A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...
In cancer research and personalized medicine, mesoporous silica nanoparticles (MSNs) have emerged as a significant breakthrough in both cancer treatment and diagnosis. MSNs offer targeted drug delivery, enhancing therapeutic effectiveness while minimizing adverse effects on healthy cells. Due to their unique characteristics, MSNs provide targeted drug delivery, maximizing therapeutic effectiveness with minimal adverse effects on [...]
INTRODUCTION: Obexelimab is an investigational, bifunctional, non-depleting, humanized monoclonal antibody that binds CD19 and FcgammaRIIb to inhibit B cells, plasmablasts, and CD19-expressing plasma cells. In clinical trials, intravenous (IV) administration of obexelimab has been well-tolerated, and demonstrated clinical activity in patients with rheumatoid arthritis, systemic lupus erythematosus, and immunoglobulin G4-related disease. This study was performed [...]
Effective vaccination is crucial for intervening in the COVID-19 pandemic. However, with the continuous mutation of the SARS-CoV-2, existing vaccines including subunit vaccines cannot effectively prevent virus infections. Hence, there is an urgent need to enhance the immunogenicity of existing vaccines to induce a more potent and durable immune response. We previously found that periodic [...]
BACKGROUND: 9MW1911 is a high-affinity human IgG4 monoclonal antibody targeting ST2, the human IL-33 receptor. It may have anti-inflammatory effects by blocking the IL-33/ST2 pathway. This first-in-human trial (NCT05803902) aimed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity and pharmacodynamics of 9MW1911 in healthy participants. METHODS: This phase I, randomized, double-blind, placebo-controlled study enrolled 48 healthy [...]
The presence of treatment-emergent anti-drug antibodies (ADAs) can pose challenges during biotherapeutic development by compromising drug safety and efficacy. Early assessment of immunogenicity risk is essential to mitigate these risks. Dendritic cells (DCs) are crucial for priming CD4 T cells and necessary for effective antibody production. Therefore, DC internalization has been investigated as a predictive [...]
INTRODUCTION: As a crucial asset for human health and modern medicine, an increasing number of biotherapeutics are entering the clinic. However, due to their complexity, these drugs have a higher potential to be immunogenic, leading to the generation of anti-drug antibodies (ADAs). Clinically significant ADAs have an impact on pharmacokinetics (PK), pharmacodynamics (PD), effectiveness, and/or [...]
The advent of large molecule therapeutics has revolutionized treatment options for previously unmet medical needs. This advent has also led to an increased impact of immunogenicity on drug efficacy and safety. In order to maximize the potential of large molecule therapeutics, immunogenicity-related liabilities must be identified as early in development as possible, using an integrated [...]
Bringing safe and effective drugs to patients is of utmost importance for the pharmaceutical industry, with immunogenicity (IG) being a critical factor that influences both aspects. Biotherapeutics can elicit unwanted immune responses, potentially leading to (severe) safety implications, reduced patient benefits, and may result in termination of development. Therefore, understanding IG risks throughout drug development [...]
Chimeric antigen receptor-engineered T cell therapies (CAR-T) are becoming powerful immunotherapeutic tools for treating malignancies, especially hematological malignancies. Like other biological drugs, CAR-T cell products can trigger unwanted immune responses in patients receiving the treatment. This might lead to treatment failure or life-threatening consequences. This immunogenicity could also affect the CAR-T cells' cellular kinetics and [...]
