Lead Application Scientist East Coast USA Agenda What is Cell Therapy Types of Cell Therapy Common Cell Types used Development and Manufacturing of Cell Therapies Why is it important to ChemoMetec ID: 998486
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1. Cell TherapyRodney Jarvis, PhDLead Application ScientistEast Coast, USA
2. AgendaWhat is Cell Therapy?Types of Cell TherapyCommon Cell Types usedDevelopment and Manufacturing of Cell TherapiesWhy is it important to ChemoMetec?
3. AgendaWhat is Cell Therapy?Types of Cell TherapyCommon Cell Types usedDevelopment and Manufacturing of Cell TherapiesWhy is it important to ChemoMetec?
4. Cell TherapyWhat is Cell Therapy?In general, cell therapy is the use of human cells to treat disease.Allogenic- the use of healthy donor cells for treatmentAutologous- the use of the patients own cells for treatmentwww.apceth.com/apcct/uploads/2017/09/apceth-grafik-09-2017.jpg
5. AgendaWhat is Cell Therapy?Types of Cell TherapyCommon Cell Types usedDevelopment and Manufacturing of Cell TherapiesWhy is it important to ChemoMetec?
6. Common Cell Types Used in Cell TherapyImmunoTherapyT-cell Therapy (i.e CAR-T, TCR, and CAR-NK)T-cellsNK-cellsDendritic Cells (Dendritic Vaccines)Regenerative MedicineMesenchymal Stem Cells (MSC’s)Induced Pluripotent Stem Cells (iPSC’s)Human Embryonic Stem Cells (hESC’s)Neural Progenitor Cells (NPC’s)Hematopoietic Stem Cell (HSC’s)
7. Why These Markets are so ImportantInstruments sold in US 2018-2019
8. Types of Cell TherapyImmunoTherapyCAR-T/CAR-NK Cell TherapyTCR Cell TherapyRegenerative Medicine
9. Cell Therapy
10. Cell TherapyImmune System- biological defense system in humans and other vertebrates which fights disease-causing organisms/cells. Classified as Innate and Adaptive ImmunityT-Cell- The primary immune cell that provides adaptive immunityTargets foreign cells as well as Cancerous/Pre-Cancerous CellsCAR-T- Chimeric Antigen Receptor T-cells are genetically engineered to target specific cancer expressing antigensGenetically Engineered- intentional modification of an organism/cell by altering its genetic codeCancer- a disease where an organism's cells acquire mutations that lead to uncontrolled growthLeukemia- A specific type of cancer of progenitor blood cells
11. Cell TherapyCAR-T (Chimeric Antigen Receptor-T cell therapy)- Is a type of ImmunotherapyImmunotherapy- Using immune cells to target and kill cancerThis treatment is highly effective. EX:Novartis’ Kymriah is used to treat relapsed B-cell Acute Lymphoblastic Leukemia (B-ALL) in children and showed an 83% remission rate in these previously untreatable children.
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16. Cell Therapy- The Novartis Approach
17. CAR-T MarketGlobal CAR-T Market to reach 2.99 Billion by 2025NA American Market 1.6 BillionFirst FDA Approved Therapies:17
18. Cell TherapyCAR-T (Chimeric Antigen Receptor-T cell therapy)- Who else is doing it?
19. Cell TherapyHow we fit into the CAR-T Manufacturing Process
20. El SegundoSanta MonicaOceansideGaithersburg, MDFrederick, MD
21. Springhouse, PAR&DMalvern, PAQC & Vector ManufacturingRaritan, NJGMP Manufacturing
22. Cell TherapyCAR-NK (Chimeric Antigen Receptor-Natural Killer Cell therapy)- Natural Killer cells differ from T-cells in that they do not need prior activation through receptors to perform cytotoxic abilitiesAre found to be less likely to cause Cytokine Storm SyndromeCan be harvested from Umbilical Cord Blood and modified for Allogenic TreatmentCustomers doing this include NantKwest and Nkarta
23. Cell TherapyT-Cell Receptor (TCR) Therapy Is another type of Immunotherapy which uses the natural TCR from patientsT-cells that are already found in patient that recognize cancer cellsThis TCR is cloned and reintroduced into the T-cells in greater numbers.We have some customers doing this (i.e. Adaptimmune, PACT pharma, and TCR2)
24. Types of Cell TherapyImmunoTherapyCAR-T/CAR-NK Cell TherapyTCR Cell TherapyRegenerative Medicine
25. Why These Markets are so ImportantInstruments sold in US 2018-2019
26. Cell TherapyStem Cell treatments (Regenerative Medicine)Mesenchymal Stem Cells (MSC)Induced Pluripotent Stem Cells (iPSC)Embryonic Stem Cells (ESC)Neural Progenitor Cells (NPC)Hematopoietic Stem Cells (HSC)
27. Cell Type Differentiation (in a nutshell!)All living organisms start as a single cellThrough Transcriptional Regulation and Cell-Cell signaling cells differentiateEnd with every cell type necessary for the organism to survive
28. Cell Therapy-Stem Cell treatments (Regenerative Medicine)
29. 29Mesenchymal Stem CellsBone MarrowAdipose-Derived
30. Cell Therapy- Bone Marrow Derived MSC’s
31. Customers in Cell Therapy and Their ProcessMayo- (as seen in video) focused on Bone Marrow derived MSCCollect Bone Marrow derived sample from patientRe-inject into affected joint with no major modification to the cells
32. Customers in Cell Therapy and Their ProcessIngeneron works with Adipose derived MSCLipoaspiration of MSC from Patient FatEnzymatic degradation of non MSC materialSeveral wash and filter steps
33. InGeneron“We need to use a cell counting device that is FDA-compliant and capable of counting Adipose-derived Regenerative Cells rapidly and efficiently. After testing several options, we found that the NucleoCounter® NC-200™ device fits our required criteria and consistently delivers rapid, reproducible and reliable results.We did not have to perform any additional validation studies related to cell counting and viability due to the FDA’s familiarity with the instrument. Selecting the NC-200™ as our automatic cell counter saved us time during the FDA application as well as during training of the staff at the clinical sites. The quality of the device and the excellent customer service that ChemoMetec provides has been a key factor in the successful initiation of our clinical program.” Glenn Winnier, PhD, Chief Scientic Officer
34. MSC Manufacturing for Regenerative MedicineMSC are adherent cells (require something to attach to)Can be grown in flasks but this causes growth constraints and inefficient growthBioreactors and microcarriers lead to larger production of MSC
35. Counting MSC from BioreactorsCells can be grown in large quantities for therapeutic useTraditionally, MSC samples must be detached from microcarriers so growth can be monitored by counting
36. How do we fit in?Especially for MSC grown on MicrocarriersWe offer the only solution for fast reliable cell counts with our A100 and B assay
37. Cell TherapyStem Cell treatments (Regenerative Medicine)iPSC’sThese cells are generated by taking differentiated cells and adding transcription factors that force them back into a stem cell stateThese cells allow for circumventing the need for obtaining controversial Embryonic Stem Cells and allow for the potential use of a patients own cells
38. iPSC’shttps://youtu.be/i-QSurQWZo0
39. Cell TherapyOther examples of Regenerative/Stem Cell therapyCardiac- Ischemic heart diseaseNeural –Spinal cord injury, ParkinsonsSkin- Skin graftingBlood – Ex: Sickle cell anemia, Hemophilia
40. Research and DevelopmentBasic Research- Bench Science and literature Discovery- Initial investigation that finds a drug or procedure that has an effect on a disease stateMode of biological action is investigated and documentedEffect of action is documentedResults from this can lead to an Investigational New Drug (IND) application and possibly clinical trials
41. Process DevelopmentOnce a process for cell therapy has been definedThere is continual development to streamline and optimize the processThis is done to increase product output and qualityDuring this phase before clinical trials and some during early trials, much of the production steps are defined. This includes methods used to evaluate the product
42. ManufacturingCell Therapy manufacturing requires expansion of donor/patient cellsCells are enriched Cells are modified, if part of processCells are ExpandedCells are then frozen to preserve them until they are needed (Allogenic) or infused back into patient (Autologous)
43. Quality ControlDuring the Manufacturing process, the cells are routinely checked for QualityThis can be anything from total count and viability, to checking to make sure they express the traits of interestAt the end of the manufacturing process, cells are checked again to make sure they adhere to SQuIPP (Safety, Quality, Identity, Purity, and Potency)
44. AgendaWhat is Cell Therapy?Types of Cell TherapyCommon Cell Types usedDevelopment and Manufacturing of Cell TherapiesWhy is it important to ChemoMetec?
45. Why These Markets are so ImportantInstruments sold in US 2018-2019
46. Why These Markets are so Important1-2 instruments
47. Why These Markets are so Important1-2 instruments3-4 instruments
48. Why These Markets are so Important1-2 instruments4-5 instruments10-100+ instruments
49. How We Address the Pain PointsHighly Accurate and Precise InstrumentsAssays that can address multiple cell types and conditionsT-cellsAggregated cellsMicrocarrier workGMP capabilitiesAs process expands and number of instruments increaseAll instruments count the sameDo not require any yearly calibrationPerform Annual PM’s, Important for GMP compliance!This allows for all counts to be comparable between all different departmentsR&D, PD, Manufacturing, and QC
50. How We Manage These AccountsProvide Excellent Customer ServiceSales being prompt with quote requests and trial requestsBringing FAS into conversation to determine best instrument for the customerFAS performing trials with customer and being available for trouble shootingShowing the value of our instruments over their current methods and competitorsOnce they purchaseFAS regularly email and visit customers Excellent trouble shooting response timesIf problems go unknown to us, customer could transition to a competitorGrow our presence within the company
51. SummaryCell Therapy makes up ~85% of our marketImmunotherapy is ~75%Regenerative Medicine ~10%And GROWING!Getting into early stage companies constitutes substantial opportunity for growthEqually important is maintaining excellent relationships with our existing customers
52. QUESTIONS?Rodney Jarvis, PhDLead Application ScientistChemoMetecrja@chemometec.com