Apellis Pharmaceuticals, Inc.

Potentia Pharmaceuticals, Inc.

Luna Innovations Inc.

Zyus Life Sciences US Ltd.

Pascal Deschatelets

Cedric Francois

Paul Olson

Federico Grossi

Monica Gerber

Patrick Johnson

Carolina Vega

Martin Emerson Rogers

Janice Paige Phillips

Alec Machiels

Gaurav Bazaz

Janice P Stevenson

Zhouning Zhang

In some aspects, the present invention provides cell-reactive compstatin analogs and compositions comprising cell-reactive compstatin analogs. In some aspects, the invention further provides methods of using cell-reactive compstatin analogs, e.g., treat a complement-mediated disorder, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ. In some aspects, the invention provides long-acting compstatin analogs and compositions comprising long-acting compstatin analogs. In some aspects, the invention further provides methods of using long-acting compstatin analogs, e.g., to treat a complement-mediated disorder, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ. In some aspects, the invention provides targeted compstatin analogs and compositions comprising targeted compstatin analogs. In some aspects, the invention further provides methods of using targeted compstatin analogs, e.g., to treat a complement-mediated disorder, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ.

Dosing regimens and related compositions and methods

Dosing regimens

In some aspects, the invention provides methods of treating a subject in need of treatment for a chronic complement-mediated disorder. In some aspects, the invention provides methods of treating a subject in need of treatment for a Th17-associated disorder. In some aspects, the invention provides methods of treating a subject in need of treatment for a chronic respiratory system disorder. In some aspects, the invention provides methods of administering a complement inhibitor to a subject. In some embodiments, a method of treating a subject comprises administering multiple doses of a complement inhibitor to the subject according to a dosing schedule that leverages the prolonged effect of complement inhibition in chronic respiratory disorders. In some embodiments, a subject has chronic obstructive pulmonary disease. In some embodiments, a subject has asthma.

Methods of treating chronic disorders with complement inhibitors

In some aspects, the present invention provides cell-reactive compstatin analogs and compositions comprising cell-reactive compstatin analogs. In some aspects, the invention further provides methods of using cell-reactive compstatin analogs, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ. In some aspects, the invention provides long-acting compstatin analogs and compositions comprising long-acting compstatin analogs. In some aspects, the invention further provides methods of using long-acting compstatin analogs, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ. In some aspects, the invention provides targeted compstatin analogs and compositions comprising targeted compstatin analogs. In some aspects, the invention further provides methods of using targeted compstatin analogs, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ.

Cell-reactive, long-acting, or targeted compstatin analogs and uses thereof

In some aspects, the present invention cell-penetrating compstatin analog and compositions comprising cell-penetrating compstatin analog. In some aspects, the invention further provides methods of using cell-penetrating compstatin analogs treat a complement-mediated disorder, e.g., to inhibit complement-mediated damage to a cell, tissue, or organ, to inhibit production or release of biologically active C3 cleavage products.

Cell-penetrating compstatin analogs and uses thereof

In some aspects, the present invention provides compositions and methods (e.g. methods of treating, preventing, making, etc.) comprising compstatin analogs. In some embodiments, compstatin analogs comprise a linear PEG moiety having a molecular weight of 40 kDa and coupled to each of two compstatin analog moieties; wherein each compstatin analog moiety is a CA28-AEEAc-Lys moiety, the linear PEG moiety is coupled to the Lys side chain of each CA28-AEEAc-Lys moiety via a carbamate and each CA28-AEEAc-Lys moiety is Ac-Ile-Cys*-Val-(1Me)Trp-Gln-Asp-Trp-Gly-Ala-His-Arg-Cys*-Thr-[NH-CH2CH2OCH2CH2OCH2-C(&#x2550;O)]-Lys-NH2 (SEQ ID NO: 51); wherein the Cys* groups are joined by a disulfide bond.

Cell-reactive, long-acting, or targeted compstatin analogs and related compositions and methods

In some aspects, methods of detecting complement activation in vivo, e.g., in an eye, are provided. In some embodiments, methods comprise detecting drusen containing or in close proximity to complement activation. In some embodiments methods comprise detecting one or more drusen having inflamed endothelium underlying or in close proximity thereto. In some embodiments methods comprise detecting eye-derived extracellular microvesicles, e.g., exosomes, in a body fluid. In some embodiments any of the methods further comprises treating a subject at risk of developing AMD, GA, or advanced AMD or at increased likelihood of rapid progression of AMD with a complement inhibitor. In some aspects, agents useful for performing one or more of the methods are described.

Detection of high risk drusen

In some aspects, the present disclosure provides a computer program product for assembling a database comprising electronic data modules, which may be electronic medical records (EMRs). The present disclosure can also be used for purposes such as implementing a system in which physicians and patents can be provided with applications that they can use to confirm that various disease related events have taken place. This can be performed using active diagnosis modules (ADMs) comprising schedules of events. These ADMs and their events can also be used to populate a database which can be used for purposes such as identifying, recruiting and tracking participants in clinical trials.

Schedule-based electronic medical record modules, applications, and uses thereof

In some aspects, the present invention provides compositions and methods of treatment comprising long-acting compstatin analogs. In some aspects, long-acting compstatin analogs comprise a clearance-reducing moiety attached to two compstatin analog moieties, wherein each compstatin analog moiety comprises a cyclic peptide comprising the amino acid sequence as set forth in SEQ ID NO: 28 extended by a lysine residue or a sequence comprising a lysine residue at the N-terminus, C-terminus, or both, wherein the lysine residue is separated from the cyclic portion of the peptide by a spacer comprising 8-amino-3,6-dioxaoctanoic acid (AEEAc) moiety, the clearance reducing moiety comprises a polymer, wherein each end of the polymer is linked to one of the compstatin analog moieties by way of a linker moiety that is or comprises a carbamate, and wherein the polymer is a PEG having an average molecular weight of about 40 kDa.

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Louisville, KY, United States of America

Pascal Deschatelets