# US 2019/0167726 A1 · Dosing Regimens for the Mobilization of Hematopoietic Stem Cells

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- Number: US 2019/0167726 A1
- Document ID: US20190167726A1
- Status: Published application
- Kind: A1
- Application number: 16/101,676
- Filed: 2018-08-13
- Published: 2019-06-06
- Earliest claimed: 2017-12-06
- Assignees: Magenta Therapeutics, Inc.
- Applicants: Magenta Therapeutics, Inc.
- Inventors: Dwight Morrow; Patrick C. Falahee; Anthony Boitano; Michael P. Cooke; Kevin A. Goncalves
- Web page: https://patentgrep.com/patent/US20190167726A1

## CPC

- A61K 35/28: Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
- A61K 31/4427: containing further heterocyclic ring systems
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61P 37/00: Drugs for immunological or allergic disorders
- C12N 5/0647: {Haematopoietic stem cells; Uncommitted or multipotent progenitors}
- A61K 2035/124: {the cells being hematopoietic, bone marrow derived or blood cells}
- Y02A 50/30: Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- Y02A: TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE

## Abstract

Described herein are compositions and methods useful for mobilizing populations of hematopoietic stem and progenitor cells within a subject, as well as for determining whether samples of mobilized cells are suitable for release for ex vivo expansion and/or therapeutic use. In accordance with the composition and methods described herein, mobilized hematopoietic stem and progenitor cells can be withdrawn from a donor and administered to a patient for the treatment of various disorders, including hematopoietic diseases, metabolic disorders, cancers, and autoimmune diseases, among others.

## Claims

### Claim at position 1

-115. (canceled)

### Claim 116

A method of treating a stem cell disorder in a human patient, the method comprising infusing into the patient a therapeutically effective amount of hematopoietic stem cells or progeny thereof mobilized by a method comprising:
administering to a human donor (i) Gro-β T at a dose of about 150 μg/kg, wherein the Gro-β T is administered intravenously to the donor and (ii) about 240 μg/kg plerixafor or a pharmaceutically acceptable salt thereof, wherein the plerixafor or pharmaceutically acceptable salt thereof is administered subcutaneously to the donor.

### Claim at position 3

-118. (canceled)

### Claim 119 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a hemoglobinopathy disorder selected from the group consisting of sickle cell anemia, thalassemia, Fanconi anemia, aplastic anemia, and Wiskott-Aldrich syndrome.

### Claim 120 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a myelodysplastic disorder.

### Claim 121 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a congenital or acquired immunodeficiency disorder.

### Claim at position 7

-123. (canceled)

### Claim 124 (depends on 121)

The method of claim 121, wherein the acquired immunodeficiency is human immunodeficiency virus or acquired immune deficiency syndrome.

### Claim 125

(canceled)

### Claim 126 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a metabolic disorder selected from the group consisting of glycogen storage diseases, mucopolysaccharidoses, Gaucher's Disease, Hurlers Disease, sphingolipidoses, and metachromatic leukodystrophy.

### Claim 127

(canceled)

### Claim 128 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a cancer selected from the group consisting of leukemia, lymphoma, multiple myeloma, and neuroblastoma.

### Claim 129

(canceled)

### Claim 130 (depends on 128)

The method of claim 128, wherein the cancer is acute myeloid leukemia, acute lymphoid leukemia, chronic myeloid leukemia, chronic lymphoid leukemia, multiple myeloma, diffuse large B-cell lymphoma, or non-Hodgkin's lymphoma.

### Claim 131 (depends on 116)

The method of claim 116, wherein the stem cell disorder is a disorder selected from the group consisting of adenosine deaminase deficiency and severe combined immunodeficiency, hyper immunoglobulin M syndrome, Chediak-Higashi disease, hereditary lymphohistiocytosis, osteopetrosis, osteogenesis imperfecta, storage diseases, thalassemia major, systemic sclerosis, and systemic lupus erythematosus.

### Claim 132

(canceled)

### Claim 133 (depends on 116)

The method of claim 116, wherein the stem cell disorder is an autoimmune disorder selected from the group consisting of multiple sclerosis, human systemic lupus, rheumatoid arthritis, inflammatory bowel disease, treating psoriasis, Type 1 diabetes mellitus, acute disseminated encephalomyelitis, Addison's disease, alopecia universalis, ankylosing spondylitisis, antiphospholipid antibody syndrome, aplastic anemia, autoimmune hemolytic anemia, autoimmune hepatitis, autoimmune inner ear disease, autoimmune lymphoproliferative syndrome, autoimmune oophoritis, Balo disease, Behcet's disease, bullous pemphigoid, cardiomyopathy, Chagas' disease, chronic fatigue immune dysfunction syndrome, chronic inflammatory demyelinating polyneuropathy, Crohn's disease, cicatrical pemphigoid, coeliac sprue-dermatitis herpetiformis, cold agglutinin disease, CREST syndrome, Degos disease, discoid lupus, dysautonomia, endometriosis, essential mixed cryoglobulinemia, fibromyalgia-fibromyositis, Goodpasture's syndrome, Grave's disease, Guillain-Barre syndrome, Hashimoto's thyroiditis, Hidradenitis suppurativa, idiopathic and/or acute thrombocytopenic purpura, idiopathic pulmonary fibrosis, IgA neuropathy, interstitial cystitis, juvenile arthritis, Kawasaki's disease, lichen planus, Lyme disease, Meniere disease, mixed connective tissue disease, myasthenia gravis, neuromyotonia, opsoclonus myoclonus syndrome, optic neuritis, Ord's thyroiditis, pemphigus vulgaris, pernicious anemia, polychondritis, polymyositis and dermatomyositis, primary biliary cirrhosis, polyarteritis nodosa, polyglandular syndromes, polymyalgia rheumatica, primary agammaglobulinemia, Raynaud phenomenon, Reiter's syndrome, rheumatic fever, sarcoidosis, scleroderma, Sjögren's syndrome, stiff person syndrome, Takayasu's arteritis, temporal arteritis, ulcerative colitis, uveitis, vasculitis, vitiligo, vulvodynia, and Wegener's granulomatosis.

### Claim 134 (depends on 116)

The method of claim 116, wherein the hematopoietic stem cells are autologous with respect to the patient.

### Claim 135 (depends on 116)

The method of claim 116, wherein the hematopoietic stem cells are allogeneic with respect to the patient.

### Claim 136 (depends on 116)

The method of claim 116, wherein the hematopoietic stem cells are HLA-matched with respect to the patient.

### Claim 137 (depends on 116)

The method of claim 116, wherein the hematopoietic stem cells have been genetically modified.

### Claim 138 (depends on 116)

The method of claim 116, wherein the endogenous gene encodes a major histocompatibility complex protein.

### Claim at position 23

-144. (canceled)

### Claim 145 (depends on 116)

The method of claim 116, further comprising isolating the hematopoietic stem cells or progeny thereof by drawing peripheral blood from the donor.

### Claim 146 (depends on 116)

The method of claim 116, further comprising using apheresis to collect the hematopoietic stem cells or progeny thereof from the donor.

### Claim 147 (depends on 116)

The method of claim 116, wherein the Gro-β T has a purity of at least about 95% relative to the deamidated versions of these peptides.

### Claim at position 27

-150. (canceled)

### Claim 151 (depends on 137)

The method of claim 137, wherein the hematopoietic stem cells have been genetically modified to disrupt an endogenous gene.