HemaCare Corporation Company Overview. Comprehensive Product and Service Solutions for BioResearch and Cellular Therapy

Transcription

1 HemaCare Corporation Company Overview Comprehensive Product and Service Solutions for BioResearch and Cellular Therapy 2015

2 HemaCare Corporation Leading provider of apheresis products, human blood cells, and apheresis collection services. Market leader 250,000 apheresis procedures 30 years Provides apheresis collections and human-derived blood components for preclinical and clinical applications in biologics immunotherapy cell therapy assay development, and medical devices Support preclinical research, clinical studies from Phase I to Phase III, and commercialization Recognized three years in a row by Dendreon as a top apheresis collection performer for PROVENGE, the only FDA-approved cellular immunotherapy product

3 Our History 1970 s 1980 s 1990 s 2000 s 2010 s Founded as a blood bank Began therapeutic apheresis operations Participated in leukapheresis clinical trial collections and stem cell collections programs Partnered with Dendreon to provide apheresis materials for test runs and began cellular therapy clinical trial collections Began Provenge patient collections Expanded the BioResearch Products and Services Division 3

4 Accreditations The HemaCare Advantage FDA registered with cgmp and cgtp requirements State of California Biologics Production and Clinical Lab AABB and CLIA accredited Joint Commission, FACT, DHS, and OSHA compliant World Class Scientific Advisory Board Scott Burger, M.D. Chairman; Advanced Cell & Gene Therapy, LLC Mahendra Rao, M.D., Ph.D. The New York Stem Cell Foundation Aby Mathew, Ph.D. CTO; BioLife Solutions, Inc. Stephen Minger, Ph.D. Global Head of Research; GE Healthcare A comprehensive research and cellular therapy offering 4

5 The HemaCare Advantage Our Donors Make The Difference Donors are the critical source for any research project. Recruited and developed our own healthy donor pool since 1978 Pedigreed Donor Database We know our donors Recall donors / repeat collections They are qualified and tested each donation Qualification, serological and infectious testing performed We can recruit donors for specific investigator needs Age, gender, height, weight, ethnicity Medical history vaccinations, diet, family history Lifestyle characteristics HLA type, other specific laboratory test results Other specific parameters All donors are qualified per FDA regulations and IRB-approved informed consent. 5

6 How HemaCare Participates in the Research and Cellular Therapy Value Chain Continuum Research & Process Development Clinical Trials Commercialization/Cell Therapy Pre-trial studies Research and development using cells Cell manufacturing process development Cell processing Cell isolation/purification Testing (development & qualifying analytical devices & equipment) Apheresis collection CRO/consulting Cell manufacturing process development Testing (development & qualifying analytical devices & equipment) Apheresis collection Cell manufacturing process development Testing (development & qualifying analytical devices & equipment) Apheresis products (normal & mobilized) PBMCs (fresh & frozen) Purified cells (fresh & frozen) Bone marrow products Cord blood products Disease state cells & plasma Characterized PBMCs Customized cell processing projects Contract laboratory services Cryopreservation/biobanking Global sourcing and distribution Disease state tissues Contract cell manufacturing services Apheresis collection National Network Apheresis consulting Consulting services Logistics Distribution Cold chain management Contract laboratory services Contract cell manufacturing services CRO partnerships & alliances Apheresis collection National Network Apheresis consulting Consulting services Logistics Distribution Cold chain management Contract laboratory services Contract cell manufacturing services Value Chain Activities Current HemaCare Product and Services Future Vision for HemaCare Product and Services

7 HemaCare BioResearch Customers - Confidential 7

8 Products and Services Human-derived Primary Cells (healthy and disease state) Peripheral Blood Mobilized Peripheral Blood Bone Marrow Cord Blood Tissue Specimens Biopreservation Media Cellular Therapy Solutions Leukapheresis collections for: Process development Immuno-cellular therapy clinical trials Commercial immuno-cellular therapy (i.e. PROVENGE ) 8

9 Human-derived Primary Cells

10 Human-derived Primary Cells Peripheral Blood Mobilized Peripheral Blood Bone Marrow Cord Blood Leukopaks Platelets Whole Blood Plasma Serum Mononuclear Cells CD3 Pan T Cells CD4 Helper T Cells CD8 Cytotoxic T Cells CD14 Monocytes CD19 B Cells CD4/CD25 Regulatory T Cells CD56 NK Cells Dendritic Cells Macrophages Neutrophils Mononuclear Cells CD14 Monocytes CD34 Stem Cells CD34 Depleted Mononuclear Cells CD133 Stem Cells Unprocessed Bone Marrow Mononuclear Cells Mesenchymal Stem Cells CD34 Stem Cells CD34 Depleted Mononuclear Cells CD133 Stem Cells Unprocessed Cord Blood Mononuclear Cells CD3 Pan T Cells CD4 Helper T Cells CD8 Cytotoxic T Cells CD14 Monocytes CD19 B Cells CD34 Stem Cells CD133 Stem Cells CD56 NK Cells Neutrophils Mesenchymal Stem Cells Plasma Dendritic Cells (CD34 derived) 10

11 Disease-state Primary Cells HemaCare provides disease-state biological products from the following indications: (more indications are continuously added) Autoimmune Disease Diabetes Mellitus Type 2 Rheumatoid Arthritis Systemic Lupus Erythematosus (SLE) Cancer Acute Myeloid Leukemia (AML) Breast Cancer Chronic Lymphocytic Leukemia (CLL) Colorectal Cancer Gastric Cancer Lung Cancer Multiple Myeloma Non-Hodgkin s Lymphoma Prostate Cancer Inflammatory Bowel Disease Crohn's Disease Infectious Disease Hepatitis B Virus (HBV) Hepatitis C Virus (HCV) Herpes Simplex Virus 1 Herpes Simplex Virus 2 Human Immunodeficiency Virus (HIV) Syphilis 11

12 Targeted Donor Recruitment Customizable Our diverse donor pool enables us to provide the match customers need to successfully complete their study: Healthy donors Disease-state donors and patients HLA specific (low and high resolution) CMV negative/positive Blood type specific Diverse demographics Gender and/or age specific Specific lifestyle characteristics Ability to provide both repeat and unique donors All products are collected from donors and patients who are qualified for participation in accordance with Institutional Review Board (IRB)-approved informed consents. 12

13 Customizable Customized Collections And Cell/Tissue Processing Following cgmp and cgtp collection guidelines is key to successful research. We provide: Protocol-specific collections - Your Way Fulfillment of specific cell requests Multiple cell types from same donor Density separation of white blood cells Positive or negative immunomagnetic cell selection methods Custom primary cell cultures Fresh or frozen cell formats Custom cell number/volume per unit Packaging and shipping to customer specifications (i.e. shipping media, buffers, containers) Cold chain services for shipments anywhere globally 13

14 Human Cells: Standardizing Living Biological Raw Material Through Quality Processes The quality and consistency of Human blood-derived cells are critical to enable research for cell therapy, tissue-engineered products, and ex vivo gene therapy products. Controlling cell collections Training Experienced staff Quality Systems 14

15 Best In Class Partners = High Quality Products HemaCare aligns itself with partners with high quality standards BioLife Solutions: HypoThermosol and CryoStor. Miltenyi Biotec: Prodigy, CliniMACS, and AutoMACS BioCision: CoolCell

16 High-quality Leukapheresis Raw Material Collected at HemaCare Count (x10 6 ) ± SD Total Nucleated Cells 14, Mononuclear Cells 12, n= 1,202 leukapheresis products 16

17 Longevity Study - LeukoPak Actual Viable Cell Count in Autologous Plasma 17

18 Peripheral Blood Mononuclear Cells (PBMCs) Sources for Mononuclear cells Peripheral blood Mobilized peripheral blood Bone Marrow Cord blood n = 20 18

19 Cryopreserved PBMC Functional Assay: CMV Culture INF-γ and TNF-α intracellular staining on day 6 PBMCs stimulated with CMV peptide 19

20 Cellular Therapy Solutions

21 Cellular Therapy Solutions A highly flexible, modularized portfolio of Cellular Therapy Solutions Process Development Clinical Trials Commercialization Customized Apheresis for Process Development Apheresis Collection Network Cold Chain and Logistics Cryopreservation and Biopreservation Services Patient Collections within a Standardized Apheresis Network

22 Apheresis Expertise in a Quality Systems Framework Optimize cell collection by controlling and qualifying the apheresis processes based on cgmp and cgtp requirements Qualification of donors Qualification of vendors, equipment, supplies and sites Monitoring equipment, supplies and environment Development and maintenance of procedures Comprehensive staff training and qualification Documentation and records management Management of exceptions and deviations Monitoring quality indicators for process improvement Internal and external inspections 22

23 Apheresis Collection Network The HemaCare Advantage Established network of collection centers Rapidly add new centers as needed Standardized protocols and procedures across all sites 24/7/365 support from clinical, logistics, regulatory, and training experts Single point of contact 23

24 Logistics Management The HemaCare Advantage Complete HemaCare oversight of all controlled room temperature to cold chain logistics Oversight of samples (apheresis materials) from collection to final destination HemaCare owned, maintained, and validated shipping containers (shippers) Best in class biopreservation media Cryostor CS10 Hypothermosol FRS Transport of global samples to US 24

25 Controlled Room Temperature to Cold Chain Logistics HemaCare owned, maintained, and validated room temperature, cold, and cryopreservation containers for all sample types 1. Cryopreserved materials LN2 transport dewars 2. Frozen materials (-80 0 Celsius) Proprietary containers 3. Fresh materials (2-8 0 or Celsius) Proprietary containers 25

26 Cryopreservation Support Cellular Therapy Solutions Short Term Support cryopreservation consistency during process development Medium Term Support validation of cryopreservation process for clinical trials Long Term (Commercialization) Provide a centralized solution for ALL cryopreservation of patient apheresis collection material 26

27 In Conclusion Collecting blood-based cellular products in a manner that minimizes variability brings a higher degree of reproducibility to the research project or manufacturing effort Quality-based controls such as standardized SOPs, staff training and competency assessments, equipment management, and monitoring of quality indicators reduce this variability Relationships with other best in class organizations allows for consistent product development and collaborations focused in improving the cell manufacturing process. Use of cgmp, serum-free, protein-free biopreservation media such as HypoThermosol and CryoStor shows great promise to enable worldwide shipment of fresh or frozen cellular products, extending shelf-life of cell therapy products. HemaCare has the knowledge, experience, and capability to provide total apheresis, room temperature to cold chain, and cryopreservation and storage solution Complete in-house control of the entire process from collection to final destination Just-in-time delivery of viable, high quality apheresis materials to any location globally The only supplier with true, validated, global reach 27

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