Ameri's Project – University of Copenhagen

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Ameri's Project

About Jacqueline Ameri

Assistant professor Jacqueline Ameri started her second post doc in the lab of professor Henrik Semb at DanStem in 2012, where she continued her research on the in-depth characterization of the hESC-derived pancreatic progenitors and how to direct their fate to glucose-responsive insulin-producing beta cells. In 2015, Jacqueline was appointed Assistant Professor and Project Manager for the newly founded cell-therapy program in diabetes in the Section for Strategic Translational Stem Cell Research and Therapy (TransStem) in the Semb laboratory. The short-term aim is to produce GMP graded hPSC-derived beta cells to initiate a phase 1 trial to assess the safety of hPSC-derived beta cells. The long-term aim of this academic program is to develop up-scaled manufacturing protocols of mature beta cells for future clinical needs. In parallel with her academic activities, she has as co-founder and CEO spearheaded a spin-out initiative (PanCryos) supported by Novo Seeds, Copenhagen Spin Outs, and Accelerace.

The Project

The overall goal of the project Jacqueline is currently managing, at TransStem, is to manufacture GMP certified stem cell derived beta cells to initiate a phase 1 trial aiming to assess the safety of hPSC-derived beta cells. More specifically, the work involves GMP adaptation of our current differentiation protocol, screening of GMP cell lines, and the development of new strategies for expansion of pancreatic progenitors and their conversion into glucose-responsive beta cells. To translate both recent and future discoveries in a commercially-viable product for clinical trials in Type 1 Diabetesthere will be a close collaboration between the academic team at TransStem and the impending spin-out company PanCryos.

In vitro differentiated hESC-derived insulin producing cells (in white)
co-expressing the transcriptional regulator ISLET1 (ISL1, in red)

Future Plans

Over a decade of experience has revealed the challenges and complexity but also the great potential and possibilities of this field and Ameri is today equipped to tackle one of healthcare's big challenges through her pioneering work.

"When I started my PhD studies in 2004, there was a lot of hype regarding human embryonic stem cells (hESCs) and how they could be used in treatment of various diseases. Being an enthusiastic postgraduate student and full of expectations, I was determined that I would be able to make a significant contribution to the field.  However, during my lengthy PhD studies I realized how optimistic I had been in my beliefs and expectations and started to appreciate the complexity of the project I had been given. Ten years later I finally succeeded in designing a protocol that allows us to generate hESC-derived beta cells through in vitro differentiation. Accomplishing this has reignited my hope in achieving a curative cell based therapy for type 1 Diabetes."

In the near future, Ameri aims to shift her entire focus on spinning out PanCryos, which is founded on IP generated through her research at DanStem, and continue her efforts in establishing a safe stem cell based therapy for diabetes patients.

PanCRYOS - Developing a safe and scalable cell therapy for Type 1 diabetes

PanCryos is a prospective spin out company arising from DanStem, University of Copenhagen, aiming to develop a next generation stem cell derived allogenic cell therapy (PanINSULA™) for type 1 diabetes, based on novel IP in the differentiation of stem cells into mature beta cells and in the purification of pancreatic progenitor cells. PanCryos has assembled a team with experts in stem cell biology, islet transplantations, business and regulatory guidance and is currently funded by pre-seed funding from Novo Seeds and a KU POC grant. We are seeking both partners and investors that could support our efforts in reaching the goal of a first-in-class phase I/II clinical trial.

PanCryos in the News:

Funding and Awards

Received in 2015, (~2.5M DKK) from Novo Nordisk Foundation for her project entitled “Developing methods for expandable production of human pluripotent stem cell-derived insulin-producing beta cells for cell therapy in diabetes”.

In 2016, she was also awarded a proof of concept grant (0.5M DKK) from Copenhagen University to support the establishment of the spin-out company “PanCryos”, founded on IP generated by her research at DanStem.

Publications and Patents 

Ramond, Cyrille, Nicolas Glaser, Claire Berthault, Jacqueline Ameri, Jeannette Schlichting Kirkegaard, Mattias Hansson, Christian Honoré, Henrik Semb, Raphaël Scharfmann (2017). Reconstructing human pancreatic differentiation by mapping specific populations during development. eLife 2017;6:e27564, doi: 10.7554/eLife.27564.

Ameri J, Borup R, Prawiro C, Ramond C, Schachter KA, Scharfmann R, and Semb H. Efficient generation of glucose-responsive beta cells from isolated GP2+ human pancreatic progenitors.  2017 Cell Reports, http://dx.doi.org/10.1016/j.celrep.2017.03.032

Ameri J, Ståhlberg A, Pedersen J, Johansson JK, Johannesson MM, Artner I, Semb H. FGF2 Specifies hESC-Derived Definitive Endoderm into Foregut/Midgut Cell Lineages in a Concentration-Dependent Manner.  Stem Cells 2010; 28: 45-56.

Fischer Y, Ganic E, Ameri J, Xian X, Johannesson M, Semb H. NANOG Reporter Cell Lines Generated by Gene Targeting in Human Embryonic Stem Cells.  PLoS ONE. 2010 Sep 2; 5(9):pii:e12533.

Johannesson M, Ståhlberg A, Ameri J, Sand FW, Norrman K, Semb H. FGF4 and retinoic acid direct differentiation of hESCs into PDX1-expressing foregut endoderm in a time- and concentration-dependent manner.  PLoS ONE. 2009;4(3):e4794.

Rintisch C, Ameri J, Olofsson P, Luthman H, Holmdahl R. Positional cloning of the Igl genes controlling rheumatoid factor production and allergic bronchitis in rats. Proc Natl Acad Sci U S A. 2008 Sep 16;105(37):14005-10. doi: 10.1073/pnas.0803956105.

Patents:

Inventors: Jacqueline Ameri and Henrik Semb, "INDUCED DERIVATION OF SPECIFIC ENDODERM FROM hPS CELL-DERIVED DEFINITIVE ENDODERM." Assignee: Novo Nordisk A/S and Cellartis AB. WO/2010/136583.

Inventors: Jacqueline Ameri and Henrik Semb, "ISOLATION OF BONA FIDE PANCREATIC PROGENITOR CELLS" Applicant: University of Copenhagen. WO/2016/170069


Funding and support agencies: