CD82

ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland. The workshop will continue to drive the momentum for the sense of urgency needed by the patient community to accelerate LGMD drug development. Continue Reading LGMD Scientific Drug Development Workshop The Speak Foundation logo Academic medical experts, senior leaders from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), highly experienced drug developers, and other specialists will come together to discuss LGMD pathophysiology, clinical outcomes and patient-focused drug development, surrogate endpoints and the accelerated approval pathway. The meeting will focus on six LGMD subtypes: LGMD2A/R1, LGMD2B/R2, LGMD2C/R3, LGMD2D/R4, LGMD2E/R5, and LGMD2I/R9. "In September 2022, at the LGMD community's first Externally-Led Patient Focused Drug Development Meeting (EL-PFDD), the FDA heard perspectives of patients and caregivers on the health effects, daily impacts, treatment goals, and decision factors that would be considered when seeking out or selecting a treatment," explains Kathryn Bryant, founder of the Speak Foundation. "This workshop is a continuing step to work with the FDA and LGMD scientific leaders on pushing critical drug development forward. The time is now." LGMD Scientific Workshop session presenters will include CBER Director Dr. Peter Marks, and CDER's Office of New Drugs (OND) Director, Dr. Peter Stein. Other confirmed speakers include Dr. Jerry R. Mendell, retired attending neurologist of Nationwide Children's Hospital, Dr. Nicholas Johnson of Virginia Commonwealth University, Dr. Louise Rodino-Klapac of Sarepta Therapeutics, Dr. Douglas Sproule of ML Bio Solutions, a BridgeBio affiliate, Dr. Katherine Mathews of University of Iowa Health Care, Dr. Matthew Wicklund of University of Texas San Antonio, Dr. Peter Kang of University of Minnesota Medical School and Dr. Lindsay Alfano of Nationwide Children's Hospital. "As a translational clinician scientist, devoting a lifetime of work toward improving the lives of patients with muscular dystrophy, workshops like the LGMD Scientific meeting are important venues for discussing the current scientific understanding of LGMD and the implications for drug development." said Dr. Jerry R. Mendell. "Bringing key LGMD stakeholders together to share ideas and research findings in a public forum is the most productive way to find solutions and advance therapies that will improve the lives of LGMD patients." The LGMD Scientific Workshop will be a focused discussion with clinicians, scientific researchers, drug developers, regulators, patients and patient advocates on the characteristics of LGMD that drive drug development programs. The meeting will be held virtually and by invitation for in-person attendance. Proceedings will be recorded and shared with the broader patient and scientific communities through The Speak Foundation. More details will be added soon to: The Speak Foundation - Limb Girdle Muscular Dystrophy. About The Speak Foundation: The Speak Foundation is a 501(c)(3) nonprofit charity, which is focused on supporting patients living with all forms of LGMD and on facilitating development of treatments for these diseases. About Limb Girdle Muscular Dystrophy: Limb-Girdle Muscular Dystrophy (LGMD) is a group of muscular dystrophies caused by mutations in one of over 30 genes. The age of onset, symptoms, progression, and inheritance pattern varies among genetic subtypes, but generally result in severe disability and are often life-threatening due to cardiac and respiratory complications. There are estimated to be some 20,000+ people living with a form LGMD in the United States. Media Contact: Rachel DeConti [email protected] 860-209-0492 SOURCE The Speak Foundation

Clinical Presentations to Include Initial Clinical Data of FT576 BCMA-targeted CAR NK Cell Product Candidate for Multiple Myeloma and FT819 CD19-targeted CAR T-cell Product Candidate for B-cell Lymphoma Preclinical Data of FT555 GPRC5D-targeted CAR NK Cell Collaboration Candidate for Multiple Myeloma to be Jointly Presented with Janssen Novel Approaches to Eliminate Patient Conditioning and Promote Functional Persistence of Off-the-shelf, iPSC-derived Cell Therapies to be Highlighted SAN DIEGO, Nov. 03, 2022 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, today announced that fourteen presentations for the Company’s induced pluripotent stem cell (iPSC) product platform were accepted for presentation at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition being held in New Orleans, Louisiana, or virtually, December 10-13, 2022. The presentations include initial Phase 1 clinical data for FT576, the Company’s multiplexed-engineered, iPSC-derived CAR NK cell product candidate targeting BCMA for multiple myeloma, and for FT819, the Company’s iPSC-derived CAR T-cell product candidate targeting CD19 for B-cell lymphoma. In addition, the Company will jointly present with its collaboration partner Janssen preclinical data for FT555, a multiplexed-engineered, iPSC-derived CAR NK cell product candidate targeting GPRC5D, a tumor-associated orphan G-protein-coupled receptor found to be highly expressed on myeloma cells. In May, Janssen exercised its commercial option to FT555, and the companies are conducting IND-enabling activities to support first-in-human clinical investigation of FT555 for the treatment of multiple myeloma as monotherapy and in combination with the CD38-targeted monoclonal antibody daratumumab. The Company will also highlight multiple novel strategies to eliminate the need for patient conditioning for off-the-shelf cell therapy. These strategies include arming iPSC-derived effector cells with an alloimmune defense receptor (ADR), which targets 41BB-expressing alloreactive host immune cells to promote expansion, persistence, and anti-tumor activity; the combined genetic ablation of the adhesion molecules CD54 and CD58, which reduces immune synapse formation resulting in host immune cell evasion; and the genetic ablation of CD38 in combination with CD38-targeted monoclonal antibody therapy, which uniquely targets and depletes CD38-expressing activated host immune cells. Details of the ASH presentations are as follows: Clinical Programs Interim Phase I Clinical Data of FT819-101, a Study of the First-Ever, Off-the-Shelf, iPSC-Derived TCR-Less CD19 CAR T-Cell Therapy for Patients with Relapsed / Refractory B-Cell MalignanciesPublication Number: 2000Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DInterim Phase I Clinical Data of FT576 As Monotherapy and in Combination with Daratumumab in Subjects with Relapsed / Refractory Multiple MyelomaPublication Number: 2004Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DA Phase I Study of FT538, an Off-the-Shelf, Multiplexed-Engineered, iPSC-Derived NK Cell Therapy in Combination with Daratumumab in Relapsed / Refractory Multiple MyelomaPublication Number: 4639Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster IIIPresentation Date / Time: Monday, December 12, 2022, 6:00 PMRoom: Ernest N. Morial Convention Center, Hall D Preclinical Programs FT555: Off-the-Shelf CAR-NK Cell Therapy Co-Targeting GPRC5D and CD38 for the Treatment of Multiple MyelomaPublication Number: 1992Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DA Novel Dual-Antigen Targeting Approach Enables Off-the-Shelf CAR NK Cells to Effectively Recognize and Eliminate the Heterogenous Population Associated with AMLPublication Number: 4623Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IIIPresentation Date / Time: Monday, December 12, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall D iPSC Product Platform Alloimmune Defense Receptor Harnesses Host Immune Cell Activation to Potentiate Functional Persistence and Anti-Tumor Activity of Off-the-Shelf, Cell-Based Cancer TherapyPublication Number: 1986Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DCombined Genetic Ablation of CD54 and CD58 in CAR Engineered Cytotoxic Lymphocytes Effectively Averts Allogeneic Immune Cell RejectionPublication Number: 481Session Name: 703. Cellular Immunotherapies: Basic and Translational IVPresentation Date / Time: Sunday, December 11, 2022; 9:30 AM Room: Ernest N. Morial Convention Center, 388-390iPSC-Derived CD38-Null NK Cells in Combination with CD38-Targeted Antibody: A Dual Therapeutic Strategy to Enable ADCC and Eliminate Host Immune Cells in Multiple MyelomaPublication Number: 3288Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster II Presentation Date / Time: Sunday, December 11, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall DA CD3 Fusion Receptor (CD3-FR) Uniquely Enables Compatibility of Allogeneic CAR-T and -NK Cells with T Cell Engagers to Enhance Antitumor Function and Limit Antigen EscapePublication Number: 3308Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IIPresentation Date / Time: Sunday, December 11, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall DMulti-Antigen Targeting By Novel Combination of CAR-T Cells and hnCD16 Transgene, Yields in Complete Tumor Clearance Via Antibody Dependent Cellular CytotoxicityPublication Number: 4605Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IIIPresentation Date / Time: Monday, December 12, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall DThe Development of Allogeneic Tips-Derived TCR- CAR+ CD8αβ T CellsPublication Number: 4624Session Name: 703. Cellular Immunotherapies: Basic and Translational: Poster IIIPresentation Date / Time: Monday, December 12, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall DCharacterization of Engineered Macrophages and Other Myeloid Cells Differentiated from CD34+ Hematopoietic Progenitor Cells Derived from Pluripotent Stem CellsPublication Number: 1218Session Name: 501. Hematopoietic Stem and Progenitor Cells and Hematopoiesis: Basic and Translational: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DHigh-Density Cryopreservation of Off-the-Shelf CAR-NK Cells Facilitates On-demand Treatment AccessPublication Number: 2045Session Name: 711. Cell Collection and Processing: Poster IPresentation Date / Time: Saturday, December 10, 2022; 5:30 PM Room: Ernest N. Morial Convention Center, Hall DCD82 Is Sufficient to Uniquely Identify Pluripotent Stem Cell-Derived Hemogenic Endothelium with the Hematopoietic Lineage Potency to Give Rise to Bona Fide LymphocytesPublication Number: 2532Session Name: 501. Hematopoietic Stem and Progenitor Cells and Hematopoiesis: Basic and Translational: Poster IIPresentation Date / Time: Sunday, December 11, 2022; 6:00 PM Room: Ernest N. Morial Convention Center, Hall D 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 are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with 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 designed to overcome 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 350 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 patients with cancer. 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 pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit 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 Company's product candidates, clinical studies and preclinical research and development programs, its ongoing and planned clinical studies, the safety and therapeutic potential of the Company’s product candidates, and expectations regarding the Company’s collaborations, including the objectives, plans and goals of its collaboration with Janssen. 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’s product candidates may not demonstrate the requisite safety or efficacy to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that the Company’s 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