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Compositions, devices and methods for factor VII therapy

A technology of composition and polymer, applied in the field of composition, device and method for factor VII therapy, capable of solving problems such as short half-life

Pending Publication Date: 2022-03-22
СИГИЛОН ТЕРАПЬЮТИКС ИНК
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, rFVIIa has a short half-life, thus requiring multiple doses over a short period of time in hemophiliacs to manage active bleeding episodes

Method used

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  • Compositions, devices and methods for factor VII therapy
  • Compositions, devices and methods for factor VII therapy
  • Compositions, devices and methods for factor VII therapy

Examples

Experimental program
Comparison scheme
Effect test

example 1

[0427] Example 1: Generation and Culture of Exemplary Engineered ARPE-19 Cells

[0428] use Figure 8 The indicated expression vectors generate FVII secreting cells into which a foreign nucleotide sequence encoding a precursor FVII protein has been inserted using standard cloning methods. To achieve this, ARPE-19 cells were co-transfected with PiggyBac containing the transposase plasmid together with the FVII expression vector, and stably transfected cells were cultured in complete growth medium containing puromycin. The FVII expression vector was identical to the vector in Figure 6, except that one or two transcription units were inserted between the 5'ITR and 3'ITR. In addition to the promoter sequence and the inserted FVII coding sequence or FVII albumin fusion sequence, the backbone sequence of each transcription unit is Figure 6A and Figure 6B The corresponding sequences shown in are identical, for example, each transcription unit has the same Kozak sequence and the s...

example 2

[0438] Example 2: FVII secretion from exemplary engineered ARPE-19 cells

[0439] To quantify FVII protein secretion by cells, polyclonal populations of ARPE-19 cells engineered with the different FVII expression constructs described in Example 1 were trypsinized as described above and 500,000 cells were plated in 6-well tissue Place 2 ml of growth medium in a single well of a Petri dish. The engineered cells were allowed to settle and attach to the dish for 3-4 hours before the growth medium was replaced with fresh medium. One day (24 hours) after the medium change, the growth medium containing secreted FVII was collected and placed on ice. Cells were washed with phosphate-buffered saline and trypsinized with 0.05% trypsin EDTA as previously described. Cells were harvested and counted using trypan blue on a Countess II cell analyzer. FVII protein levels of the engineered cells described above were determined using conditioned growth medium and run on a commercially availab...

example 3

[0440] Example 3: Synthesis of Exemplary Compounds of Formula (I)

[0441] General plan

[0442] The following procedures describe methods for preparing exemplary compounds for use in the implantable device preparations described herein. The compounds provided herein can be prepared from readily available starting materials using modifications of the specific synthetic schemes described below that are well known to those skilled in the art. It will be appreciated that where typical or preferred process conditions (ie, reaction temperatures, times, mole ratios of reactants, solvents, pressures, etc.) are given, other process conditions can also be used unless otherwise stated. Optimum reaction conditions may vary with the particular reactants or solvent used, but such conditions can be determined by one skilled in the art by routine optimization procedures.

[0443] Furthermore, as will be apparent to those skilled in the art, conventional protecting groups may be necessary t...

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Abstract

Described herein are RPE cells engineered to secrete FVII proteins, as well as compositions, pharmaceutical formulations, and implantable devices comprising these engineered RPE cells, and methods of making and using them for treating patients suffering from hemophilia or FVII deficiency.

Description

[0001] priority claim [0002] This application claims priority to U.S. Provisional Application No. 62 / 824,963, filed March 27, 2019, and U.S. Provisional Application No. 62 / 907,386, filed September 27, 2019. The disclosure of each of the foregoing applications is incorporated herein by reference in its entirety. [0003] sequence listing [0004] This application contains a Sequence Listing that has been submitted electronically in ASCII format and is hereby incorporated by reference in its entirety. Said ASCII copy, created on March 25, 2020, is named S2225-7029WO_SL.txt and is 122,698 bytes in size. Background technique [0005] Factor VII (FVII) plays a key role in coagulation, a series of reactions in which plasma inactive zymogens are converted to active enzymes and lead to the formation of insoluble fibrin clots. The inactive form of FVII is expressed in the liver and secreted into the blood as a single-chain zymogen. Following injury to blood vessels, tissue factor...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K47/64
CPCA61K47/62A61K47/6925A61K47/61A61K47/6903A61K48/00C12N9/647C07K2319/31C07K2319/50A61K9/5073A61K9/0024A61K9/5036C07K14/745C12N15/85C12N2800/90C12N9/6437C12N5/0621A61K35/30A61K38/36A61P7/04C12Y304/21021C12N2510/00A61K9/4816A61K9/4891C12N15/52
Inventor L.E.巴尼M.博雷加德G.卡莫纳F.C.冈萨雷斯R.海德布雷赫特E.E.约翰斯顿R.J.米勒O.奥康纳M.A.奥伯利D.佩里特J.A.塞韦尔D.M.史密斯O.维塞P.K.沃顿Z.伊恩
Owner СИГИЛОН ТЕРАПЬЮТИКС ИНК
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