# US 2019/0169575 A1 · Sc-beta cells and compositions and methods for generating the same

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- Number: US 2019/0169575 A1
- Document ID: US20190169575A1
- Status: Published application
- Kind: A1
- Application number: 16/273,076
- Filed: 2019-02-11
- Published: 2019-06-06
- Earliest claimed: 2013-06-11
- Assignees: President and Fellows of Harvard College; Janssen Biotech, Inc.
- Applicants: President and Fellows of Harvard College; Janssen Biotech, Inc.
- Inventors: Quinn P. Peterson; Felicia J. Pagliuca; Douglas A. Melton; Jeffrey R. Millman; Michael Saris Segel; Mads Gurtler; Alireza Rezania; Benjamin Fryer
- Web page: https://patentgrep.com/patent/US20190169575A1

## CPC

- C12N 5/0678: {Stem cells; Progenitor cells; Precursor cells}
- A61K 35/39: Pancreas; Islets of Langerhans (Langerhans cells of epidermis A61K35/36)
- A61P 3/10: for hyperglycaemia, e.g. antidiabetics
- A61P 5/48: of the pancreatic hormones
- A61P 5/50: for increasing or potentiating the activity of insulin
- C12N 5/0606: {Pluripotent embryonic cells, e.g. embryonic stem cells [ES] (embryonic germ cells C12N5/0611, induced pluripotent stem cells C12N5/0696)}
- C12N 5/0676: {Pancreatic cells}
- C12N 5/0696: {Artificially induced pluripotent stem cells, e.g. iPS}
- C12N 2501/11: Epidermal growth factor [EGF]
- C12N 2501/117: Keratinocyte growth factors (KGF-1, i.e. FGF-7; KGF-2, i.e. FGF-12)
- C12N 2501/15: Transforming growth factor beta (TGF-β)
- C12N 2501/16: Activin; Inhibin; Mullerian inhibiting substance
- C12N 2501/375: Thyroid stimulating hormone [TSH]
- C12N 2501/385: of the family of the retinoic acid recptor, e.g. RAR, RXR; Peroxisome proliferator-activated receptor [PPAR]
- C12N 2501/395: Thyroid hormones
- C12N 2501/40: Regulators of development
- C12N 2501/41: Hedgehog proteins; Cyclopamine (inhibitor)
- C12N 2501/727: Kinases (EC 2.7.)
- C12N 2501/998: Proteins not provided for elsewhere
- C12N 2501/999: Small molecules not provided for elsewhere
- C12N 2506/02: from embryonic cells
- C12N 2506/03: from non-embryonic pluripotent stem cells
- C12N 2506/22: from pancreatic cells
- C12N 2506/45: from artificially induced pluripotent stem cells
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- 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

## Abstract

Disclosed herein are methods, compositions, kits, and agents useful for inducing β cell maturation, and isolated populations of SC-β cells for use in various applications, such as cell therapy.

## Claims

### Claim 1

A method for differentiating PDX1-positive pancreatic progenitor cells into NKX6.1 positive pancreatic progenitor cells with a culture medium comprising a) KGF and b) SANT1, wherein the culture medium is substantially free of a Protein Kinase C (PKC) Activator.

### Claim 2 (depends on 1)

The method of claim 1, wherein the PKC Activator is Phorbol 12,13-dibutyrate (PdBu) or TPB.

### Claim 3 (depends on 1)

The method of claim 1, wherein the PKC Activator is PdBu.

### Claim 4 (depends on 1)

The method of claim 1, wherein the PCK Activator is TPB.

### Claim 5 (depends on 1)

The method of claim 1, further comprising retinoic acid (RA).

### Claim 6

A method for differentiating PDX1-positive pancreatic progenitor cells into NKX6.1 positive pancreatic progenitor cells with a culture medium comprising a) KGF and b) SANT1, wherein the culture medium is substantially free of a BMP signaling pathway inhibitor.

### Claim 7 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is noggin, chordin, gremlin, and follistatin and LDN 193189

### Claim 8 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is noggin.

### Claim 9 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is chordin.

### Claim 10 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is gremlin.

### Claim 11 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is follistatin.

### Claim 12 (depends on 6)

The method of claim 6, wherein the BMP signaling pathway inhibitor is LDN 193189.