Abstract Number: 2458 • ACR Convergence 2025
Obecabtagene autoleucel (obe-cel), a CD19-targeting Autologous Chimeric Antigen Receptor T-cell Therapy (CAR T) with a fast off-rate binding domain, in Patients (pts) with Severe, Refractory Systemic Lupus Erythematosus (srSLE): Preliminary Results from the Phase I CARLYSLE Study
Background/Purpose: Pts with srSLE have limited remaining treatment options and represent a population with an unmet need. CD19 CAR Ts have potential for deep depletion…Abstract Number: 2103 • ACR Convergence 2025
PCRX-201 High-Capacity Adenovirus Serotype 5 Gene Therapy Demonstrates Sustained Clinical Efficacy and Safety in Patients With Knee Osteoarthritis
Background/Purpose: Knee OA is a common disease affecting ~15 million people in the United States. There is an urgent unmet need for additional OA treatments…Abstract Number: 1701 • ACR Convergence 2025
Gene Editing of HLA-Class II DRB1*04:01 at Position 82 Abrogates Binding of Citrullinated Arthritogenic Peptides and Collagen
Background/Purpose: Rheumatoid arthritis (RA) is a chronic autoimmune disease with a strong genetic association to Class II HLA-DRB1*04:01. Presentation of arthritogenic peptides bound to DRB1*04:01…Abstract Number: 0310 • ACR Convergence 2025
Evaluate Safety, Tolerability, and Pharmacodynamics of a Single Intra-Articular Injection of GNSC-001 Gene Therapy in Subjects with Knee Osteoarthritis: 12-month Results from DONATELLO
Background/Purpose: IL-1 contributes significantly to osteoarthritis (OA) pathology, driving inflammation and cartilage degradation. Prior therapies targeting IL-1 have shown limited success due to inadequate drug…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…
ACR Meeting Abstracts