular signaling pathways, and its concentration is therefore regulated very precisely in the cell.2

Deregulation of the calcium balance causes disturbances of the intracellular signaling cascades, which can lead to cell death. Researchers have now shown that excessive calcium influx through specific ion channels, Cav2.3 channels of the so-called R-type, can contribute significantly to the development of Parkinson disease.1

Researchers were able to prevent the death of dopamine-producing nerve cells by genetically switching off the activity of the Cav2.3 channels. The ion channel Cav2.3 has so far not been associated with Parkinson disease. Further research on dopamine-producing neurons, which have developed from human so-called induced pluripotent stem cells, shows that signaling cascades similar to those that cause Parkinson sensitivity in the animal models are also active in human neurons.1

It had been previously hypothesized that another calcium channel, Cav1.3, plays a central role in the development of Parkinson disease. However, a recently completed clinical trial in which Cav1.3 channels were blocked did not show protection against Parkinson disease.2

This new study provides evidence as to why this clinical trial failed to show protective effects and suggests that selective Cav2.3 inhibitors should be tested as a drug to treat Parkinson disease, the authors concluded.2

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Channel in Nerve Cell May be Key in Unlocking Parkinson Disease Therapy - Pharmacy Times