Granted Patent
B2
US 12,304,900 · App. 18/581,700 · Granted May 20, 2025
Islet cell manufacturing compositions and methods of use
Inventors:
Austin Thiel (Southborough, MA); Jihad Yasin (Gardner, MA); Evrett Thompson (Wobum, MA); Felicia J. Pagliuca (Boston, MA)
Assignee:
Vertex Pharmaceuticals Incorporated
C07D401/14C07D401/04C07K14/495C07K14/50C12N5/0676C12N2501/117C12N2501/155C12N2501/16C12N2501/385C12N2501/41
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Abstract
Disclosed herein are compositions and methods useful for manufacturing SC-β cell, and isolated populations of SC-β cells for use in various applications, such as cell therapy.
Claims (24)
1. A composition comprising a population of PDX1-negative primitive gut tube cells and a BMP signaling pathway inhibitor, wherein the BMP signaling pathway inhibitor is a compound of Formula A,
2. The composition of claim 1 , wherein the composition further comprises a growth factor from the transformation growth factor β (TGF-β) superfamily.
3. The composition of claim 2 , wherein the growth factor from the transformation growth factor β (TGF-β) superfamily is selected from the group consisting of an Inhibin, an Activin, a Mullerian inhibiting substance (MIS), a bone morphogenic protein (BMP), decapentaplegic (dpp), Vg-1, monoclonal nonspecific suppressor factor (MNSF), growth differentiating factor 8 (GDF8), and growth differentiating factor 11 (GDF11).
4. The composition of claim 2 , wherein the growth factor from the transformation growth factor β (TGF-β) superfamily comprises Activin A.
5. The composition of claim 4 , wherein the composition comprises about 2 ng/ml to about 50 ng/ml of Activin A.
6. The composition of claim 1 , wherein the composition comprises about 0.1 μM to about 0.3 μM of the BMP signaling inhibitor.
7. The composition of claim 1 , wherein the composition further comprises keratinocyte growth factor (KGF).
8. The composition of claim 1 , wherein the composition further comprises SANT1.
9. The composition of claim 1 , wherein the composition further comprises retinoic acid.
10. The composition of claim 1 , wherein the composition further comprises PdbU.
11. The composition of claim 1 , wherein the composition further comprises thiazovivin.
12. The composition of claim 1 , wherein the composition further comprises activin A and keratinocyte growth factor (KGF).
13. The composition of claim 1 , wherein the composition further comprises activin A and SANT1.
14. The composition of claim 1 , wherein the composition further comprises activin A and retinoic acid.
15. The composition of claim 1 , wherein the composition further comprises activin A and PdbU.
16. The composition of claim 1 , wherein the composition further comprises activin A and thiazovivin.
17. The composition of claim 1 , wherein the composition comprises two or more of the following: keratinocyte growth factor (KGF), SANT1, retinoic acid, PdbU, and thiazovivin.
18. The composition of claim 1 , wherein the composition comprises three or more of the following: keratinocyte growth factor (KGF), SANT1, retinoic acid, PdbU, and thiazovivin.
19. The composition of claim 1 , wherein the composition comprises four or more of the following: keratinocyte growth factor (KGF), SANT1, retinoic acid, PdbU, and thiazovivin.
20. The composition of claim 1 , wherein the composition comprises keratinocyte growth factor (KGF), SANT1, retinoic acid, PdbU, and thiazovivin.
21. The composition of claim 17 , wherein the composition comprises activin A.
22. The composition of claim 18 , wherein the composition comprises activin A.
23. The composition of claim 19 , wherein the composition comprises activin A.
24. The composition of claim 20 , wherein the composition comprises activin A.
Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Jul 1, 2024
From: THIEL, AUSTIN; YASIN, JIHAD; THOMPSON, EVRETT; PAGLIUCA, FELICIA J.
To: SEMMA THERAPEUTICS, INC.
Reel/Frame 067889/0564 →
MERGER
Recorded Jul 1, 2024
From: SEMMA THERAPEUTICS, INC.
To: VERTEX PHARMACEUTICALS INCORPORATED
Reel/Frame 067889/0647 →
Continuity (5)
Continuation
PCTUS2018061364
· Nov 15, 2018
Provisional Application
62669170
· May 9, 2018
Provisional Application
62586808
· Nov 15, 2017
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