公开(公告)号：US20200206318A1

公开(公告)日：2020-07-02

申请号：US16802118

申请日：2020-02-26

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Miranda Chapman ,  Bruce Ewenstein ,  Bettina Ploder

IPC: A61K38/37 ,  A61K38/36 ,  A61P7/04

Abstract: The present invention relates to a method for treating gastrointestinal bleeding in a subject with severe von Willebrand Disease comprising administering to the subject at least one dose of recombinant von Willebrand Factor (rVWF) ranging from about 40 IU/kg to about 100 IU/kg, wherein the first dose further comprises recombinant Factor VIII (rFVIII).

公开(公告)号：US20200155413A1

公开(公告)日：2020-05-21

申请号：US16746583

申请日：2020-01-17

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Mark David Schweiss ,  Daniel Edward Roush ,  Stefan Holzner ,  Seth Dale Jones ,  Tejas Dhyani ,  Michelle Shah ,  Madeleine Clare Gibson ,  Jessica Chung ,  Anthony Martin Looper

IPC: A61J1/20 ,  A61M5/14

Abstract: A device is provided for pooling a fluid from a container unit having at least one container, and includes an inlet port having at least one inlet channel configured for receiving the fluid or ambient air, and an outlet port having at least one outlet channel configured for delivering the fluid to an attachment. Both inlet and outlet ports are disposed on the device. A cavity is provided for accommodating insertion of the container unit for pooling the fluid from the at least one container. At least one spike is disposed in the cavity and configured for puncturing a stopper of the at least one container when the container unit transitions from an upper position to a lower position.

公开(公告)号：US20200018752A1

公开(公告)日：2020-01-16

申请号：US16490702

申请日：2018-03-02

Applicant: BAXALTA INCORPORATED ,  BAXALTA GMBH

Inventor： Marian BENDIK ,  Roman NECINA ,  Ernst BOEHM ,  Michael GRANINGER ,  Christian FIEDLER

IPC: G01N33/569 ,  G01N1/42 ,  G01N1/36 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

Abstract: Provided herein are methods of measuring the qualitative and/or quantity attributes of gene therapy vector preparations. In certain embodiments, the gene therapy vector preparations are AAV preparations (e.g., AAV8). In certain embodiments the methods comprise determining the potency or dose of the AAV preparation using ELISA or ELISA in combination with CryoTEM.

公开(公告)号：US20190336588A1

公开(公告)日：2019-11-07

申请号：US16322837

申请日：2017-08-04

Applicant: BAXALTA INCORPORATED ,  BAXALTA GMBH

Inventor： Bruce EWENSTEIN ,  Brahm GOLDSTEIN ,  Bernhard MAJER ,  Paolo ROSSATO ,  Friedrich SCHEIFLINGER ,  Marietta TURECEK

IPC: A61K38/48 ,  A61P11/00 ,  A61P7/02

Abstract: The disclosure provides compositions and methods for treating, ameliorating, and/or preventing a vaso-occlusive crisis (VOC) in a subject suffering from sickle cell disease (SCD). The disclosure also provides compositions and methods for treating, ameliorating, and/or preventing lung injury in a subject suffering from or at risk of suffering from acute lung injury (ALI) and/or acute respiratory distress syndrome (ARDS). The disclosure provides A Disintegrin And Metalloproteinase with Thrombospondin type 1 motif, member-13 (ADAMTS13) or a composition comprising ADAMTS13 for treating, ameliorating, and/or preventing the VOC, or for treating, ameliorating, and/or preventing lung injury in a subject suffering from or at risk of suffering from ALI and/or ARDS.

公开(公告)号：US10414793B2

公开(公告)日：2019-09-17

申请号：US15281616

申请日：2016-09-30

Applicant: BAXALTA INCORPORATED ,  BAXALTA GMBH

Inventor： Stefan Haider ,  Andreas Ivens ,  Hanspeter Rottensteiner ,  Juergen Siekmann ,  Peter Turecek ,  Oliver Zoechling

IPC: C07K14/62 ,  C07K1/107 ,  A61K47/60 ,  A61K47/61 ,  C07K1/20 ,  C07K1/34 ,  C07K14/755

Abstract: The invention relates to materials and methods of conjugating a water soluble polymer to an oxidized carbohydrate moiety of a therapeutic protein comprising contacting the oxidized carbohydrate moiety with an activated water soluble polymer under conditions that allow conjugation. More specifically, the present invention relates to the aforementioned materials and methods wherein the water soluble polymer contains an active aminooxy group and wherein an oxime or hydrazone linkage is formed between the oxidized carbohydrate moiety and the active aminooxy group on the water soluble polymer, and wherein the conjugation is carried out in the presence of a nucleophilic catalyst.

公开(公告)号：US20190271706A1

公开(公告)日：2019-09-05

申请号：US16298520

申请日：2019-03-11

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Mantas Malisauskas ,  Christian Lubich ,  Thomas Prenninger ,  Birgit Reipert

IPC: G01N33/68 ,  G01N33/487 ,  G01N15/14 ,  G01N15/10 ,  C12Q1/56 ,  G01N33/86

Abstract: The present specification discloses methods of characterizing a population of particles using a particle analyzer. Particles may be characterized by size, absolute number, whether the particle is non-proteinaceous or proteinaceous, whether a proteinaceous particle has or lacks a certain physical property, or any combination thereof.

公开(公告)号：US20190225648A1

公开(公告)日：2019-07-25

申请号：US16359763

申请日：2019-03-20

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Friedrich Scheiflinger ,  Michael Dockal

IPC: C07K7/06 ,  C07K7/08 ,  C07K1/04 ,  C07K14/755

Abstract: A peptide or peptide derivative comprising: (i) WDLYFEIVW (SEQ ID NO:1); or (ii) a variant amino acid sequence comprising one, two, three or four L-amino acid substitutions in WDLYFEIVW (SEQ ID NO: 1); or (iii) the retro-inverso variant of the peptide or peptide derivative of either one of parts (i) and (ii), wherein said peptide or peptide derivative has procoagulant activity. A peptide or peptide derivative comprising: (i) an amino acid sequence comprising imfwydcye; or (ii) a variant amino acid sequence comprising one, two, three, four, five or six amino acid substitutions in imfwydcye, wherein said peptide or peptide derivative has procoagulant activity.

公开(公告)号：US20190183981A1

公开(公告)日：2019-06-20

申请号：US16285394

申请日：2019-02-26

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Peter Turecek ,  Juergen Siekmann

IPC: A61K38/37 ,  A61K47/60 ,  C07K14/745 ,  A61K47/61

CPC classification number: A61K38/37 ,  A61K38/00 ,  A61K47/60 ,  A61K47/61 ,  C07K14/745

Abstract: The invention is a proteinaceous construct comprising a Factor VIII molecule which is conjugated to a water-soluble polymer via carbohydrate moieties of Factor VIII, and methods of preparing same.

公开(公告)号：US20190142907A1

公开(公告)日：2019-05-16

申请号：US16258488

申请日：2019-01-25

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Kurt Schnecker ,  Eva Haidweger ,  Peter Turecek

IPC: A61K38/36 ,  A61K47/22 ,  A61K38/17 ,  A61K47/26 ,  A61K9/08 ,  A61K47/12 ,  A61K38/16 ,  A61K9/19 ,  A61K47/18

Abstract: The present invention provides long-term stable pharmaceutical formulations of lyophilized recombinant von-Willebrand Factor (rVWF) and methods for making and administering said formulations.

公开(公告)号：US20190091299A1

公开(公告)日：2019-03-28

申请号：US16030659

申请日：2018-07-09

Applicant: Baxalta Incorporated ,  Baxalta GmbH

Inventor： Miranda Chapman ,  Bruce Ewenstein ,  Bettina Ploder

IPC: A61K38/37 ,  A61P7/04 ,  A61K38/36

Abstract: The present invention relates to a method for treating gastrointestinal bleeding in a subject with severe von Willebrand Disease comprising administering to the subject at least one dose of recombinant von Willebrand Factor (rVWF) ranging from about 40 IU/kg to about 100 IU/kg, wherein the first dose further comprises recombinant Factor VIII (rFVIII).