Dermatomyositis

Subject Enrollment This study will consent and enroll 20 subjects total. • For Arm 1, 10 subjects with Idiopathic Pulmonary Arterial Hypertension (IPAH) will be consented and enrolled. For Arm 2, 10 subjects with Connective Tissue Disease Associated Pulmonary Arterial Hypertension (PAH-CTD) will be consented and enrolled. Study Design This study will be observational. Subjects in both arms of the trial will undergo a 129Xe MRI/MRS at timepoints of baseline, 3 months, 6 months, and 12 months. In addition to the this, data from standard of care assessments, such as labs, echocardiography, and six-minute walk distance (6MWD), will also collected at these timepoints. Primary Study Endpoints The primary endpoint for this trial will be the change in defect + low percentage of RBC signal on hyperpolarized 129Xe MRI from baseline to 12 months Secondary Study Endpoints There will be several secondary endpoints for this trial: * Change in regional and global RBC Oscillation Amplitudes on hyperpolarized 129Xe MR spectroscopy from baseline to 12 months * Change in 6MWD from baseline to 12 months * Change in NTproBNP from baseline to 12 months * Change in WHO FC from baseline to 12 months Primary Safety Endpoints There will be several primary safety endpoints for this trial: * Frequency of Adverse Events (AE) and/or Serious Adverse Events (SAE) * Withdrawals due to adverse event or death * Incidence of Adverse Events of Significant Interest (AESI): * Electrocardiogram and any findings * Physical examination and vital signs

The most clinically meaningful way to discover new targets of T cells in autoimmune diseases is to study the tissues of patients with active autoimmune disease mediated organ inflammation. These tissues contain both cytotoxic and helper T cells that are driving their disease, and these T cells are being guided by TCRs that recognize tissue-specific targets. By collecting tissue when a patient has active inflammation, it is possible to determine which T cells are activated and undergoing clonal expansion in the patient's diseased organ. TScan has developed a genome-wide, high-throughput technology to determine the natural, physiological target of any TCR (Kula, 2019). The goal of this study is to isolate T cells from inflamed tissues and matched blood samples and/or matched normal tissues (for patients with inflammatory bowel diseases). T cell clones that are expanded in diseased tissues relative to blood or normal tissues will be selected and the targets of their TCRs will be defined using TScan's genome-wide, high-throughput target ID technology. The goal of this study is to discover a collection of peptide targets, along with their associated TCRs to be developed as new tolerogenic therapies for patients with autoimmune diseases.

A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19 CAR NK cells (KN5501) in patients with relapsed/refractory B-cell related autoimmune diseases.15 patients are planned to be enrolled in the dose-escalation trial (6×10\^9 cells, 9×10\^9 cells). The primary objective of the study is to evaluation of the safety and feasibility of KN5501 for the treatment of relapsed/refractory B-cell related autoimmune diseases. The secondary objective is to evaluate the effectiveness of KN5501 for the treatment of relapsed/refractory B-cell related autoimmune diseases. The exploratory objective is to evaluate expansion, persistence and ability to deplete CD19 positive B cells of KN5501 in patients with relapsed/refractory B-cell related autoimmune diseases.

This is an open-label, dose escalation study in patients with relapsed and refractory autoimmune diseases. Study drug, TI-0032-III injection, is composed of lipid nanoparticles (LNPs) targeting T cells that encapsulate circular RNA encoding the CD19 chimeric antigen receptor (CAR), which is a therapeutic biological product. It is clinically intended for the treatment of various relapsed and refractory B cell-related autoimmune diseases, such as systemic lupus erythematosus, sjögren's syndrome, systemic sclerosis, idiopathic inflammatory myositis, and antiphospholipid syndrome.

This study evaluates the safety and efficacy of YTS109 cells in adults with relapsed/refractory autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), including LN and SLE-ITP, Sjogren's Syndrome, etc. Aproximately 18 patients aged 18-65 will receive a single infusion of YTS109 cells. The dose groups are set to commence at 3×10⁶ STAR -T cells/kg, employing a 3+3 escalation principle for dose titration. The primary objective of this study is to evaluate the safety of YTS109 cells therapy in treating recurrent/refractory autoimmune diseases, while the secondary objectives are to assess the efficacy of YTS109 cells as well as their pharmacokinetic and pharmacodynamic characteristics. The primary endpoint is observations of types, severity, and frequency of adverse events (AEs) and efficacy assessment. This single-arm, open-label trial will enroll patients across The First Affiliated Hospital of Anhui Medical University.

Background: Autoimmune diseases (AIDs) are a group of disorders in which the immune system mistakenly targets and attacks the body's own tissues, leading to tissue damage. Based on the sites of involvement, autoimmune diseases can be broadly classified into two categories: organ-specific autoimmune diseases, such as myasthenia gravis affecting the nervous system, type 1 diabetes mellitus involving the destruction of pancreatic islet cells, and autoimmune hepatitis targeting the liver; and systemic autoimmune diseases that affect multiple tissues and organs, reflecting an imbalance in the immune system, such as systemic lupus erythematosus（SLE）, Sjögren's syndrome（SS）, and systemic sclerosis（SSc）. Recently, the therapeutic advancements have been made in the management of AIDs. However, particularly the patients with relapsed/refractory, continue to face significant unmet clinical needs. CAR-T cell therapy has emerged as one of the innovative therapeutic modalities for autoimmune diseases, characterized by its controllable safety profile and durable therapeutic efficacy, warranting further clinical exploration and investigation in the future. YTS109 cell is a universal allogeneic STAR-T cell therapy targeting CD19, designed to efficiently eliminate B cells in patients with AIDs and mitigate autoimmune responses. Design: This is a Single-Center, Single-Arm, prospective exploratory clinical trial designed to evaluate the safety profile, preliminary therapeutic efficacy, and pharmacokinetic/pharmacodynamic (PK/PD) characteristics of YTS109 cell therapy in patients with relapsed/refractory autoimmune diseases. Approximately 6-12 patients aged 18-65 will receive a single infusion of YTS109 cells (1.5×10⁶ cells/kg). The primary endpoint is observations of types, severity, and frequency of adverse events (AEs) and efficacy assessment. This study was approved by the IRB of Chinese People's Liberation Army (PLA) General Hospital. (Approval Number: S2025-233-01), All participants provided written informed consent.

This study evaluates the safety and efficacy of YTS109 cells in adults with relapsed/refractory autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), including LN and SLE-ITP, Sjogren's Syndrome, etc. Aproximately 18 patients aged 18-65 will receive a single infusion of YTS109 cells. The dose groups are set to commence at 3E6 STAR -T cells/kg, employing a 3+3 escalation principle for dose titration. The primary objective of this study is to evaluate the safety of YTS109 cells therapy in treating recurrent/refractory autoimmune diseases, while the secondary objectives are to assess the efficacy of YTS109 cells as well as their pharmacokinetic and pharmacodynamic characteristics. The primary endpoint is observations of types, severity, and frequency of adverse events (AEs) and efficacy assessment. This single-arm, open-label trial will enroll patients across Institute of Hematology \& Blood Diseases Hospital.

The purpose of this long-term follow-up (LTFU) study is to collect delayed adverse events (AEs) and understand the persistence of KYV-101 (autologous CAR T cell product; gene-modified product), in participants who have been administered KYV-101 (gene-modified product; autologous CAR T cell product). This LTFU protocol will be open to any participant who received at least one infusion of KYV-101 in a previous Kyverna sponsored clinical trial or Investigator Initiated Trial (IIT).

Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed. Secondary Objectives: In patients receiving a non-licensed CBU: * Assess incidence of transmission of infection * Assess incidence of serious infusion reaction * Determine 1 year overall survival after cord blood transplantation * Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV * Assess cumulative incidence of chronic GVHD * Determine platelet engraftment of \>20,000 mcL and \>50,000 mcL

ADI-202300103 is a phase 1 multicenter, open label, dose finding and dose expansion, safety/efficacy study in patients with autoimmune disease. The study will consist of different periods including screening, lymphodepletion, treatment, and follow-up