The NYSCF Research Institute

Transcription

1 Regenerative Medicine Research Update Susan L. Solomon The NYSCF Research Institute New York Pharma Forum February 25, 2016

2 Regenerative Medicine Research Regenerative medicine uses laboratory grown human cells or tissue to treat injured or diseased cells This is a paradigm shift in healthcare It involves the convergence of small molecules and biologics, cell therapy, gene therapy, and biomaterials Cell Therapy Small Molecules/Biologics Biomaterials Gene Therapy

3 Current focus Cancer immunotherapy, cardiovascular, genetic disorders, neurodegenerative diseases, ophthalmology, wound healing, and diabetes Cell Therapy Embroynic Stem Cell (ESC) Induced Pluripotent stem Cells (ipsc) Hematopoietic stem cells Adult Progenitor cells Mesenchymal Stem Cells (MSC) Cord Blood, Placental, Amniotic Tissue Engineering (Biologics + Scaffolds) Synthetic or Natural Biomaterials Allografts & Autografts Decellularized Tissue Matrix Cells, Growth Factors Bioreactors Gene/Gene-Modified Therapies /Gene Editing Therapy Gene Replacement Immunotherapies Genome Editing

4 Pluripotent Stem Cells

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6 A golden age for regenerative medicine Clinical trials for macular degeneration

7 Clinical trials Clinical trials for macular degeneration with ESCs Pete Coffey and Lyndon da Cruz

8 Personalized clinical trials First personalized clinical trials began in 2014 for macular degeneration with ipscs RIKEN/Foundation for Biomedical Research and Dr. Masayo Takahashi

9 Clinical trial for Parkinson s disease One of the first clinical trials using pluripotent stem cells for a neurodegenerative disease Developed by Lorenz Studer at MSKCC Found that in a mouse model, dopaminergic neurons engrafted in the brains of mice and produced dopamine

10 Mitochondrial replacement therapy Mutations in mitochondrial DNA can cause severe disorders and even death We can now replace the mitochondria in human eggs The United Kingdom Parliament voted to approve this therapy in the UK In the United States, we are awaiting approval from the FDA to move forward into patients

11 New tools in regenerative medicine - optogenetics Optogenetics allows us to control cells using light Dr. Studer used optogenetics to turn off dopaminergic neurons that were transplanted into the brains of mice Turning off the cells stopped dopamine production and vice versa NYSCF Robertson Investigator Ed

12 New tools in regenerative medicine gene editing Gene editing aims to transfer genetic material into cells to provide them with new functions Stem cells (ESC, ipsc, MSC, adult tissues) are able to be edited by gene therapy/editing for use in regenerative medicine

13 CRISPR gene editing Allows for more precise gene editing New technologies continually being developed Recently announced CRISPR-Cpf1, next-gen gene editing NYSCF Robertson Investigator Feng Zhang

14 Fight Cancer With Immunotherapy Immunotherapies Using the bodies own immune system to fight diseases such as cancer Stimulating an individual s own immune system to attack cancer cells Immunotherapies have been in development for over a century

15 Chimeric antigen receptor (CAR) T cells CAR-T cells: T-cells are engineered to produce chimeric antigen receptors (CARs). CARs allow the T cells to recognize a protein on tumor cells

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17 Clinical trial for ALS First disease model using ipscs was developed by Dr. Kevin Eggan with ALS patients Using ipsc model, discovered an epilepsy drug on the market which was protective of motor neurons Clinical trial is now recruiting patients at MGH

18 NYSCF Programs NYSCF Research Institute NYSCF Innovators: Fellows and Investigator s NYSCF Conferences and Symposia

19 Pipeline of Leading Investigators NYSCF - Druckenmiller Fellows 50 NYSCF - Robertson Investigators 35 NYSCF Research Institute scientists 45 19

20 NYSCF Conference and Symposia NYSCF s Annual Translational Stem Cell Research Conference Symposium for top stem cell researchers, policy makers and industry NYSCF Conferences & Public Outreach Brings together stem cell scientists from around the world October 26-27, 2016

21 NYSCF Research Institute 45 full time NYSCF researchers Invested $150M+ in stem cell research Leader in developing stem cell technologies and disease modeling Proven ability to develop curative technologies

22 NYSCF Research Institute NYSCF GLOBAL STEM CELL ARRAY : Building AUTOMATED infrastructure STEM CELL to industrialize PRODUCTION, stem DIFFERENTIATION, cell research MANIPULA Objectiv es Objectives: Scale-up stem cell research Establish stem cell-based dr ug discovery platforms Reproducible stem cell production Functionalize human genetics to accelerate the Parallel derivation & culture at scale development of stem cell-der ived inter ventions Quantitative quality control assays Reproducible panels of differentiated cells Diverse and disease populations Connect Genotype to phenotype: in vitro GWAS Array Features Clinical trials in a dish Reproducible stem cell production by automation Fully quality controlled Genetically diverse patient populations Industrial production scale Representative Stem Cell Lines Reprogramming Technology Integration-free reprogramming

23 Automating stem cell derivation and differentiation Biopsy Fibroblast Bank Freeze Data driven batching Batch Thaw ipsc reprogramming Freeze ipsc isolation Data driven thaw Expansion & Arraying of ipscs Stage 1 Stage 2 Stage 3 QC for pluripotency & differentiation IPS Bank Freeze Traceable and standardized environment for production of ipscs and differentiated cells Minimize manual manipulations & maximize parallel processing Control for variation between runs with reference ips and HESC lines Standardized QC assays as a screening tool for consistent quality

24 Existing challenges with stem cells Not standardized Not diverse Not scalable

25 Automating stem cell production The NYSCF Global Stem Cell Array

26 NYSCF Disease Research Areas Bone regeneration Cancer Diabetes / auto-immune diseases Heart disease Macular degeneration/retinal disease Neural disorders ALS Alzheimer s disease Parkinson s disease Multiple sclerosis Neuropsychiatric

27 Automated differentiation Forebrain Neurons Dopaminergic Neurons Beta Cells Cardiac Cells 27

28 The NYSCF Global Stem Cell Array TM Automates stem cell production to provide the consistency needed for meaningful drug testing and cell therapies Represents the global diversity of the world s population Replicate diseases in a dish, using the human cells that are affected by those diseases (not mouse cells) Anticipate how different people will respond to drugs before clinical trials

29 Select NYSCF Research Highlights 2011: First personalized embryonic stem cells 2012: Preventative cure for rare diseases affecting children 2013: First-ever personalized bone grafts 2014: Modeling Alzheimer s disease in the dish 2014: Personalized stem cells from diabetic patients 2014: Accelerated method for making cells affected in multiple sclerosis 2014: New possible treatment for Parkinson s disease 2015: Made stem cells from small amounts of blood

30 Extensive Institutional Collaborations & Key Relationships (50+) International Collaborations Australia China Israel Japan Korea Sweden Switzerland United Kingdom

31 Partnership and collaboration opportunities 1. ipsc generation 2. Disease modeling 3. Differentiated cells for therapy and drug screening 4. Precision medicine Drug screening Predictive diagnostics 5. Data mining 6. Licensing partnership

32 Regenerative Medicine And Healthcare New era in medicine Moving from treatments to cures Reducing health care costs Now a medical and commercial reality A transformation leading to revolutionary products aimed at improving the quality of life Better Health Living Longer Healing Faster

33 Precision medicine the future Precision Medicine: Gene Sequencing Is Not Enough A Path for Precision Medicine Genetic Test Impacts Chemo Choices In Surprising Ways In the future, treatments tailored to patients Precision medicine: The future of medicine? Precision Medicine: Working Toward Custom- Fitted Cures Stem cells are key to precision medicine