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
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
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
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
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
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: 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
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
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: 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
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: 0031 • ACR Convergence 2022
Discovery of GSTT1 Gene That Promotes Osteogenic Differentiation of Adipose Stem Cells
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
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
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
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
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
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
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…
ACR Meeting Abstracts