Alexion Pharmaceuticals, Inc.

Provided herein are compositions and methods for treating a human patient with a complement-associated condition (e.g., PNH or aHUS) by subcutaneously co-administering to the patient a hyaluronidase (e.g., rHuPH20) and an anti-C5 antibody, or antigen binding fragment thereof (e.g., ravulizumab).

Provided are methods for clinical treatment of complement-associated conditions comprising administering to the patient an anti-C5 antibody, or antigen binding fragment thereof, wherein the anti-C5 antibody, or antigen binding fragment thereof, is administered (or is for administration) subcutaneously according to a particular clinical dosage regimen (i.e., at a particular dose amount and according to a specific dosing schedule). In one embodiment, the patient has previously been treated with eculizumab (SOLIRIS®) or ravulizumab (ULTOMIRIS®); particularly intravenously administered SOLIRIS® or ULTOMIRIS®.

Provided are methods for clinical treatment of neuromyelitis optica spectrum disorder (NMOSD) using an anti-C5 antibody, or antigen binding fragment thereof.

Featured are compositions and methods for treating bone mineralization disorders, such as hypophosphatasia (HPP). In some embodiments, the methods described herein are useful for treating adult subjects with HPP and/or treating or preventing bone fractures.

Provided herein are antibodies which bind to ceruloplasmin and are useful for various applications, including detecting ceruloplasmin and immunocapturing ceruloplasmin in biological samples. The antibodies are useful in methods of measuring non-ceruloplasmin-bound copper concentrations and labile-bound copper concentrations in biological samples.

This disclosure provides peptide based compounds, compositions, and methods to treat medical disordeds, such as complement-mediated disorders, including complement C1s-mediated disorders, such as acute antibody-mediated rejection, amyotrophic lateral sclerosis, autoimmune blistering disease, bullous pemphigoid, geographic atrophy, or Guillain-Barre Syndrome, comprising administering to a subject in need thereof a therapeutically effective amount of the compound.

Methods of treating a ciliopathy-associated disease are disclosed, including administering to a subject in need thereof an effective amount of a compound that targets at least one G-protein coupled receptor. Methods for identifying therapeutic agents for treating a disease having a ciliopathy are provided, including providing an animal model system of the ciliopathy for testing a putative therapeutic agent; administering a disruptive agent to the animal, treating the administered animal with the putative therapeutic agent, comparing the measurable phenotype of the treated animal with that of the animal without treatment, and identifying the therapeutic target for treating a ciliopathy, when the measurable phenotype of the treated animal is reduced as compared with that of the animal without treatment.

Provided are methods for clinical treatment of neuromyelitis optica spectrum disorder (NMOSD) using an anti-C5 antibody, or antigen binding fragment thereof.

Featured are polypeptides that include soluble alkaline phosphatases, mutants, fragments, fusion proteins thereof, and methods of use thereof, for treating bone mineralization disorders, such as hypophosphatasia (HPP), and symptoms thereof. The polypeptides include a soluble alkaline phosphatase (sALP) or fragment thereof, which is derived from a naturally occurring alkaline phosphatase (ALP).

Described herein are isolated monovalent antibodies or antibody fragments thereof that bind human properdin. Such antibodies are useful in methods of treatment for diseases mediated by alternative complement pathway dysregulation.