Global Market for Regenerative Medicine (RM), 2019

1.1 Statement of the Report
1.2 Executive Summary

2.1 A Bird’s Eye View of the RM Industry
2.1.1 Number of RM Companies
2.1.2 Major Therapeutic Platforms and Enabling Technologies in RM Industry
2.1.3 RM Product Approvals in 2017/2018
2.1.4 Total RM Clinical Trials
2.1.5 Total RM Clinical Trials by Technology Type
2.1.6 Patient Impact of Three U.S.-Approved RM Products, 2018
2.1.7 Select Anticipated RM Industry Clinical Trial Data Readouts: 2019
2.2 RM Industry-Total Global Financings: 2018
2.2.1 Total Financings to RM Industry by Type and Year, 2016-2018
2.2.2 Corporate Partnerships (Upfront Payments) in RM Industry, 2018
2.2.3 Private Placements and Venture Financings in RM Industry, 2018
2.2.4 Public Offerings (IPOs & Follo-Ons) in RM Industry, 2018
2.2.5 Total Merger & Acquisition within RM Industry by Year, 2016-2018
2.3 Market Access for Regenerative Medicines as of January 2019
2.4 Anticipated RM Product Launches
2.4.1 Predicted RM Treated Patient Numbers
2.5 Top 20 RM Companies by Pipeline Size
2.6 RM Research Institutes
2.6.1 RM Research Institutes in the U.S.
2.6.2 RM Research Institutes in Europe
2.6.3 RM Research Institutions in Australia
2.6.4 University Research to Products in the Market

3.1 Cell Therapy Timeline
3.2 Types of Stem Cells Used in Cell Therapy
3.2.1 Hematopoietic Stem Cells (HSCs)
3.2.2 Induced Pluripotent Stem Cells (iPSCs)
3.2.3 Mesenchymal Stem Cells (MSCs)
3.2.3.1 Number of MSCs in Clinical Trials
3.2.3.2 Indications for which MSCs are tested in Clinical Trials
3.3 Neural Stem Cells (NSCs)
3.3.1 Indications Addressed by NSCs in Clinical Trials
3.4 Embryonic Stem Cells (ESCs)
3.4.1 Clinical Trials using Human Embryonic Stem Cells (hESCs)
3.4.2 Number of Clinical Trials using hESC-Derived Cells in 2018
3.5 NoTable Events in Cell Therapy Space in 2018
3.6 Top 15 Cell Therapy Companies

4.1 Number of Companies Developing Gene-Modified Cell Therapy Products
4.2 CAR-T Cell Therapy
4.2.1 Global Distribution of CAR-T Cell Therapy Companies
4.2.2 Select Events in Cell-Based Immuno-Onclogy Space, 2018
4.3 T-Cell Receptor (TCR) Therapy
4.4 Natural Killer (NK) Cell Therapies
4.5 Tumor Infiltrating Lymphocytes (TILs) Therapy
4.6 Marrow-Infiltrating Lymphocytes (MILs)
4.7 Gamma-Delta T-Cells Therapy
4.7.1 Big Biopharma’s Interest in Gamma-Delta T-Cells Therapy
4.8 Dendritic Cell (DC) Therapy
4.8.1 Cancer Types Targeted by Dendritic Cell Therapy Studies
4.8.2 Ongoing Clinical Trials Testing DC Vaccines

5.1 Gene Augmentation Therapy
5.2 Gene Inhibition Therapy
5.3 Suicide Gene Therapy
5.4 The Process of DNA Transfer in Gene Therapy
5.5 Ex vivo vs. in vivo Gene Therapies
5.5.1 Ex vivo Gene Therapy
5.5.2 In vivo Gene Therapy
5.6 Autologous vs. Allogeneic Gene Therapies in Development
5.7 Number of Gene Therapy Companies in the World
5.8 Gene Therapy Pipeline Volumes, 1995-2018
5.9 Big Pharma and Cell Therapy/Gene Therapy Development
5.10 Number of Gene Therapy Products Predicted to be Approved in Next Three Years
5.11 Gene Therapy Product Candidates Granted RMAT Designation
5.12 Gene Therapy Companies Active in Neurodegenerative Disorders (ND)
5.12.1 Gene Therapy Developers for Neurodegenerative Diseases
5.12.2 Neurodegenerative Disorders Addressed by Gene Therapy Studies
5.12.3 Anticipated Events in Neurodegenerative Disease Space of Gene Therapy
5.12.4 Important Events in Gene Therapy Space in 2018
5.13 Leading Gene Therapy StartUps

6.1 Smart Biomaterials in TE
6.1.1 Biosynthetic Materials
6.1.2 3D PrinTable Inks
6.2 Synthetic and Naturally-Derived Scaffolds
6.3 Tissue Substitutes
6.3.1 Cell-Based Tissues
6.3.2 Collagen
6.4 Two Key Developments in Tissue Engineering Space in 2018

7.1 Viral Vectors
7.1.1 Physical Properties of Commonly used Viral Vectors
7.1.2 Retrovirus Vectors
7.1.3 Adenovirus Vectors
7.1.4 Herpes Simplex Viral Vectors
7.1.5 Lentivirus Vectors (LV)
7.1.5.1 Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
7.1.5.2 Companies Developing Lentivirus Vectors
7.1.6 Adeno-Associated Virus Vectors
7.1.6.1 Types of Diseases in Clinical Trials using AAV
7.1.6.2 Genes Transduced by AAV for Specific Diseases
7.1.6.3 Companies Developing AAV Vector
7.1.6.4 The Predominantly-Used Viral Vectors
7.2 Non-Viral Vectors
7.2.1 Microinjection
7.2.2 Particle Bombardment
7.2.3 Laser Iradiation
7.2.4 Electroporation
7.2.5 Magnetofection
7.2.6 Sonoporation

8.1 Number of People Participating in RM Clinical Trials
8.2 Clinical Trials by Therapeutic Category in the RM Sector, 2018
8.3 Cell Therapy Clinical Trials
8.3.1 Cell Therapy Clinical Trials by Indication
8.3.2 Mesenchymal Stem Cells in Clinical Trials
8.3.3 Anticipated Clinical Events in Cell Therapy Space
8.3.3.1 Remestemcel-L
8.3.3.2 Tab-Cel
8.3.3.3 MultiStem
8.4 Clinical Trials in Gene-Modified Cell Therapies Space
8.4.1 Most Popular Target Antigens for CAR-T Cell Therapies
8.4.2 Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
8.4.3 Antigens Targeted by CAR-T in Solid Cancers in Clinical Trials
8.4.4 Anticipated Developments in CAR-T Space
8.5 Number of Gene Therapy Clinical Trials
8.5.1 Therapy Clinical Trials by Geography
8.5.2 Diseases Targeted by Gene Therapy
8.5.3 Gene Types Transduced in Clinical Trials
8.5.4 Vectors used in Gene Therapy Clinical Trials
8.5.5 Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
8.5.6 Domination of Oncology in Gene Therapy Drug Development
8.5.7 Dominance of Viral Vectors in Gene Therapy
8.5.8 Gene Therapy Alliances Volume and Value, 2012-2018
8.5.9 Anticipated Clinical Events in Gene Therapy Space, 2019/2020
8.6 Number of Tissue Engineering Clinical Trials
8.6.1 Anticipated Clinical Events in Tissue Engineering Space

9.1 Number of RM Products Approved by Geography
9.1 Approval of RM Products by Year
9.2 RM Products with MA Worldwide by Therapy Type
9.2.1 Indications Addressed by the Approved RM Products
9.3 Approved RM Products in Australia
9.3.1 Ortho-ACI
9.4 RM Products Approved in Canada
9.5 Gene Therapy Product Approved in China
9.6 Approved RM Products in Europe
9.6.1 Yescarta (Axicabtagene ciloleucel)
9.6.2 Kymriah (Tisagenlecleucel)
9.6.3 Alofisel
9.6.4 Spherox
9.6.5 Zalmoxix
9.6.6 Strimvelis
9.6.7 Imlygic (Talimogene laherparepvec)
9.6.8 Holoclar
9.6.9 RM Products with Withdrawn Approvals in Europe
9.7 Approved RM Products in India
9.7.1 Cartigrow
9.7.2 Ossgrow
9.7.3 Apceden
9.7.4 Stempeucel
9.8 RM Products Approved in Japan
9.8.1 TEMCELL HS Inj.
9.8.2 HeartSheet
9.8.3 JACC
9.9 RM Products Approved in New Zealand
9.9.1 Prochymal
9.10 RM Products Approved in South Korea
9.10.1 KeraHeal-Allo
9.10.2 Neuronata-R
9.10.3 Cupistem
9.10.4 Cartistem
9.10.5 Cellgram-AMI
9.10.6 CureSkin Inj.
9.10.7 Queencell
9.10.8 Kaloderm
9.10.9 RMS Ossron
9.10.10 Immuncell LC
9.10.11 CreaVax-RCC
9.10.12 Kera Heal
9.10.13 Chondron
9.11 Approved RM Products in the U.S.
9.11.1 HPC, Cord Blood
9.11.2 Luxturna (voretigene neparvovec-rzyl)
9.11.2.1 Mechanism of Action
9.11.3 Yescarta (axicabtagene ciloleucel)
9.11.3.1 Mechanism of Action
9.11.4 Kymriah (tisagenlecleucel)
9.11.4.1 Mechanism of Action
9.11.5 MACI
9.11.6 Clevecord
9.11.7 HPC, Cord Blood (Bloodworks)
9.11.8 Imlygic (talimogenelaherparepvec)
9.11.9 HPC, Cord Blood (LifeSouth Community Blood Centers)
9.11.10 Allocord
9.11.11 Ducord
9.11.12 HPC, Cord Blood (Clinimmune Labs)
9.11.13 Gintuit
9.11.14 Hemacord
9.11.15 Laviv (Azficel-T)
9.11.16 Provenge (Sipuleucel-T)
9.12 U.S. RM Products with RMAT Designation
9.13 Pricing of RM Products
9.14 RM Products Expected to be Approved in Late 2019 or 2020

10.1 Cost of Manufacture
10.1.1 Cost of Manufacture in a Partially-Automated Facility
10.1.2 Cost of Manufacture in a Fully-Automated Facility
10.1.3 Net Present Cost (NPC) of the Three Methods
10.2 In-House Gene Therapy Manufacturing Capabilities of Select Big and Mid Pharma
10.3 External Gene Therapy Manufacturing by Big and Mid Pharma
10.4 Cell and Gene Therapy Manufacturing Capacities in North America
10.5 Cell and Gene Therapy Manufacturing Capacities in Europe

11.1 Appropriate Phase for Outsourcing
11.2 Important Points Condirered while Fixing a CMO/CDMO
11.3 In-House vs. Outsourcing
11.4 Number of Cell Gene Therapy CMOs/CDMOs in Europe
11.5 Projected Growth of CMOs and CDMOs
11.6 Projected Growth of Cell Manufacturing Capacity

12.1 Global Market for RM Products
12.1.1 Global RM Products Market by Geography
12.2 Global Market for Cell Therapy Products
12.3 Global Market for Gene Therapy Products
12.3.1 Competitive Landscape in Gene-Modified Cell Therapy Products
12.3.2 Global Market for Viral Vector and Plasmid DNA Manufacturing
12.4 Global Market for Tissue-Engineered Products

13.1 4D Molecular Therapeutics, LLC
13.1.1 Key Capabilities
13.2 Abeona Therapeutics, Inc.
13.3 Adaptimmune Therapeutics plc
13.4 Admedus Ltd.
13.4.1 CardioCel
13.4.2 CardioCel 3D
13.4.3 VascuCel
13.5 Advanced Bioscience Laboratories, Inc.
13.5.1 Biomanufacturing Services
13.5.1.1 Manufacturing of Virus-Based Products
13.5.1.2 Manufacturing of Protein-Based Products
13.5.2 Aseptic Filling Services
13.5.3 Immunology and Biomarker Testing
13.5.4 Contract Research Assays
13.5.5 Clinical Immunology Solutions
13.5.6 Preclinical Solutions
13.5.7 In vivo and ex vivo Preclinical Studies
13.5.8 Product Development Solutions
13.5.9 Government Partnering
13.5.10 Translational Product Development
13.6 Adverum Biotechnologies, Inc.
13.7 Akouos, Inc.
13.8 Aldevron, LLC
13.8.1 Services
13.9 Allogene Therapeutics, Inc.
13.10 AlloSource, Inc.
13.11 Amarna Therapeutics B.V.
13.11.1 Simian Virus 40 (SVac) Platform
13.12 Ambys Medicines, Inc.
13.13 American Gene Technologies International, Inc.
13.14 Amgen, Inc.
13.14.1 IMLYGIC (Talimogene laherparepvec)
13.15 Amicus Therapeutics, Inc.
13.16 Anterogen Co., Ltd.
13.16.1 Cupistem Injection
13.16.2 Queencell
13.17 APAC Biotech Pvt., Ltd.
13.17.1 APCEDEN
13.18 apceth Biopharma GmbH
13.19 AskBio/Asclepios Biopharmaceutical, Inc.
13.20 Asterias Biotherapeutics, Inc.
13.21 Atara Biotherapeutics, Inc.
13.22 Athersys, Inc.
13.22.1 MultiStem
13.23 ATVIO Biotech Ltd.
13.24 Audentes Therapeutics, Inc.
13.24.1 AT132 for X-Linked Myotubular Myopathy
13.24.2 AT342 for Crigler-Najjar Syndrome
13.24.3 AT982 for Pompe Disease
13.24.4 AT307 for CASQ2-CPVT
13.25 AveXis, Inc.
13.25.1 AVXS-101
13.26 Avita Medical Ltd.
13.26.1 RECELL System
13.26.2 ReGenerCell
13.26.3 ReNovaCell
13.27 AVROBIO, INC.
13.27.1 AVR-RD-01 for Fabry Disease
13.27.2 AVR-RD-02 for Gaucher Disease
13.27.3 AVR-RD-03 for Pompe Disease
13.27.4 AVR-RD-04 for Cystinosis
13.28 Batavia Bioscience B.V.
13.28.1 Research Services
13.28.2 Development Services
13.28.3 Characterization Services
13.28.4 Manufacturing Services
13.28.5 Consultancy Services
13.29 Bellicum Pharmaceuticals, Inc.
13.30 Benitec Biopharma Limited
13.31 Betalin Therapeutics
13.31.1 Engineered Micro-Pancreas (EMP)
13.32 BioCardia, Inc.
13.33 Biomay AG
13.33.1 Biomay’s Pipeline
13.34 BioReliance Corporation
13.35 Biosolution Co., Ltd.
13.36 Biostage, Inc.
13.36.1 Cellspan Esophageal Implants
13.36.2 Cellspan Bronchial Implants
13.36.3 Cellspan Tracheal Implants
13.37 Bioverativ, Inc.
13.38 Biovian Oy
13.39 bluebird bio, Inc.
13.39.1 Partnering
13.39.2 Current Partners
13.40 BlueRock Therapeutics
13.40.1 BlueRock’s Pipeline
13.41 Bone Therapeutics SA
13.41.1 ALLOB
13.41.2 JTA-004
13.42 Brainstorm Cell Therapeutics, Inc.
13.42.1 NurOwn
13.43 Brammer Bio, LLC
13.43.1 Services
13.43.2 Process Development
13.43.3 Gene Therapy Manufacturing
13.43.4 Viral Vectors
13.44 Caladrius Biosciences, Inc.
13.45 Carina Biotech
13.45.1 CNA1003
13.46 CARsgen Therapeutics, Ltd.
13.47 Casebia Therapeutics
13.48 Cell Cure Neurociences Ltd.
13.48.1 OpRegen
13.49 Cellerant Therapeutics, Inc.
13.50 Cell Medica Limited
13.51 Cell Therapy Catapult, Ltd.
13.51.1 Capabilities
13.52 Cellular Biomedicine Group, Inc.
13.53 Cellular Dynamics International, Inc.
13.54 Celonic AG
13.55 Celyad SA
13.56 Cevec Pharmaceuticals GmbH
13.56.1 Services
13.57 Farmaceutici S.p.A.
13.57.1 Holoclar
13.58 Cobra Biologics Ltd.
13.58.1 DNA Platform Process, Development and Scale Up
13.58.2 Viral Vector Platform Process
13.59 co-don AG
13.59.1 ACT3D
13.60 CombiGene AB
13.60.1 Epilepsy Project
13.61 Corestem, Inc.
13.61.1 NEURONATA-R inj.
13.62 CRISPR Therapeutics AG
13.63 CureVac
13.64 Cynata Therapeutics, Ltd.
13.65 CytomX Therapeutics, Inc.
13.66 Cytori Therapeutics, Inc.
13.67 Delphi Genetics S.A.
13.67.1 Services
13.68 Dendreon Pharmaceuticals, Inc.
13.68.1 PROVENGE
13.69 DNAlite Therapeutics, Inc.
13.70 Editas Medicine, Inc.
13.71 Elixirgen Therapeutics, LLC
13.72 Enzyvant, Inc.
13.72.1 RVT-802
13.73 Eukarys
13.74 Eureka Therapeutics, Inc.
13.74.1 ARTEMIS
13.74.2 E-ALPHA
13.75 Fate Therapeautics, Inc.
13.76 Fibrocell Technologies, Inc.
13.76.1 Fibrocell’s Pipeline
13.77 FinVector Vision Therapies Oy
13.77.1 Services
13.78 Freeline Therapeutics Ltd.
13.79 FUJIFILM Diosynth Biotechnologies, Inc.
13.79.1 Services
13.80 Gamida Cell Ltd.
13.81 GC Cell
13.82 Gemini Therapeutics, Inc.
13.83 GEG Tech
13.83.1 Lenti-ONE Vectors
13.84 Genethon
13.85 Generation Bio Co.
13.86 Genzyme Corporation
13.86.1 Epicel
13.87 Glycostem Therapeutics BV
13.87.1 Technology
13.87.2 oNKord
13.88 Hemostemix, Inc.
13.89 Histogenics Corporation
13.96 Homology Medicines, Inc.
13.96.1 Gene Editing Platform
13.97 Horama SAS
13.98 Humacyte, Inc.
13.99 Immune Design Corporation
13.100 Immusoft Corporation
13.100.1 ISP Technology
13.101 Innovative Cellular Therapeutics Co., Ltd.
13.102 Intellia Therapeutics, Inc.
13.103 Iovance Biotherapeutics, Inc.
13.104 JCR Pharmaceuticals Co., Ltd.
13.104.1 TEMCELL HS Inj.
13.105 J-TEC
13.106 JW CreaGene Co., Ltd.
13.106.1 Products
13.107 Kadimastem Ltd.
13.107.1 AstroRx
13.107.2 Incapsulin
13.108 Kaneka Eurogentec S.A.
13.109 KBI Biopharma, Inc.
13.110 Kiadis Pharma N.V.
13.110.1 ATIR101
13.111 Kite Pharma, Inc.
13.111.1 Yescarta
13.112 Kolon Lifescience, Inc.
13.112.1 Invossa-K inj.
13.113 Krystal Biotech, Inc.
13.114 LakePharma
13.114.1 Viral Vector Services
13.115 Lion TCR Pte. Ltd.
13.116 Lonza Group Ltd.
13.116.1 Custom Development and Manufacturing
13.117 Lysogene S.A.
13.117.1 MPS IIIA
13.117.2 GM1 Gangliosidosis
13.118 Magenta Therapeutics, Inc.
13.118.1 Magenta’s Programs
13.118.1.1 Transplant Conditioning
13.118.1.2 Stem Cell Expansion
13.118.1.3 Stem Cell Harvesting
13.118.1.4 Post Transplant Complications
13.119 MassBiologics
13.120 MaxCyte, Inc.
13.120.1 Products
13.120.1.1 MaxCyte STX
13.120.1.2 MaxCyte VLX
13.120.1.3 MaxCyte GT
13.121 Medigene AG
13.122 MEDIPOST CO., Ltd.
13.122.1 CARTISTEM
13.123.2 PNEUMOSTEM
13.123.3 NEUROSTEM
13.124 MeiraGTx Holdings plc
13.124.1 Meira’s Pipeline
13.124.2 AAV-CNGB3
13.124.3 AAV-RPE65
13.124.4 AAV-RPGR
13.124.5 AAV-AQP1
13.124.6 AAV-GAD
13.124.7 AAV-UPF1
13.125 Mesoblast Limited
13.126 Miromatrix Medical, Inc.
13.126.1 MIRODERM
13.126.2 MICROMESH
13.127 MolMed S.p.A
13.127.1 MolMed’s Clinical Trial Programs
13.127.2 MolMed’s GMP Solutions
13.128 multimmune GmbH
13.128.1 Platform Technology
13.129 Mustang Bio, Inc.
13.129.1 MB-101
13.129.2 MB-102
13.129.3 MB-103
13.129.4 MB-104
13.129.5 MB-105
13.129.6 MB-106
13.129.7 MB-107
13.130 Myonexus Therapeutics, Inc.
13.131 NantKwest, Inc.
13.132 NexImmune, Inc.
13.133 Nightstar Therapeutics plc
13.134 Neuralstem, Inc.
13.135 Nohla Therapeutics, Inc.
13.136 Novartis AG
13.136.1 Kymriah
13.137 Novasep Holding SAS
13.137.1 Biopharmaceutical Manufacturing Solutions
13.138 Noveome Biotherapeutics, Inc.
13.139 NuVasive, Inc.
13.139.1 Osteocel
13.139.2 AttraX Scaffold
13.140 Orchard Therapeutics plc
13.140.1 Strimvelis
13.140.2 OTL-101
13.140.3 OTL-102
13.140.4 OTL-103
13.140.5 OTL-200
13.140.6 OTL-201
13.140.7 OTL-202
13.140.8 OTL-300
13.141 Orgenesis, Inc.
13.141.1 MaSTHerCell
13.142 Orthocell Limited
13.143 Osiris Therapeutics, Inc.
13.143.1 Grafix
13.143.2 Grafix PL PRIME
13.143.3 Cartiform
13.143.4 BIO4
13.144 Orthofix Medical, Inc.
13.144.1 Trinity ELITE
13.144.2 Trinity EVOLUTION
13.144.3 VersaShield
13.145 Oxford BioMedica plc
13.145.1 Oxford BioMedica’s Partnering Activities
13.146 Oxford Genetics Ltd.
13.146.1 Scalable AAV Custom Solutions
13.147 Paragon Bioservices, Inc.
13.147.1 Services
13.148 Pharmicell Co., Ltd.
13.148.1 Cellgram AMI
13.149 Pluristem Therapeutics, Inc.
13.150 Posedia Therapeutics, Inc.
13.151 Precision BioSciences, Inc.
13.151.1 ARCUS Genome Editing Platform
13.152 Prevail Therapeutics, Inc.
13.152.1 Prevail’s Focus
13.153 PROMETHERA Biosciences S.A.
13.154 PTC Therapeutics, Inc.
13.155 Quethera Ltd.
13.155.1 Technology
13.156 Recipharm AB
13.157 Regenerative Medical System (RMS)
13.157.1 Chondron
13.157.2 Ossron
13.158 REGENXBIO, Inc.
13.158.1 NAV Technology
13.158.2 Therapeutic Programs
13.158.3 Product Candidates
13.158.3.1 RGX-314
13.158.3.2 RGX-501
13.158.3.3 RGX-111
13.158.3.4 RGX-121
13.158.3.5 RGX-181
13.158.4 Partnerships and Licensing
13.159 ReNeuron Group plc
13.160 Renova Therapeutics, Inc.
13.161 Richter-Helm BioLogics GmbH & Co. KG.
13.161.1 Process Development
13.161.2 Analytics
13.161.3 Production
13.162 Rocket Pharmaceuticals, Inc.
13.163 RoosterBio, Inc.
13.163.1 MSCs in different Formats
13.163.2 Bioprocess Media
13.163.3 Plug & Play Systems
13.163.4 CliniControl Products
13.164 RoslinCT
13.164.1 Services
13.165 Rubius Therapeutics, Inc.
13.166 Sangamo Therapeutics, Inc.
13.167 Sarepta Therapeutics, Inc.
13.168 SCM Lifesciences Co., Ltd.
13.169 Semma Therapeutics Inc.
13.169.1 SCbeta Cells
13.170 Sentien Biotechnologies, Inc.
13.170.1 SBI-101
13.171 Sibiono GeneTech Co., Ltd.
13.171.1 Gendicine
13.172 Sirion Biotech GmbH
13.172.1 Viral Vectors
13.172.2 Technologies
13.173 Solid Biosciences, Inc.
13.173.1 SGT-001 (Microdystrophin Gene Transfer)
13.174 Spark Therapeutics, Inc.
13.174.1 Luxturna (voretigene neparvovec)
13.174.2 Spark Therapeutics’ Pipeline
13.175 SQZ Biotechnologies
13.175.1 SQZ Platform
13.176 Stempeutics Research Pvt., Ltd.
13.176.1 Stempeucel
13.176.2 Stempeucare
13.176.3 Stempeutron
13.177 Synpromics Ltd.
13.177.1 Technology
13.177.2 PromPT
13.178 Takara Bio, Inc.
13.178.1 Services and Support
13.179 Terumo Corporation
13.179.1 HeartSheet
13.180 The Cell and Gene Therapy Catapult
13.180.1 Catapult’s Industrialization Team
13.180.2 Catapult’s Manufacturing Center at Stevenage
13.180.3 Catapult’s Regulatory Team
13.181 Translate Bio, Inc.
13.182 Triumvira Immunologics USA, Inc.
13.183 TxCell S.A.
13.183.1 Technology
13.183.2 CAR-Treg Transplantation (TX200)
13.183.3 CAR-Treg Dermatology
13.183.4 CAR-Treg Multiple Sclerosis
13.184 Ultragenyx Pharmaceutical, Inc.
13.184.1 DTX301
13.184.2 DTX401
13.185 uniQure N.V.
13.186 Vericel Corporation
13.186.1 MACI
13.186.2 Epicel
13.187 Vertex Pharmaceuticals, Inc.
13.188 VGXI, Inc.
13.189 ViaCyte, Inc.
13.189.1 Product Candidates
13.189.1.1 PEC-Direct
13.189.1.2 PEC-Encap
13.190 Vibalogics GmbH
13.191 Vybion, Inc.
13.191.1 INT41
13.192 Vigene Biosciences
13.193 Virovek, Inc.
13.193.1 Custom-Made AAVs
13.194 Vivebiotech SL
13.194.1 Lentisoma Technology
13.194.2 Services
13.195 Vivet Therapeutics
13.195.1 Vivet’s Approach
13.196 Voyager Therapeutics, Inc.
13.197 Waisman Biomanufacturing
13.197.1 Services and Products
13.198 WindMIL Therapeutics, Inc.
13.198.1 Unmodified MILs
13.198.2 MIL CARs
13.198.3 genMILs
13.199 Wuxi App Tec, Inc.
13.199.1 Services
13.200 Xalud Therapeutics, Inc.
13.200.1 XT-101

Figure 2.1: Number of RM Companies by Geography, 2018
Figure 2.2: Total RM Clinical Trials by Phase, 2017-2018
Figure 2.3: Total RM Clinical Trials by Technology Type
Figure 2.4: RM Industry-Total Global Financings: 2018
Figure 2.5: Total Financings to RM Industry by Type and Year, 2016-2018
Figure 2.6: Total Merger & Acquisition within RM Industry by Year, 2016-2018
Figure 2.7: Predicted CGT Product Launches, 2018-2030
Figure 2.8: Predicted Treated Patient Numbers, 2018-2030
Figure 3.1: Number of Clinical Trials using MSCs by Phase of Development
Figure 3.2: Percent (%) Share of Indications using MSCs in Clinical Trials
Figure 3.3: Types of Diseases Targeted by Neural Stem Cells in Clinical Trials
Figure 4.1: Geographical Distribution of CAR-T Cell Therapy Companies
Figure 4.2: Cancer Types Targeted by Dendritic Cell Therapy Studies
Figure 5.1: Diagrammatic Representation of Gene Augmentation Therapy
Figure 5.2: Schematic Representation of Gene Inhibition Therapy
Figure 5.3: Diagrammatic Representation of Therapy by Suicide Genes
Figure 5.4: Illustration Showing the Viral Vector Transferring the Gene into Patient’s Cell
Figure 5.5: Autologous vs. Allogeneic Gene Therapies in Development
Figure 5.6: Number of Gene and Gene-Based Medicine Companies by Geography
Figure 5.7: Gene Therapy Pipeline Growth by Year
Figure 5.8: Minor Role Played by Big Pharma in Cell/Gene Therapy Development
Figure 5.9: Number of Gene Therapy Products to Gain FDA Approval by 2022
Figure 5.10: Gene Therapy Companies Active in Neurodegenerative Diseases by Geography
Figure 7.1: Utilization of Lentivirus Vectors by Disease Type
Figure 7.2: Percent Shares of Companies in Lentiviral Vector Market
Figure 7.3: Types of Diseases in Clinical Trials using AAV
Figure 7.4: Percent Shares of Companies in AAV Vector Market
Figure 8.1: Number of Clinical Trials in RM Sector, 2017-2018
Figure 8.2: Number of Patient Participation in RM Clinical Trials, 2018
Figure 8.3: Number of Cell Therapy Clinical Trials, 2017-2018
Figure 8.4: Cell Therapy Clinical Trials by Indication, 2018
Figure 8.5: Clinical Trials using MSCs by Indication
Figure 8.6: Number of Gene-Modified Cell Therapy Clinical Trials, 2017-2018
Figure 8.7: The Popular Target Antigens for CAR-T in Clinical Trials
Figure 8.8: Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
Figure 8.9: Antigens Targeted by CAR-T in Solid Cancers in Clinical Trials
Figure 8.10: Number of Gene Therapy Clinical Trials, 2017-2018
Figure 8.11: Gene Therapy Clinical Trials by Geography
Figure 8.12: Diseases Targeted by Gene Therapy, 2018
Figure 8.13: Gene Types Transduced in Clinical Trials, 2018
Figure 8.14: Vectors used in Gene Therapy Clinical Trials, 2018
Figure 8.15: Gene Therapy for Neurodegenerative Indications by Type, 2018
Figure 8.16: Domination of Oncology in Gene Therapy Drug Development
Figure 8.17: Domination of Viral Vectors in Gene Therapy Drug Development
Figure 8.18: Gene Therapy Alliances Volume and Value, 2012-2018
Figure 8.19: Number of Clinical Trials in TE Space, 2017-2018
Figure 9.1: Number of RM Products with MA by Region/Country
Figure 9.2: Type of RM Products with MA Worldwide by Product Type, 2018
Figure 9.3: RM Products with MA Worldwide by Therapy Type, 2018
Figure 9.4: Number of RM Products with MA Worldwide by Indication, 2018
Figure 10.1: Expenditure Categories in Cell Products Manufacturing
Figure 10.2: The Impact of Head Count on Manufascturing Cost
Figure 10.3: Cost of Production in a Partially Automated Facility
Figure 10.4: Cost of Production in a Fully Automated Facility
Figure 10.5: NPC of Manual, Partially Automated and Fully Automated Processes
Figure 11.1: The Appropriate Phase for Outsourcing a Cell or Gene Therapy Project
Figure 11.2: Important Points Considered while Fixing a CDMO
Figure 11.3: Share of In-House and CDMO Manufacture of Cell and Gene Therapies
Figure 11.4: Number of Cell Gene Therapy CMOs/CDMOs in Europe
Figure 11.5: Projected Growth of Outsourcing Market, 2018-2025
Figure 11.6: Projected Growth of Cell Manufacturing Capacity, 2018-2025
Figure 12.1: Global Market for Regenerative Medicinal Products, 2018-2025
Figure 12.2: Global Market for RM Products by Geography, 2018-2025
Figure 12.3: Global Market for Approved Cell Therapy Products, 2018-2025
Figure 12.4: Global Market for Gene Therapy Products, 2018-2025
Figure 12.5: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
Figure 12.6: Global Market for Tissue-Engineered Products, 2018-2025

Table 2.1: Number of RM Companies by Geography, 2018
Table 2.2: Major Therapeutic Platforms and Enabling Technologies in RM Industry
Table 2.3: RM Product Approvals in 2017/2018
Table 2.4: Total RM Clinical Trials by Phase, 2017/2018
Table 2.5: Total RM Clinical Trials by Technology Type
Table 2.6: Patient Impact of Three RM Products, 2018
Table 2.7: Select Anticipated RM Industry Clinical Trial Data Readouts: 2019
Table 2.8: RM Industry-Total Global Financings, 2018
Table 2.9: Total Financings to RM Industry by Type and Year, 2016-2018
Table 2.10: Corporate Partnerships (Upfront Payments) in RM Industry, 2018
Table 2.11: Private Placements and Venture Financings in RM Industry, 2018
Table 2.12: Public Offerings (IPOs & Follow-Ons) in RM Industry, 2018
Table 2.13: Total Merger & Acquisitions Within RM Industry by Year, 2015-2018
Table 2.14: Market Access for Regenerative Medicines as of January 2019
Table 2.15: Top 20 RM Companies by Pipeline Size
Table 2.16: RM Research Institutes in North America
Table 2.17: RM Research Institutes in Europe
Table 2.18: RM Research Institutes in Australia
Table 2.19: From University Research to Products in the Market
Table 3.1: Timeline of NoTable Events in Cell Therapy Sector
Table 3.2: Common Indications for HSCT
Table 3.3: Leading iPS Cell Therapy Companies
Table 3.4: MSC-Based Products with Marketing Approval (MA)
Table 3.5: Select List of Companies Active in Clinical Trials using Neural Stem Cells
Table 3.6: Select Clinical Trials that Use hESCs
Table 3.7: The 29 Clinical Trials using hESCs, 2018
Table 3.8: Select Events in Cell Therapy Sector, 2018
Table 3.9: Top 15 Cell and Gene Therapy Biotechs to Watch
Table 4.1: Number of Companies Developing Gene-Modified Cell Therapy Products
Table 4.2: Geographical Distribution of CAR-T Cell Therapy Companies
Table 4.3: Select Events in Cell-Based Immuno-Onclogy Space, 2018
Table 4.4: Examples of Clinical Trials Involving T-Cell Receptors (TCRs)
Table 4.5: Select NK Cell Threrapies in Clinical and Preclinical Devrelopment for Cancer
Table 4.6: Examples of Clinical Trials using TILs
Table 4.7: Known Gamma-Delta T-Cell Therapy Companies
Table 4.8: Select Ongoing Clinical Trials Testing DC Vaccines
Table 5.1: Approved Gene Therapy Products (ex vivo and in vivo)
Table 5.2: Number of Gene and Gene-Based Medicine Companies by Geography
Table 5.3: Gene Therapy Product Candidates Granted RMAT Designation, 2018
Table 5.4: Gene Therapy Companies Active in ND by Region, 2018
Table 5.5: Gene Therapy Developers for Neurodegenerative Disorders, 2018
Table 5.6: Neurodegenerative Disorders Addressed by Gene Therapy Studies
Table 5.7: Select Anticipated Events in ND Space of Gene Therapy, 2018
Table 5.8: Select Events in Gene Therapy and Genome Editing Space, 2017/2018
Table 5.9: Leading Gene Therapy Startups
Table 6.1: Approved TE Products
Table 6.2: Select Events in Tissue Engineering Sector, 2018
Table 7.1: Attributes of Commonly used Viral Vectors
Table 7.2: Retroviruses: A Snapshot
Table 7.3: Adenoviruses: A Snapshot
Table 7.4: Herpes Simplex Viruses (HSV): A Snapshot
Table 7.5: Lentiviruses: A Snapshot
Table 7.6: Adeno-Associated Viruses: A Snapshot
Table 7.7: Genes Transduced by AAV for Specific Diseases
Table 7.8: Dominating Viral Vectors in Gene Therapy Drug Development
Table 7.9: Microinjection Method of Gene Delivery
Table 7.10: Particle Bombardment Method of Gene Delivery
Table 7.11: Laser Irradiation Method of Gene Delivery
Table 7.12: Electroporation Method of Gene Delivery
Table 7.13: Magnetofection Method of Gene Delivery
Table 7.14: Sonoporation Method of Gene Delivery
Table 8.1: Number of Clinical Trials in RM Sector, 2017-2018
Table 8.2: Number of Patient Participation in RM Clinical Trials, 2018
Table 8.3: Clinical Trials by Therapeutic Category in the RM Sector, 2018
Table 8.4: Number of Cell Therapy Clinical Trials, 2017-2018
Table 8.5: Number of Clinical Trials using MSCs
Table 8.6: Select Anticipated Clinical Data and Events in Cell Therapy Sector
Table 8.7: Number of Gene-Modified Cell Therapy Clinical Trials, 2017-2018
Table 8.8: Antigens other Than CD19 in Clinical Trials Targeted by CAR-T
Table 8.9: Select Anticipated Clinical Data and Events in CAR-T Cell Therapy Sector
Table 8.10: Number of Gene Therapy Clinical Trials, 2017-2018
Table 8.11: Gene Therapy Clinical Trials by Geography
Table 8.12: Diseases Targeted by Gene Therapy, 2018
Table 8.13: Gene Types Transduced in Clinical Trials, 2018
Table 8.14: Vectors used in Gene Therapy Clinical Trials, 2018
Table 8.15: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type, 2018
Table 8.16: Domination of Oncology in Gene Therapy Drug Development
Table 8.17: Gene Therapy Alliances Volume and Value, 2012-2018
Table 8.18: Select Anticipated Clinical Data and Events in Gene Therapy Sector
Table 8.19: Number of Clinical Trials in TE Space, 2017-2018
Table 8.20: Select Anticipated Clinical Data and Events in Tissue Engineering Space
Table 9.1: RM Products with MA by Region/Country
Table 9.2: RM Products with MA Worldwide by Year
Table 9.3: RM Products with MA Worldwide by Product Type, 2018
Table 9.4: RM Products with MA Worldwide by Therapy Type, 2018
Table 9.5: Number of RM Products with MA Worldwide by Indication, 2018
Table 9.6: Cell Therapy Product with MA in Australia by TGA
Table 9.7: Cell Therapy and Gene Therapy Products with MA in Canada by Health Canada
Table 9.8: Gene Therapy Product with MA in China by CFDA
Table 9.9: RM Products with MA in Europe by EMA
Table 9.10: RM Products with MA Withdrawn in the E.U. by EMA
Table 9.11: Cell Therapy Products with MA in India by DCGI
Table 9.12: Cell and Tissue Engineered Products with MA in Japan by PMDA
Table 9.13: Cell Therapy Products with MA in New Zealand by MEDSAFE
Table 9.14: RM Products with MA in South Korea by MFDS
Table 9.15: RM Products with MA in the U.S. by FDA
Table 9.16: RM Products with RMAT Designation by FDA
Table 9.17: Pricing of RM Products
Table 9.18: The Five Forthcoming RM Products
Table 10.1: Expenditure Categories in Cell Products Manufacturing
Table 10.2: The Impact of Head Count on Labor Cost
Table 10.3: Cost of Production in a Partially Automated Facility
Table 10.4: Cost of Production in a Fully Automated Facility
Table 10.5: In-House Gene Therapy Manufacturing Capabilities of Select Big and Mid Pharma
Table 10.6: External Gene Therapy Manufacturing by Big and Mid Pharma
Table 10.7: Cell and Gene Therapy Manufacturing Capacities in North America
Table 10.8: Cell and Gene Therapy Manufacturing Capacities in Europe
Table 11.1: Select CDMOs in Cell and Gene Therapy Sector
Table 11.2: The Appropriate Phase for Outsourcing a Cell or Gene Therapy Project
Table 11.3: Important Points Considered While Fixing a CDMO
Table 11.4: Share of In-House and CDMO Manufacture of Cell and Gene Therapies
Table 12.1: Global Market for RM Products by Therapy Type, 2018-2025
Table 12.2: Global Market for RM Products by Geography, 2018-2025
Table 13.3: Global Market for Approved Cell Therapy Products, 2018-2025
Table 12.4: Global Market for Gene Therapy Products, 2018-2025
Table 12.5: Competitive Landscape in Gene-Modified Cell Therapy Products
Table 12.6: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
Table 12.7: Global Market for Tissue-Engineered Products, 2018-2025
Table 13.1: 4D Molecular Therapeutics’ Product Pipeline
Table 13.2: Abeona Therapeutics’ Product Pipeline
Table 13.3: Adaptimmune’s Pipeline Programs
Table 13.4: Allogene Therapeutics’ Pipeline of Allogeneic T-Cell Product Candidates
Table 13.5: Amicus’ Gene Therapy Pipeline
Table 13.6: apceth’s Pipeline Overview
Table 13.7: AskBio’s Therapeutic Pipeline
Table 13.8: Asterias’ Pipeline Programs
Table 13.9: Atara’s Pipeline of T-Cell Immunotherapy and CAR-T Product Candidates
Table 13.10: Athersys’ Clinical Programs using MultiStem
Table 13.11: Audentes’ Product Candidates
Table 13.12: AVROBIO’s Gene Therapy Programs
Table 13.13: Bellicum Pharmaceuticals’ Product Candidates
Table 13.14: Benitec’s Product Pipeline using Gene Silencing Technologies
Table 13.15: BioCardia’s Advanced Pipeline
Table 13.16: Biomay’s Pipeline of Products
Table 13.17: Biosolution’s Products and Product Candidates
Table 13.18: Bioverativ’s Product Pipeline
Table 13.19: bluebird bio’s Product Pipeline
Table 13.20: Brainstorm’s Clinical Programs
Table 13.21: Caladrius’ Proprietary Technology Platforms and Pipeline Programs
Table 13.22: CARsgen Therapeutics’ Product Candidates
Table 13.23: Casebia’s Pipeline at-a-glance
Table 13.24: Cellerant’s Program Pipeline for Blood Disorders
Table 13.25: Cell Medica’s Product Pipeline
Table 13.26: CBG’s Immuno-Oncology Stem Cell Therapy Pipeline
Table 13.27: Celyad’s Programs’ Status at a Glance
Table 13.28: Corestem’s Key Clinical Pharmaceutical Development
Table 13.29: CRISPR Therapeutics’ Clinical Programs
Table 13.30: CureVac’s RNA-Based Product Pipeline
Table 13.31: CytomX’s Pipeline of Probody Therapeutics
Table 13.32: Cytori’s Cell Therapy Development Pipeline
Table 13.33: Editas’ Diverse Product Pipeline
Table 13.34: Elixirgen’s Pipeline for Genetic and Age-Related Diseases
Table 13.35: Eureka’s Development Pipeline
Table 13.36: Fate Therapeutics’ Cell Therapy Product Candidates Pipeline
Table 13.37: Fibrocell’s Personalized Biologics Pipeline
Table 13.38: Freeline’s Pipeline of Gene Therapy Treatments in Development
Table 13.39: Gamida Cell’s Pipeline Programs
Table 13.40: Current Status of GC Cell’s Clinical Activities
Table 13.41: Gemini’s Pipeline
Table 13.42: Genethon’s Products in Development
Table 13.43: Hemostemix’s Clinical Pipeline
Table 13.44: Horama’s Product Pipeline
Table 13.45: Immune Design’s Product Pipeline
Table 13.46: ICT’s Pipeline Programs
Table 13.47: Intellia’s In Vivo and Ex Vivo R&D Pipeline
Table 13.48: Iovance’s Clinical Pipeline
Table 13.49: JCR’s RM Products in Development
Table 13.50: Kite’s CAR-T Pipeline
Table 13.51: Kolon’s Gene Therapeutics in Development
Table 13.52: Krystal Biotech’ Pipeline Programs for Orphan Skin Disease
Table 13.53: Lion TCR’s Product Candidates
Table 13.54: Magenta’s Product Candidates
Table 13.55: Medigene’s Immunotherapy Pipeline Projects
Table 13.56: MeiraGTX’s Pipeline
Table 13.57: Mesoblast’s Advanced Product Candidates
Table 13.58: MolMed’s Product Candidates
Table 13.59: Mustang Bio’s Product Pipeline
Table 13.60: Myonexus’ Clinical Stage LGMD Gene Therapy Pipeline
Table 13.61: NantKwest’s Clinical Trials using Cryopreserved haNK Cells
Table 13.62: NexImmune’s Adoptive Cellular Therapy (ACT) Programs
Table 13.63: Nightstar Therapeutics’ Pipeline Programs
Table 13.64: Neuralstem’s Cell Therapy Programs
Table 13.65: Nohla’s Donor Cell Therapy Programs
Table 13.66: Noveome’s Clinical Pipeline
Table 13.67: Orchard Therapeutics’ Pipeline
Table 13.68: Oxford BioMedica’s Proprietary Product Pipeline
Table 13.69: Oxford BioMedica’s Partnered Products
Table 13.70: Oxford BioMedica’s IP Enabled and Royalty Bearing Products
Table 13.71: Pharmicell’s Clinical Trials for Commercialization
Table 13.72: Posedia’s Product Pipeline
Table 13.73: PROMETHERA’s Product Pipeline
Table 13.74: PTC Therapeutics’ Gene Therapy Programs for CNS Disorders
Table 13.75: REGENXBIO’s Product Pipeline
Table 13.76: REGENXBIO’s Partners & Licensees Pipeline
Table 13.77: ReNeuron’s Program Pipeline
Table 13.78: Renova’s Product Pipeline
Table 13.79: Rubius’ Pipeline Programs
Table 13.80: Sangamo’s Proprietary Programs
Table 13.81: Sangamo’s Proprietary Programs
Table 13.82: Sarepta’s Huge Product Pipeline
Table 13.83: SCM Lifesciences’ Product Pipeline
Table 13.84: Sentien’s SBI-101 Clinical Programs
Table 13.85: Spark Therapeutics’ Pipeline
Table 13.86: Translate Bio’s Two Lead Product Candidates
Table 13.87: Triumvira’s Product Pipeline
Table 13.88: uniQure’s Product Pipeline
Table 13.89: Vertex Pharmaceuticals’ Product Candidates
Table 13.90: ViaCyte’s Product Pipeline
Table 13.91: Vybion’s Drug Pipeline
Table 13.92: Voyager’s Pipeline Programs