Abstracts tagged "gene therapy"

Abstract Number: 0002 • ACR Convergence 2024
Preclinical Evaluation of BCMA And/or CD19 Nanobody-based Single or Compound CAR-T Cells Targeting B and Plasma Cells Associated with Autoimmune Disorders
Byeong Hyeok Choi¹, Vincent M DeStefano², Masayuki Wada², Kevin G Pinz¹, Greg Deener², Nabil Hagag¹, Ahmed Abdel-Razek¹, Yu Ma³, Jing Luo³ and Yupo Ma², ¹iCell Gene Therapeutics Inc., Stony Brook, NY, ²iCell Gene Therapeutics, Inc., Stony Brook, NY, ³iCar Bio Therapeutics Ltd, Zhongshan City, Guangdong, China (People's Republic)
Background/Purpose: CD19 single and BCMA-CD19 compound chimeric antigen receptor (CAR) T cell therapy has demonstrated remarkable activity against autoimmune disorders, although relapses with CD19 CAR…
Abstract Number: 0019 • ACR Convergence 2024
A Novel Product Candidate (CPTX2309) for In Vivo mRNA Engineering of Anti-CD19 CAR T Cells Utilizing Novel CD8-Targeted Lipid Nanoparticles
Theresa Hunter¹, Ferran Soldevila¹, Yan Zhang¹, Brittany Ross¹, Daiki Matsuda¹, Yanjie Bao¹, John Li¹, Michelle Nguyen¹, Matthew Butcher¹, MIchael Pica¹, Claudia Fernandez¹, James Vestal¹, Goutam Mondal¹, Yi Kuo¹, Jeffrey Chen¹, Josephine Nguyen², Young Yoon Choi², Diana Galvan¹, Duy Nguyen¹, Donald Jhung², Stuart Sievers¹, Steven Tanis¹, Cory Bentley², Michael Rosenzweig¹, Priya Karmali¹, Adrian Bot¹, Haig Aghajanian¹ and Gregor Adams¹, ¹Capstan Therapeutics, San Diego, CA, ²Capstan Therapeutics, San Diego
Background/Purpose: Ex vivo chimeric antigen receptor (CAR) T cell therapies have revolutionized cancer treatment and are demonstrating clinical efficacy in various autoimmune disease indications. Despite…
Abstract Number: 0876 • ACR Convergence 2024
C-CAR168 as a Novel Anti-CD20/BCMA Bispecific Autologous CAR-T Therapy for the Treatment of Autoimmune Diseases
jiaqi Huang¹, Xin Yao¹, Xiaobing Luo¹, Xiaoteng Lv², Yutian Wei², Michael Patrick¹, Fei Wang², Yi Hong² and Yihong Yao¹, ¹AbelZeta Inc., Rockville, MD, ²AbelZeta Inc., Shanghai, China (People's Republic)
Background/Purpose: B cells are pivotal in autoimmune disease pathogenesis as they produce autoantibodies and undergo aberrant maturation processes, and B-cell dysregulation and the production of…
Abstract Number: 1184 • ACR Convergence 2024
Sustained Clinical Effects After a Single Intra-articular Injection of PCRX-201 for Moderate-to-Severe Osteoarthritis of the Knee
Stanley Cohen¹, Philip G Conaghan², Marc Hochberg³, Alan Kivitz⁴, Nino Joy⁵, Derek Jackson⁵, Masato Nakazawa⁵, Mary DiGiorgi⁵ and Jonathan Slonin⁵, ¹Metroplex Clinical Research Center, dallas, TX, ²Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, and NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom, ³University of Maryland School of Medicine, Baltimore, MD, ⁴Altoona Center for Clinical Research, Duncansville, PA, ⁵Pacira BioSciences, Inc., Tampa, FL
Background/Purpose: Osteoarthritis of the knee (OAK) is a common and severe disease. Current treatments provide temporary pain relief, demonstrating unmet need. PCRX-201 is a high…
Abstract Number: 1733 • ACR Convergence 2024
Safety and Efficacy of CABA-201, a Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Patients with Immune-Mediated Necrotizing Myopathy and Systemic Lupus Erythematosus from the RESET-MyositisTM and RESET-SLETM Clinical Trials
Saira Sheikh¹, Tahseen Mozaffar², Vimal Derebail¹, Natalie Grover¹, Jonathan Hogan³, Courtney Little³, Yvonne White³, Claire Miller³, Rebecca Estremera³, Jenell Volkov³, Daniel Nunez³, Jason Stadanlick³, Mallorie Werner³, Zachary Vorndran³, Alexandra Ellis³, Jazmean Williams³, Justin Cicarelli³, Quynh Lam³, thomas Furmanak³, Chris Schmitt³, Fatemeh Nezhad³, Dan Thompson³, Samik Basu³ and David Chang³, ¹University of North Carolina at Chapel Hill, Chapel Hill, NC, ²University of California, Irvine, Irvine, CA, ³Cabaletta Bio, Philadelphia, PA
Background/Purpose: CD19 targeting chimeric antigen receptor (CAR) T cells have demonstrated durable drug-free responses and remission in patients with idiopathic inflammatory myopathies (IIM) and systemic…
Abstract Number: 0314 • ACR Convergence 2023
Lack of Systemic Effects from Intra-articular XT-150 for the Treatment of Moderate to Severe Pain Due to OA of the Knee: Safety Results from a Phase 2 Trial
Leonard Kapural¹, Stephen D. Collins², Eric Grigsby³, Mark McBride⁴, Jayson Rieger⁵, Morgan Stokes⁶, Howard Rutman⁶ and Flavia Cicuttini⁷, ¹Center for Clinical Research, Winston-Salem, NC, ²Xalud Therapeutics, Inc., Chicago, IL, ³Neurovations Clinical Research, Napa, CA, ⁴Instat Clinical Research, Chatham, NJ, ⁵PBM Capital, Charlottesville, VA, ⁶Xalud Therapeutics, Inc., Berkeley, CA, ⁷Monash University, Melbourne, Australia
Background/Purpose: This study (NCT04124042) was designed to assess the safety, tolerability, and efficacy of XT-150, a non-viral gene therapy, in adult participants with moderate to…
Abstract Number: 0818 • ACR Convergence 2023
Efficacy of XT-150, a Novel Non-Viral Gene Therapy Delivering IL-10v, on Moderate to Severe Pain Due to Osteoarthritis of the Knee: Results of a Phase 2 Trial
Eric Grigsby¹, Stephen D. Collins², Leonard Kapural³, Mark McBride⁴, Jayson Rieger⁵, Morgan Stokes⁶, Howard Rutman⁶ and Flavia Cicuttini⁷, ¹Neurovations Clinical Research, Napa, CA, ²Xalud Therapeutics, Inc., Chicago, IL, ³Center for Clinical Research, Winston-Salem, NC, ⁴Instat Clinical Research, Chatham, NJ, ⁵PBM Capital, Charlottesville, VA, ⁶Xalud Therapeutics, Inc., Berkeley, CA, ⁷Monash University, Melbourne, Australia
Background/Purpose: Interleukin (IL)-10 is a regulator of cytokine activity and has the potential to restore homeostasis in osteoarthritis (OA) of the knee. Unfortunately, relatively short…
Abstract Number: 0859 • ACR Convergence 2023
Single Injection AAV2-hFGF18 Demonstrates Superior Safety and Efficacy over Repeat rhFGF18 Protein Injections in a Model of Induced Osteoarthritis
Alex Goraltchouk¹, Judith Hollander², Francesco Luppino³, Li Zeng² and Alexey Seregin³, ¹Remedium Bio, Inc, Needham, MA, ²Tufts University, Boston, MA, ³Remedium Bio, Inc., Needham, MA
Background/Purpose: Osteoarthritis (OA) is the largest unmet need in rheumatology, affecting 1 in 7 adults, with no approved disease modifying treatments. A recent placebo-controlled clinical…
Abstract Number: 0031 • ACR Convergence 2022
Discovery of GSTT1 Gene That Promotes Osteogenic Differentiation of Adipose Stem Cells
Eugene Lee, GUNIL IM and ji-Yun Koh, Dongguk University, Goyang, Republic of Korea
Background/Purpose: In this study, we describe the discovery of a new GSTT1 gene that promotes the osteogenic differentiation of adipose stem cells (ASCs). While adipose-derived…
Abstract Number: 1506 • ACR Convergence 2020
Correction of Sjögren’s Syndrome Fluid Secretion Deficits in Salivary Gland Acinar Cells by Aquaporin-1 Gene Transfer
Paola Perez¹, Blake Warner², Sandra Wainer¹, Youngmi Ji¹, Thomas Pranzatelli¹ and Jay Chiorini¹, ¹Nidcr, National Institute of Health, Bethesda, MD, ²National Institute of Dental and Craniofacial Research, Bethesda
Background/Purpose: The hallmark clinical complaints in Sjögren’s syndrome (SS) are dry mouth and dry eyes related to salivary and lacrimal glands dysfunction. Reduced salivation reflects…
Abstract Number: 1974 • 2019 ACR/ARP Annual Meeting
The Rat Homolog to FX201, a Gene Therapy in Development for the Treatment of Osteoarthritis, Demonstrates Dose-Dependent Decreases in the Severity of Cartilage and Bone Lesions Following Anterior Cruciate Ligament Transection
Rebecca Senter ¹, Rogely Boyce², Monika Chabicovksy ³, Emily Walsh Martin ¹, Geneviève Langevin-Carpentier ⁴ and Neil Bodick ¹, ¹Flexion Therapeutics, Burlington, ²Beechy Ridge ToxPath LLC, Clay, ³MC Toxicology Consulting, Vienna, Austria, ⁴Charles River Laboratories, Senneville, Canada
Background/Purpose: Acute injury to the anterior cruciate ligament (ACL) is a common cause of posttraumatic OA in humans, and ACL transection (ACLT) in rats is…
Abstract Number: 1677 • 2015 ACR/ARHP Annual Meeting
Disease-Regulated Expression of Anti-Inflammatory Interleukin-10 for the Treatment of Rheumatoid Arthritis
Mathijs G.A. Broeren¹, Miranda B. Bennink¹, Onno J. Arntz², Wim van den Berg² and Fons A.J. van de Loo², ¹Experimental Rheumatology, Radboud university medical center, Nijmegen, The Netherlands, Nijmegen, Netherlands, ²Experimental Rheumatology, Radboud university medical center, Nijmegen, Netherlands
Background/Purpose: The current treatment for patients with rheumatoid arthritis (RA) consists of biological drugs, often in combination with methotrexate. However, some patients fail to respond…
Abstract Number: 1006 • 2014 ACR/ARHP Annual Meeting
Interleukin-4 As Promising, Anti-Inflammatory Transgene for Gene Therapeutic Application in Joint Diseases
Annemarie Lang¹, Johannes Neuhaus², Moritz Pfeiffenberger³, Erik Schroeder⁴, Frank Buttgereit⁵, Michael F. G. Schmidt³ and Timo Gaber⁶, ¹Berlin-Brandenburg School of Regenerative Therapies (BSRT), Berlin, Germany, ²Department of Veterinary Medicine, Research Center of Medical Technology and Biotechnology, Bad Langensalza, Germany, ³Department of Veterinary Medicine, Institute of Immunology, Freie Universität Berlin, Berlin, Germany, ⁴Department of Rheumatology and Clinical Immunology, Charité University Medicine, Berlin, Germany, ⁵Charité University Hospital, Berlin, Germany, ⁶Berlin-Brandenburg Center of Regenerative Therapies (BCRT), Berlin, Germany
Background/Purpose Interleukin- 4 (IL-4), a Th2-cell released immunomodulatory factor, has potent chondroprotective and anti-inflammatory properties able to inhibit the synthesis of IL-1β and TNFα, and…
Abstract Number: 2820 • 2013 ACR/ARHP Annual Meeting
Disease-Regulated Local Interleukin-10 Gene Therapy Diminishes Synovitis and Articular Cartilage Damage In Experimental Arthritis
Eline A. Vermeij¹, Mathijs G.A. Broeren¹, Miranda B. Bennink¹, Onno J. Arntz², Inger Gjertsson³, Wim B. van den Berg¹ and Fons A.J. van de Loo¹, ¹Rheumatology Research and Advanced Therapeutics, Department of Rheumatology, Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands, ²Rheumatology, Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands, ³Department of Rheumatology and Inflammation Research, Institute of Medicine, University of Gothenburg, Gothenburg, Sweden, Gothenburg, Sweden
Background/Purpose: Rheumatoid arthritis (RA) is a chronic destructive autoimmune disease and in most patients the disease follows an intermittent course with periods of exacerbation and…
Abstract Number: 1726 • 2013 ACR/ARHP Annual Meeting
Survivin Inhibition Disturbs Bcl-6 and Blimp-1 Control Of Lymphocyte Maturation and Alleviates Antigen-Induced Arthritis
Mattias Svensson¹, Karin Andersson¹, Malin Erlandsson¹, Ing-Marie Jonsson² and Maria Bokarewa¹, ¹Rheumatology and Inflammation Research, University of Gothenburg, Gothenburg, Sweden, ²Department of Rheumatology and Inflammation Research, Göteborgs University, Göteborg, Sweden
Background/Purpose: Survivin is a proto-oncogene known to regulate cell division and apoptosis. In patients with rheumatoid arthritis, survivin has emerged as an independent predictor of…

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