A5038567234- Complement system in diseases
- Blood groups and transfusion
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Hemoglobinopathies and Related Disorders
- Adenosine and Purinergic Signaling
- Renal Diseases and Glomerulopathies
- Platelet Disorders and Treatments
- Retinal Diseases and Treatments
- Neuroendocrine Tumor Research Advances
- Pharmacological Effects and Toxicity Studies
- Retinal Imaging and Analysis
- Blood Coagulation and Thrombosis Mechanisms
- Erythrocyte Function and Pathophysiology
- Transgenic Plants and Applications
- Virus-based gene therapy research
- Blood disorders and treatments
- Ophthalmology and Visual Impairment Studies
- Inflammation biomarkers and pathways
- Oral and gingival health research
- Retinal Development and Disorders
- Phagocytosis and Immune Regulation
- CRISPR and Genetic Engineering
- Bacillus and Francisella bacterial research
- vaccines and immunoinformatics approaches
- Cancer Mechanisms and Therapy
Apellis Pharmaceuticals (United States)
2016-2025
Harvard University
2001
Geographic atrophy (GA), a late stage of age-related macular degeneration (AMD), is major cause blindness. Even while central visual acuity remains relatively well preserved, GA often causes considerable compromise function and quality life. No treatment currently exists. We evaluated the safety efficacy pegcetacoplan, complement C3 inhibitor, for GA.Prospective, multicenter, randomized, sham-controlled phase 2 study.Two hundred forty-six patients with GA.Patients were assigned randomly in...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic complement-mediated hemolysis. C5 inhibition controls intravascular hemolysis in untreated PNH but cannot address extravascular Pegcetacoplan, pegylated peptide targeting proximal complement protein C3, potentially inhibits both and
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, life‐threatening hematologic disease characterized by chronic complement‐mediated hemolysis and thrombosis. Despite treatment with eculizumab, a C5 inhibitor, 72% of individuals remain anemic. Pegcetacoplan (APL‐2), PEGylated C3 has the potential to provide more complete control in patients PNH. This open‐label, phase Ib study was designed assess safety, tolerability, pharmacokinetics pegcetacoplan subjects PNH who remained...
AAV gene transfer is a promising treatment for many patients with life-threatening genetic diseases. However, host immune response to the vector poses significant challenge durability and safety of AAV-mediated therapy. Here, we characterize innate in human whole blood. We identified neutrophils, monocyte-related dendritic cells, monocytes as most prevalent cell subsets able internalize particles, while conventional cells were activated terms CD86 co-stimulatory molecule upregulation....
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis. Pegcetacoplan the first C3-targeted therapy approved for adults with PNH (United States), inadequate response or intolerance to C5 inhibitor (Australia), and anemia despite C5-targeted ≥3 months (European Union). PRINCE was phase 3, randomized, multicenter, open-label, controlled study evaluate efficacy safety of pegcetacoplan vs control (supportive care only; eg, blood...
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematologic disorder characterized by complement-mediated hemolysis. C5 inhibitors (eculizumab/ravulizumab) control intravascular hemolysis but do not prevent residual extravascular The newly approved complement inhibitor, pegcetacoplan, inhibits C3, upstream of C5, and has the potential to improve PADDOCK PALOMINO clinical trials assessed safety efficacy pegcetacoplan in inhibitor-naïve adults (≥ 18 years) diagnosed with...
Plain Language SummaryWhat is this summary about?This a of the results 2 clinical studies , called OAKS and DERBY, that tested medicine pegcetacoplan. Pegcetacoplan first United States Food Drug Administration (US FDA) approved treatment for geographic atrophy, commonly referred to as GA, common eye disease in older people. Geographic atrophy form late age-related macular degeneration, or AMD, usually starts after age 50. The US FDA pegcetacoplan based on from these studies. covered by...
Complement is both evolutionary and scientifically old. It predates the adaptive immunity by some 600 million years was first described in 1905 Jules Bordet Paul Ehrlich. For most of its, existence complement system has been ignored scientists clinicians due to perception it being complicated its relevance for pathogenesis human disease unclear. With recent US Food Drug Administration (FDA) approvals pegcetacoplan paroxysmal nocturnal haemoglobinuria (PNH) geographic atrophy (GA),...
Background: Autoimmune haemolytic anaemia (AIHA) is characterized by haemolysis mediated autoantibodies directed against red blood cells (RBCs). Warm antibody (wAIHA; 60%>70%) and cold agglutinin disease (CAD; 20%>25%) subclasses are defined the immunoglobulin (Ig) isotype involved its thermal optimum for binding RBCs. wAIHA IgG while 90% of CAD IgM isotypes. outcomes can be life‐threatening, but no therapies approved. Complement C3b deposition on RBCs plays a central role in AIHA,...