Gene Therapy Pipeline and Portfolio Analysis
Gene Therapy Pipeline and Portfolio Analysis
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Gene therapy has undergone transformative enhancements over the last 20 years, with improvements to technologies and advances in the pipeline that aim to invigorate the field in its second generation.
Author: Amanda Micklus
Publisher: Datamonitor Healthcare
- Description
- Contents
Overview
Background
Gene therapy has undergone transformative enhancements over the last 20 years, with improvements to technologies and advances in the pipeline that aim to invigorate the field in its second generation. A dozen products have made it to various markets worldwide, including several within the last few years, and more are expected to gain approval soon. The pipeline is dominated by oncology ex vivo targets, but there are many approaches being pursued by key players outside of this core area.
Gene therapy has overcome setbacks since its inception in the early 1970s
The death of a patient with partial ornithine transcarbamylase (OTC) deficiency in 1999, as well as reports of leukemia following delivery of gene therapy in the early 2000s, deterred development for some years; however, the current generation has benefited from improvements to technology and drug delivery.
Gene therapy developers have employed both viral and non-viral methods to deliver genes
There are several advantages to using either viral or non-viral vectors, with the former offering efficient gene transfer, and the latter leading to lower costs and greater biosafety. But both also come with drawbacks that are prompting evaluation of other delivery methods.
The EMA and FDA have both introduced regulations that foster regenerative medicine development
The EMA introduced a regulation in 2007 that created a regulatory framework for advanced therapy medicinal products (ATMPs), and the FDA established the regenerative medicine advanced therapy (RMAT) pathway in 2016 through the passage of the 21st Century Cures Act. Gene therapies are part of the scopes of both, and sponsors may take advantage of frequent interactions with regulators plus other benefits.
Hundreds of companies are involved in gene therapy development
There are approximately 427 unique companies – acting as originators or licensees – with development-stage candidates. These include very small players working on only one or two therapies, all the way up to more active companies with larger pipelines totaling around 20 programs.
Globally, there are 12 approved or launched gene therapies in the market
At the end of Q1 2019, there are 12 gene therapies approved or launched worldwide, including a nearly equal split of in vivo (seven) and ex vivo (five) therapies. All but two of the products are delivered by viral vectors, and most of the launches have been in Europe, followed by Asia.
Just over half of the pipeline is comprised of in vivo therapies
A slight majority (52%) of the pipeline is delivered in vivo. The rest of the candidates are cell and gene therapies, and within this group 57% are autologous as opposed to allogeneic. The approach taken – in vivo (gene therapy) or ex vivo (cell and gene therapy) – varies depending on many factors, including the site of the disease and how accessible those targets are.
Solid tumors are the major focus of oncology gene therapy development
The majority of oncology gene therapies (57%) are looking to address solid tumors, but hematological cancers also have a strong proportion (43%) of development in the area.
CONTENTS
7 OVERVIEW
8 INTRODUCTION TO GENE THERAPY
8 Datamonitor Healthcare insights and recommendations
8 Gene therapy is the most active modality in regenerative medicine
10 Historically, gene therapy has been riddled with setbacks
10 Gene therapy involves in vivo and ex vivo approaches
11 Bibliography
13 INNOVATIONS IN GENE DELIVERY TECHNOLOGIES
13 Datamonitor Healthcare insights and recommendations
13 Gene therapy is enabled by viral and non-viral methods
21 Bibliography
26 REGULATORY ISSUES
26 Datamonitor Healthcare insights and recommendations
26 Regulatory pathways are providing incentives for gene therapy development
38 Bibliography
41 KEY COMPANIES IN GENE THERAPY
41 Datamonitor Healthcare insights and recommendations
41 Hundreds of companies are involved in gene therapy development
43 REGENXBIO is building its internal pipeline and out-licensing its NAV platform
46 Now owned by Celgene, Juno is pushing ahead with a third-to-market CAR-T
49 Kite Pharma’s cell therapy pipeline will become the foundation of Gilead’s oncology offering
51 CAR-T therapy and directly administered gene therapy are both focuses for Novartis
54 Sarepta targets gene therapies for muscular dystrophies
56 Through collaborations and in-house development, bluebird bio is moving forward with ex vivo gene therapies in multiple diseases
57 Bibliography
60 TRENDS IN APPROVED GENE THERAPIES
60 Datamonitor Healthcare insig
60 A dozen gene therapies are approved worldwide
65 Key gene therapy approvals
68 Bibliography
72 GENE THERAPY PIPELINE TRENDS
72 Datamonitor Healthcare insights and recommendations
72 After plateauing, the gene therapy pipeline is picking up speed
74 The US leads ongoing clinical trial activity in gene therapies
75 Completed gene therapy trials have high success rates
76 Most terminated gene therapy trials are those that were planned but never initiated
77 Both gene therapy and cell and gene therapy approaches are on fairly equal ground
78 Across phases, in vivo and ex vivo therapies are split fairly equally
80 Viral vectors are used more than non-viral vectors for delivery
87 Most gene therapies in development aim to attack rare diseases and cancer
97 Outside of oncology, different diseases emerge in the lead among in vivo and ex vivo therapies
99 Bibliography
100 ONCOLOGY GENE THERAPY TRENDS
100 Datamonitor Healthcare insights and recommendations
100 The majority of gene therapies in the pipeline are for solid tumors
104 CAR therapies are dominant in ex vivo development
108 APPENDIX
108 Scope
108 Methodology
LIST OF FIGURES
9 Figure 1: Gene therapy defined: EMA and FDA definitions
11 Figure 2: Gene therapy delivery approaches, in vivo versus ex vivo
14 Figure 3: Viral and non-viral vectors – advantages and disadvantages
15 Figure 4: Key viral vector types – advantages and disadvantages
27 Figure 5: Product class inclusions in EU and US specialized regenerative medicine regulatory pathways
43 Figure 6: Most active gene therapy companies, by pipeline size
65 Figure 7: Most approved or launched gene therapies are available in Europe
73 Figure 8: Gene therapy pipeline volume, preclinical through pre-registration phase, 1995–2018
74 Figure 9: Gene therapy pipeline volume by phase, 1995–2018
75 Figure 10: Most gene therapy clinical trial activity is in the US
76 Figure 11: Completed gene therapy trials are largely successful; trials by outcomes
77 Figure 12: Top reasons why gene therapy trials are terminated
78 Figure 13: Gene therapy versus cell and gene therapy: breakdown of pipeline
79 Figure 14: Gene therapy pipeline, by in vivo or ex vivo approach and phase
80 Figure 15: Ex vivo gene therapy pipeline, by cell type and phase
81 Figure 16: Viral vector delivery is most prominent in gene therapy
82 Figure 17: In viral vector gene delivery, AAVs are most often used
83 Figure 18: Viral vector gene therapy pipeline, by vector and phase
84 Figure 19: In vivo gene therapies, viral versus non-viral delivery
85 Figure 20: In vivo gene therapies, by vector type
86 Figure 21: Ex vivo gene therapies, viral versus non-viral delivery
87 Figure 22: Ex vivo gene therapies, by vector type
88 Figure 23: Rare disease and oncology dominate gene therapy drug development
89 Figure 24: Gene therapy pipeline, by therapy area and phase
90 Figure 25: Cancer and non-oncologic rare disease diverge in ex vivo versus in vivo delivery
91 Figure 26: Viral vectors are largely used in both cancer and rare non-oncologic gene therapies
92 Figure 27: Other therapy areas are mostly being addressed by in vivo therapy
93 Figure 28: Viral vectors are also most prominent in other therapy areas
94 Figure 29: In vivo gene therapies, by therapy area and viral vector
95 Figure 30: Ex vivo gene therapies, by therapy area and viral vector
96 Figure 31: Lead molecular targets among in vivo therapies
97 Figure 32: Lead molecular targets among ex vivo therapies
98 Figure 33: Lead indications among non-oncology in vivo therapies
99 Figure 34: Lead indications among non-oncology ex vivo therapies
101 Figure 35: Oncology gene therapies are mainly addressing solid tumors
102 Figure 36: Developers focus on ex vivo therapy in cancer
103 Figure 37: Tumor type distribution between in vivo and ex vivo therapies in oncology
104 Figure 38: Top 20 oncology indications in gene therapy development, by approach
105 Figure 39: CAR therapies dominate: oncology ex vivo gene therapies, by approach
106 Figure 40: Ex vivo oncology approaches, by phase
107 Figure 41: Autologous versus allogeneic delivery in oncology ex vivo CAR therapies
LIST OF TABLES
33 Table 1: Summary of FDA guidance on cell and gene therapy
45 Table 2: REGENXBIO gene therapy pipeline
48 Table 3: Juno Therapeutics/Celgene gene therapy pipeline
51 Table 4: Kite Pharma/Gilead gene therapy pipeline
53 Table 5: Novartis gene therapy pipeline
55 Table 6: Sarepta gene therapy pipeline
57 Table 7: Bluebird bio gene therapy pipeline
62 Table 8: Approved gene therapies worldwide, as of Q1 2019
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