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Gene Therapy Market, (2nd Edition), 2018-2030

Gene Therapy Market, (2nd Edition), 2018-2030
INTRODUCTION

The concept of gene transfer into mammalian cells can be traced back to the 1920s. However, the first gene therapy, Gendicine®, was only approved in 2003 in China; since then, the domain has evolved significantly. The year 2017 was particularly eventful; despite the withdrawal of Glybera® from the European market in early 2017, the latter half of the year witnessed the approval of two gene therapies, namely Invossa™ and Luxturna™. In fact, Luxturna™ became the first gene therapy to gain approval in the US. Further, promising clinical results were reported for several gene therapies that are currently in late phases of development. The growing popularity and potential of gene therapies can be correlated with an exponential increase in the number of patents that have been filed; the cumulative number has increased from 7,300 patents in 2013 to 42,300 patents till the third quarter of 2017. Further, over the past five years, capital worth more than USD 9.8 billion has been invested by venture capital (VC) firms and government bodies to fund research activities in this domain.

The overall market is expected to witness significant growth in opportunities for a variety of stakeholders in the coming decade. It is important to highlight that several technology providers, aiming to develop and / or support the development of gene therapies, with improved efficacy and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation in this domain has also led to the discovery of novel molecular targets and strengthened the research pipelines of companies focused in this space. The capability to target diverse therapeutic areas is considered to be amongst the most prominent growth drivers of this market.

SCOPE OF THE REPORT
The ‘Gene Therapy Market (2nd Edition), 2018-2030’ report provides an extensive study on the current market landscape of gene therapies, with a prime focus on gene augmentation based therapies and oncolytic viral therapies, featuring an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:
A detailed assessment of the current market landscape of gene therapies, providing information on various drug / therapy developers, phase of development (clinical, preclinical or discovery stage) of product candidates, key therapeutic areas and indication(s), information on gene type, vector type, type of therapy (ex vivo, in vivo) and type of modification (gene augmentation, oncolytic viral therapy and others).
A discussion on the various types of viral and non-viral vectors, highlighting information on design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vectors.
A world map representation, depicting the most active geographies in terms of the presence of companies developing gene therapies, and a bull’s eye analysis highlighting the distribution of clinical pipeline candidates in terms of phase of development, type of vector and type of therapy (ex vivo / in vivo).
Comprehensive profiles of marketed drugs, highlighting their history of development, current developmental status, mechanism of action, affiliated technology, patent portfolio, dosage and manufacturing, and information on the developer.
Comprehensive profiles of clinical stage (phase II/III and above) drug candidates, highlighting their current status of development, mechanism of action, affiliated technology, patent portfolio, clinical trial information and recent developments.
A section on emerging technologies and platforms that are aiding the development of gene therapies, featuring detailed profiles of technologies that are presently being used in the development of four or more products / product candidates.
An overview of the most commonly targeted therapeutic indications and details on the gene therapies that are being developed against them.
An analysis of the investments made at various stages of development in companies that are focused in this area, including seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings.
A detailed analysis of the recently filed patents (since 2013); the study highlights the emerging trends in innovation and identifies the key players involved. In addition, it presents a high-level view on the valuation of these patents as well.
A case study on the prevalent and emerging trends in vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study includes a detailed discussion on the manufacturing processes of various types of vectors as well.
An analysis on the various factors that may form the basis for the pricing of gene therapies, featuring different models / approaches that may be adopted in order to decide the price of a product that is likely to be marketed in the coming years.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the market. Based on parameters, such as target consumer segments, likely adoption rates and expected pricing, we have provided an informed estimate of the likely evolution of the market in the short to mid-term and mid to long term, for the period 2018-2030. To account for the uncertainties associated with the development of gene therapies and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market’s evolution.

The opinions and insights presented in the report were also influenced by discussions held with senior stakeholders in the industry. The study includes detailed transcripts of discussions held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory), Michael Triplett (CEO, Myonexus Therapeutics) and Robert Jan Lamers (CEO, Arthrogen). All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

EXAMPLE HIGHLIGHTS
1. Around 300 product candidates are currently under various stages of development for a diverse range of indications. Eight gene therapies are commercially available; of these, Imlygic® (Amgen), Strimvelis® (GSK), Invossa™ (TissueGene / Kolon Life Science / Mitsubishi Tanabe Pharma) and Luxturna™ (Spark Therapeutics) were approved after 2015. Nearly 48% of the pipeline molecules are under clinical development; of these, 21 molecules are being investigated in phase II/III and above, 8 molecules in phase III (planned), 21 molecules in phase II, 48 molecules in phase I/II and 49 molecules in phase I clinical trials. However, majority (49%) of the product candidates in the pipeline are still in the preclinical and discovery stages.
2. A significant proportion (59%) of product candidates in the pipeline follow the gene augmentation modification strategy, by delivering functional copies of missing genes into diseased hosts. Other types of modifications include immunotherapy (20%; delivering genes coding for antigens in order to generate an immune response) and oncolytic therapy (10%; using replication competent viruses to infect and kill cancer cells). Late stage (phase II/III and above) oncolytic therapies that are under development include ProsAtak® (prostate cancer), Pexa-Vec (HCC) and Toca-511 (GBM). In addition, a significant share of the pipeline comprises of in vivo gene therapies against various indications.
3. The market landscape is characterized by the presence of large-sized (17), mid-sized (34) and small-sized companies (63). Some of the prominent large-sized companies engaged in this domain include (in alphabetical order) Amgen, Biogen, BioMarin Pharmaceuticals, BMS, GSK, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi. Similarly, mid-sized companies that are actively contributing to the development of gene therapies include (based on year of approval) Spark Therapeutics, Kolon Life Science and Human Stem Cell Institute. In addition, small companies, such as (in alphabetical order) Agilis Biotherapeutics, Angionetics (subsidiary of Taxus Cardium Therapeutics), AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics and XyloCor, are also actively involved in this domain.
4. Close to 25% of the products in the development pipeline are designed to treat various oncological indications, including (ordered by decreasing number of gene therapies in the pipeline) prostate cancer, bladder cancer, head and neck cancer, breast cancer, lung cancer, ovarian cancer and pancreatic cancer. Nearly 15% of therapy candidates are being developed for the treatment of metabolic disorders (ordered by decreasing number of gene therapies under development), including mucopolysaccharidosis (MPS) IIIA, MPS IIIB, ornithine transcarbamylase (OTC) deficiency and diabetes. We also observed that close to 14% of the pipeline molecules are being developed for treating ocular disorders, such as (ordered by decreasing number of gene therapies under development) retinitis pigmentosa, wet age associated macular degeneration, achromatopsia and Leber's hereditary optic neuropathy.
5. AAV (38%) is presently the preferred type of vector used by drug developers to design gene therapies. This can be attributed to the advantages offered by these vectors, such as high viral yields, mild immunogenic response, ability to infect a wide spectrum of cells and a better safety profile. Other prominent delivery vehicles include adenovirus (16.5%), lentivirus (14.5%) and non-viral vectors (12%), such as plasmid DNA.
6. Over 34,000 patents, related to gene therapies, have been filed in last five years alone. Research efforts in this domain are led by both the industry and academia. Leading academic players that are actively engaged in developing gene therapies include (in decreasing order of number of patents) University of California, University of Texas, Stanford University, Harvard University, Massachusetts Institute of Technology and Johns Hopkins University.
7. Around 240 funding instances, amounting to USD 9.5 billion worth of capital, have taken place in order to support R&D in gene therapies in the last five years. California Institute for Regenerative Medicine (CIRM), OrbiMed Advisors, Deerfield Management, Versant Ventures, Novartis Venture Fund, National Institute of Health (NIH) and 5AM Ventures have emerged as some of the prominent investors (in terms of number of funding instances).
8. With many candidates in late stages of development, the market is likely to witness several approvals and grow steadily over the coming decade. Despite the fact that the first gene therapies were approved in Asian countries, North America and the EU together are likely to capture the over 75% of the market share by 2030. Within Asia Pacific region, South Korea is expected to dominate the market by 2030.

RESEARCH METHODOLOGY
The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include
Annual reports
Investor presentations
SEC filings
Industry databases
News releases from company websites
Government policy documents
Industry analysts’ views

While the focus has been on forecasting the market over the coming 12 years, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINES
Chapter 2 provides an executive summary of the insights captured during our research. It offers a high-level view on the likely evolution of the gene therapies in the short to mid-term and mid to long term.

Chapter 3 provides a general overview of gene therapies covering their historical background, popular types (somatic versus germline; in vivo versus ex vivo), application areas and route of administration. It also includes information on the steps required to transfer gene(s) into the body, along with a discussion on the advantages and disadvantages of the process. Further, the chapter features a discussion on the ethical and social concerns related to gene therapies, and highlights certain future constraints and challenges in terms of manufacturing, reimbursements and commercial viability. It also presents details on the regulatory guidelines for these therapies in the US, Europe and China.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available vectors.

Chapter 5 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. It also features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes related to gene therapies. In addition, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 6 includes information on over 300 gene therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, target therapeutic areas and indications, phases of development, vectors, target gene segment, type of therapy (gene augmentation / oncolytic viral therapy / others) and type of somatic cell therapy (in vivo / ex vivo). In addition, it features a schematic world map representation, highlighting the key regional hubs where gene therapies are being developed for the treatment of various disorders. Further, we have provided a logo landscape of product developers in North America, Europe and the Asia Pacific region on the basis of company size.

Chapter 7 provides detailed profiles of marketed gene therapies. Each profile includes an overview of the developer and information on various other parameters, such as history of development, indication, mechanism of action, patent portfolio, current developmental status, target, clinical trial results and manufacturing information.

Chapter 8 contains detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each profile provides information on the mechanism of action, current status of development, route of administration, affiliated technology platform (if applicable), dosage form, clinical studies and key clinical trial results.

Chapter 9 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. It features brief profiles of some of the key technologies as well. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships established related to the same. In addition, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 10 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include cardiovascular disorders, hematological disorders, metabolic disorders, muscular disorders, neurological disorders, ocular disorders and oncology.

Chapter 11 presents details on various investments and grants received by companies that are engaged in this domain. It includes an analysis of the funding instances that have taken place in the period between 2013-2017, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 12 highlights our view point on the various factors that may be taken into consideration while pricing gene therapies. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt to decide the price at which their gene therapy product is likely to be marketed.

Chapter 13 provides a detailed analysis of the patents that have been filed in this domain. The study highlights emerging trends in recently filed patents, and identifies the key players that are driving the innovation in this space. Moreover, it presents a high-level view on the valuation of these patents as well.

Chapter 14 presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of key therapeutic areas (cardiovascular disorders, hematological disorders, muscular disorders, ocular disorders, oncology and others), type of vector (AAV, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy (ex vivo and in vivo) and geography (the US, EU, RoW (Australia, China, Israel Japan and South Korea)).


Chapter 15 is a summary of the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.

Chapter 16 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) Michael Triplett (CEO, Myonexus Therapeutics) and Robert Jan Lamers (CEO, Arthrogen). In addition, a brief profile of each company has been provided.

Chapter 17 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 18 is an appendix, which provides the list of companies and organizations mentioned in the report.

LIST OF COMPANIES / LIST OF COMPANIES AND ORGANIZATIONS

The following companies and organizations have been mentioned in the report.

1. 4BIO Capital
2. 5AM Ventures
3. AAVogen
4. Abbott
5. Abby Grace Foundation
6. Abeona Therapeutics
7. Abingworth
8. ABL Europe
9. Ablynx
10. Abraxis BioScience
11. Acceleron Pharma
12. Acucela (Subsidiary of Kubota Pharmaceutical)
13. Adage Capital Management
14. Aduro Biotech
15. Advanced BioScience Laboratories (ABL)
16. Advanced Biotherapeutics Consulting
17. Advantagene
18. Advaxis Immunotherapies
19. Adverum Biotechnologies
20. Agent Capital
21. Agilis Biotherapeutics
22. Ajinomoto Althea
23. AJU IB Investment
24. Aldevron
25. Alexandria Venture Investments
26. Allele Biotechnology and Pharmaceuticals
27. Ally Bridge Group (ABG)
28. Alpha-1 Foundation
29. AlphaVax
30. Amgen
31. AMSBIO
32. Anaeropharma Science
33. Anemocyte (previously known as Areta International)
34. AnGes
35. Angionetics
36. Apic Bio
37. Applied Genetic Technologies Corporation (AGTC)
38. Applied Viromics
39. ARCH Venture Partners
40. Arthrogen
41. AstraZeneca
42. Athena Vision
43. Atlas Venture
44. aTyr Pharma
45. Audentes Therapeutics
46. AveXis
47. AVROBIO
48. Baillie Gifford
49. Bamboo Therapeutics (acquired by Pfizer)
50. Batavia Biosciences
51. Bayer
52. Beckman Research Institute
53. Beijing Capital Grand (previously known as Beijing Capital Juda)
54. Benitec Biopharma
55. BioCanCell Therapeutic
56. Biogen
57. BioMarin Pharmaceutical
58. Biomay
59. Biomiga
60. BioMotiv
61. BioNTech Innovative Manufacturing Services (previously known as EUFETS)
62. BioReliance / SAFC
63. Bioverativ
64. Biovian
65. BioViva
66. Blue Sky BioServices
67. bluebird bio
68. BlueWater Angels
69. Boehringer Ingelheim BioXcellence™
70. Boehringer Ingelheim Venture Fund
71. Bpifrance
72. Brammer Bio
73. Bristol-Myers Squibb
74. Broad Institute
75. Brookside Capital Partners Fund
76. California Institute for Regenerative Medicine (CIRM)
77. Cambridge Enterprise
78. Cancer Prevention and Research Institute of Texas (CPRIT)
79. CapDecisif Management
80. Caribou Biosciences
81. Casdin Capital
82. Celgene
83. Cell and Gene Therapy Catapult
84. Cellectis
85. CellGenTech
86. Celsion
87. Center for the Development of Industrial Technology (CDTI)
88. Centre for Process Innovation
89. Centre national de la recherche scientifique (CNRS)
90. CEVEC Pharmaceuticals
91. CHDI Foundation
92. Chiesi Farmaceutici
93. Children’s Medical Research Foundation
94. Children's Hospital of Philadelphia
95. Children's Medical Research Foundation
96. Clarus Ventures
97. Clough Capital Partners
98. Cobra Biologics and Pharmaceutical Services
99. Cold Genesys
100. Columbus Venture Partners
101. Commercial & External Partnership, Industrial Affairs (CEPiA)
102. Copernicus Therapeutics
103. Cormorant Asset Management
104. Cowen
105. Creative Biogene
106. CRISPR Therapeutics
107. Cure Sanfilippo Foundation
108. CureVac
109. Cystic Fibrosis Foundation Therapeutics (CFFT)
110. Deerfield Management Company
111. Dimension Therapeutics (acquired by Ultragenyx Pharmaceutical)
112. DNAtrix Therapeutics
113. Easton Capital
114. EcoR1 Capital
115. Editas Medicine
116. Elk OrthoBiologics
117. Emergent BioSolutions
118. Eminent Venture Capital
119. enGenes
120. Ensysce Biosciences
121. Epeius Biotechnologies
122. Errant Gene Therapeutics
123. Esteve
124. Etubics
125. Eurofins Genomics
126. Eurogentec
127. European Investment Bank (EIB)
128. Expression Therapeutics
129. ExSight Capital
130. Eyevensys
131. Federal Holding and Investment Company (SFPI-FPIM)
132. Fibrocell Science
133. Fireman Capital Partners
134. FKD Therapies
135. Flagship Pioneering (previously known as Flagship Ventures)
136. Fondación Genoma España
137. Fondation Sanfilippo Suisse
138. Fondazione Telethon and Ospedale San Raffaele
139. Forbion Capital Partners
140. Foresite Capital Management
141. FoxKiser
142. F-Prime Capital Partners (previously known as Fidelity Biosciences)
143. Franklin Advisers
144. Franklin Templeton Investments
145. Fred Hutchinson Cancer Research Center
146. Freeline Therapeutics
147. Friedreich’s Ataxia Research Alliance (FARA)
148. FUJIFILM Diosynth Biotechnologies
149. Fund+
150. Fundación Sanfilippo B
151. Fundación Stop Sanfilippo
152. Further Lane Securities
153. Gene Medicine Japan (GMJ) / Kobe Biomedical Accelerator
154. GeneCure Biotechnologies
155. GeneDetect
156. GeneImmune Biotechnology
157. Genelux
158. Genentech
159. GeneOne Life Science
160. Genethon
161. Genexine
162. GenIbet Biopharmaceuticals
163. Genprex
164. GenScript Biotech
165. GenSight Biologics
166. GenVec
167. GlaxoSmithKline
168. Global BioTherapeutics
169. GlobeImmune
170. GO Capital Amorçage
171. Google Ventures (GV)
172. Gradalis®
173. Green Cross Holdings
174. Guangzhou Double Bio-products
175. Harvard University
176. HealthCap
177. Hemera Biosciences
178. Herantis Pharma
179. Hercules Capital
180. Hoffmann-La Roche
181. HORAMA
182. Horizon Discovery
183. Horizon Pharma
184. Huapont Life Sciences
185. Human Stem Cells Institute
186. I2BF Global Ventures
187. Ichor Therapeutics
188. Ichor Systems
189. ID Pharma
190. Idinvest Partners
191. Immune Technology
192. Imperial Innovations
193. Inception Capital Management
194. Index Ventures
195. Innovation Network Corporation of Japan (INCJ)
196. Innovative Genomics Institute (IGI)
197. Inovio Pharmaceuticals
198. Inserm
199. Intellia Therapeutics
200. Intrexon
201. Invesco Perpetual
202. InvivoGen
203. Ion Channel Innovations
204. Iowa State University
205. Israel Innovation Authority
206. Janssen
207. Janus Capital Management
208. Jennison Associates
209. Johns Hopkins University
210. Johnson & Johnson Development Corporation
211. JumpStart Ventures
212. Juventas Therapeutics
213. Kaneka Eurogentec
214. Khosla Ventures
215. Kiwoom Securities
216. Knight Therapeutics
217. KOLON LIFE SCIENCE
218. Krystal Group
219. Kubota Pharmaceutical
220. Kurma Partners
221. Leerink Transformation Partners
222. Leland Stanford Junior University
223. Lentigen Technology (wholly owned subsidiary of Miltenyi Biotec)
224. Leukon Investments
225. Lime Asset Management
226. Lonza
227. Louisiana State University (LSU)
228. Luminous BioSciences
229. Lundbeckfonden Ventures
230. Lysogene
231. Marsala Biotech
232. Maryland Technology Development Corporation (TEDCO)
233. Massachusetts General Hospital
234. Massachusetts Institute of Technology
235. MassBiologics
236. Medarex
237. Medigen Biotechnology
238. MedImmune
239. Medtronic
240. MeiraGTx
241. Mercia Technologies
242. Merck
243. Mercury Fund
244. Meridian Life Science
245. MERITZ Securities
246. MidCap Financial
247. Milo Biotechnology
248. Miltenyi Biotec
249. Mita Securities
250. Mitsubishi Tanabe Pharma
251. Mitsubishi UFJ Capital
252. Moderna Therapeutics
253. Molecular Diagnostic Services(MDS)
254. MolMed
255. MOLOGEN
256. Momotaro-Gene
257. Morningside Group
258. Movember Foundation
259. Myonexus Therapeutics
260. NanoCor Therapeutics
261. NanoDimension Venture Firm
262. NantCell
263. Nantes Gene Therapy Institute
264. National Cancer Institute (NCI)
265. National Eye Institute (NEI)
266. National Institute of Allergy and Infectious Diseases (NIAID)
267. National Institute on Drug Abuse (NIDA)
268. National Institutes of Health (NIH)
269. National MPS Society
270. National Securities Corporation (NSC)
271. Nature Technology
272. Neuralgene
273. Neurotech Pharmaceuticals
274. New Energy and Industrial Technology Development Organization (NEDO)
275. New Enterprise Associates (NEA)
276. New Leaf Venture Partners
277. New River Management
278. NewVa Capital Partners
279. Nightstar Therapeutics
280. Nohla therapeutics
281. Novartis
282. Novartis Venture Fund
283. Novasep
284. Novo Seeds
285. Oberland Capital
286. Okairos
287. Omega Fund Management
288. Omnes Capital
289. Omnia Biologics
290. Oncolys BioPharma
291. OncoSec Medical
292. OrbiMed Advisors
293. ORCA Therapeutics
294. Orchard Therapeutics
295. ORI Capital
296. Osage University Partners
297. Oxford BioMedica
298. Oxford Finance
299. Oxford Genetics
300. PacificGMP (a part of Abzena Group)
301. Pangu BioPharma
302. Panmure Gordon
303. Paragon Bioservices
304. Partners Innovation Fund
305. Pavilion Capital Partners
306. PeriphaGen
307. Pfizer
308. PhaseRx
309. PlasmidFactory
310. PNP Therapeutics®
311. Polaris Partners
312. Pontifax
313. Posco Capital Partners
314. Poseida Therapeutics
315. Precision BioSciences
316. Profectus BioSciences
317. Promethera Biosciences
318. PsiOxus Therapeutics
319. Puresyn
320. Quethera
321. RA Capital Management®
322. RBV Capital
323. Red Sanfilippo Foundation
324. Redmile Group
325. Regeneron Pharmaceuticals
326. REGENXBIO
327. Relieve Genetics
328. Renova Therapeutics
329. RetroSense Therapeutics (acquired by Allergan)
330. Rev1 Ventures (previously known as TechColumbus)
331. REYON Pharmaceutical
332. Richter-Helm BioLogics
333. Ridgeback Capital Management
334. Roche
335. Roche Venture Fund
336. Rock Springs Capital Management
337. Rocket Pharma
338. RTW Investments
339. Rusnano
340. SAB Technology
341. Sabby Capital
342. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET)
343. SanBio
344. Sanfilippo Children’s Foundation
345. Sanfilippo Children's Research Foundation (SCRF)
346. Sanfilippo Research Foundation (SRF)
347. Sangamo Therapeutics
348. Sanofi
349. Sanofi Genzyme
350. Sanofi-Genzyme BioVentures
351. Santen Pharmaceutical
352. Sarepta Therapeutics
353. Scancell
354. Sectoral Asset Management
355. Selecta Biosciences
356. Seventure Partners' Health for Life Capital fund
357. Sham Innovation Santé
358. Shanghai Sunway Biotech
359. Shenzhen Qianhai Taxus Industry Capital Management
360. Shenzhen Sibiono GeneTech
361. Shinhan Capital
362. Shinsei Corporate Investment
363. Shire
364. Sigma-Aldrich (now a part of Merck)
365. SignaGen Laboratories
366. Sino Biological
367. SIRION Biotech
368. Skolkovo Foundation
369. SMS Investments
370. Sofinnova Ventures
371. Song Hong Fang
372. Spark Therapeutics
373. Sphera Global Health Care
374. SR One (investment arm of GlaxoSmithKline)
375. Stratophase
376. StrideBio
377. SV Life Sciences Investors
378. Syncona Partners (a subsidiary of the Wellcome Trust)
379. Synpromics
380. Synthace
381. System Biosciences
382. T. Rowe Price
383. Takeda Pharmaceutical
384. Target ALS Foundation
385. Targeted Technology Fund
386. Targovax
387. Team Sanfilippo
388. Tekes
389. Temasek Holdings
390. Tethys Ventures
391. The Finnish Funding Agency for Technology and Innovation
392. The French Muscular Dystrophy Association (AFM-Téléthon)
393. The Jagen Group
394. The Scripps Research Institute
395. TheraBiologics
396. TheraVectys
397. Thermo Fisher Scientific
398. Third Rock Ventures
399. Third Security
400. TissueGene
401. Tocagen
402. Transgene
403. Tufts Financial Group (TFG)
404. Turenne Santé
405. TVM Capital
406. Two Blades Foundation
407. UB Securities
408. UCL Technology Fund
409. UK Innovation & Science Seed Fund (UKI2S, previously known as The Rainbow Seed Fund)
410. uniQure
411. University of California
412. University of Florida
413. University of Minnesota
414. University of Pennsylvania
415. University of Texas
416. University of Washington
417. Valneva
418. Vascular Biologics (also known as VBL Therapeutics)
419. VCN Biosciences
420. Vectalys
421. Vector Biolabs
422. Vecura (Karolinska University Hospital)
423. Venrock
424. Versant Ventures
425. Vertex Pharmaceuticals
426. VESSL (previously known as MultiGene Vascular Systems )
427. Vibalogics
428. Vical
429. Vigene Biosciences
430. Viking Global Investors
431. Virginia Biosciences Health Research Corporation (VBHRC)
432. ViroMed
433. Virovek
434. Virttu Biologics (acquired by Sorrento Therapeutics)
435. VIVEbiotech
436. Vivet Therapeutics
437. Vivo Capital
438. Voyager Therapeutics
439. Waisman Biomanufacturing
440. Wellcome Trust
441. Wellington Management Company
442. Wellstat Ophthalmics
443. Whitesun Healthcare Ventures
444. WI Harper Group
445. Woodford Investment Management
446. WuXi AppTec
447. XyloCor Therapeutics
448. Ysios Capital
449. Yuhan
450. ZIOPHARM Oncology


1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapy
3.3.2. Ex Vivo and In Vivo Gene Therapy
3.4. Routes of Administration
3.5. Mechanism of Action of Gene Therapies
3.6. Advantages and Disadvantages of Gene Therapies
3.7. Ethical and Social Concerns Related to Gene Therapies
3.8. Future Constraints and Challenges
3.8.1. Issues Related to Manufacturing
3.8.2. Issues Related to Reimbursement
3.8.3. Issues Related to Commercial Viability
3.9. Regulatory Guidelines
3.9.1. The US Scenario
3.9.2. European Scenario
3.9.3. Chinese Scenario
4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral Vectors
4.3. Types of Viral Vectors
4.3.1. Adeno-associated Viral Vectors
4.3.1.1. Overview
4.3.1.2. Design
4.3.1.3. Advantages
4.3.1.4. Limitations
4.3.2. Adenoviral Vectors
4.3.2.1. Overview
4.3.2.2. Design
4.3.2.3. Advantages
4.3.2.4. Limitations
4.3.3. Lentiviral Vectors
4.3.3.1. Overview
4.3.3.2. Design
4.3.3.3. Advantages
4.3.3.4. Limitations
4.3.4. Retroviral Vectors
4.3.4.1. Overview
4.3.4.2. Design
4.3.4.3. Advantages
4.3.4.4. Limitations
4.3.5. Other Viral Vectors
4.3.5.1. Alphavirus
4.3.5.2. Herpes Simplex Virus
4.3.5.3. Simian Virus
4.3.5.4. Vaccinia Virus
4.4. Non-Viral Vectors
4.4.1. Types of Non-Viral Vectors
4.4.1.1. Plasmid DNA
4.4.1.2. Liposomes, Lipoplexes and Polyplexes
4.4.1.3. Oligonucleotides
4.5. Methods of Transfection
4.5.1. Biolistic Methods: Gene Gun
4.5.2. Electroporation
4.5.3. Receptor Mediated Gene Delivery Methods
4.5.4. Gene Activated Matrix (GAM)
5. VECTOR MANUFACTURING
5.1. Chapter Overview
5.2. Manufacturing Viral Vectors
5.3. Viral Vector Manufacturing Processes
5.3.1. Mode of Vector Production
5.3.2. Adherent Cells Versus Suspension Cells
5.3.3. Unit Process Versus Multiple Parallel Processes
5.3.4. Cell Culture Systems for Production of Different Viral Vectors
5.4. Serum-Containing Versus Serum-Free Media
5.5. Bioprocessing of Viral Vectors
5.5.1. AAV Vector Production
5.5.2. Adenoviral Vector Production
5.5.3. Lentiviral Vector Production
5.5.4. γ -Retroviral Vector Production
5.6. Challenges Related to Vector Manufacturing
5.7. Companies Offering Contract Services for Viral and Plasmid Vectors
5.8. Partnerships
5.9. Recent Developments
6. COMPETITIVE LANDSCAPE
6.1. Chapter Overview
6.2. Gene Therapy Market: Clinical Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Indication
6.2.4. Analysis by Vector Type
6.2.5. Analysis by Gene Type
6.2.6. Analysis by Type of Modification
6.2.7. Analysis by Type of Gene Therapy (In Vivo / Ex Vivo)
6.3. Gene Therapy Market: Preclinical / Discovery Pipeline
6.3.1. Analysis by Therapeutic Area
6.3.2. Analysis by Indication
6.3.3. Analysis by Vector Type
6.3.4. Analysis by Gene Type
6.3.5. Analysis by Type of Modification
6.3.6. Analysis by Type of Gene Therapy (In Vivo / Ex Vivo)
6.4. Gene Therapy: Key Players
6.5. Gene Therapy: Prominent Hubs
6.6. Gene Therapy: Regional Landscape
7. MARKETED GENE THERAPIES
7.1. Chapter Overview
7.2. Gendicine® (Shenzhen Sibiono GeneTech)
7.2.1. Company Overview
7.2.2. History of development and Approval
7.2.3. Mechanism of Action and Vectors
7.2.4. Target Indication(s)
7.2.5. Current Status of Development
7.2.6. Manufacturing, Dosage and Sales
7.3. Oncorine® (Shanghai Sunway Biotech)
7.3.1. Company Overview
7.3.2. History of Development and Approval
7.3.3. Mechanism of Action and Vectors
7.3.4. Target Indication(s)
7.3.5. Current Status of Development
7.3.6. Dosage and Sales
7.4. Rexin-G® (Epeius Biotechnologies)
7.4.1. Company Overview
7.4.2. History of Development and Approval
7.4.3. Mechanism of Action and Vector
7.4.4. Target Indication(s)
7.4.5. Current Status of Development
7.4.6. Manufacturing, Dosage and Sales
7.5. Neovasculgen® (Human Stem Cells Institute)
7.5.1. Company Overview
7.5.2. History of Development and Approval
7.5.3. Mechanism OF Action and Vectors
7.5.4. Target Indication(s)
7.5.5. Current Status of Development
7.5.6. Manufacturing, Dosage and Sales
7.6. Imlygic® (Amgen)
7.6.1. Company Overview
7.6.2. History of Development and Approval
7.6.3. Mechanism of Action and Vectors
7.6.4. Target Indication(s)
7.6.5. Current Status of Development
7.6.6. Manufacturing, Dosage and Sales
7.7. Strimvelis® (GSK)
7.7.1. Company Overview
7.7.2. History of Development and Approval
7.7.3. Mechanism of Action and Vectors
7.7.4. Target Indication(s)
7.7.5. Current Status of Development
7.7.6. Manufacturing, Dosage and Sales
7.8. Invossa™ (TissueGene)
7.8.1. Overview of Company and Development Pipeline
7.8.2. History of Development and Approval
7.8.3. Mechanism of action and vectors
7.8.4. Target Indication(s)
7.8.5. Current Status of Development
7.8.6. Manufacturing, Dosage and Sales
7.9. Luxturna™ (Spark Therapeutics)
7.9.1. Company Overview
7.9.2. History of Development and Approval
7.9.3. Mechanism of Action and Vector
7.9.4. Target Indication(s)
7.9.5. Current Status of Development
7.9.6. Manufacturing, Dosage and Sales
8. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
8.1. Chapter Overview
8.2. Axalimogene Filolisbac: Overview of Therapy, Key Developments and Clinical Results
8.3. AVXS-101: Overview of Therapy, Key Developments and Clinical Results
8.4. Collategene® (Beperminogene Perplasmid): Overview of Therapy, Key Developments and Clinical Trials
8.5. Donaperminogene Seltoplasmid (VM202): Overview of Therapy, Key Developments and Clinical Results
8.6. E10A: Overview of Therapy, Key Developments and Clinical Trials
8.7. GS010: Overview of Therapy, Key Developments and Clinical Trials
8.8. GSK2696274: Overview of Therapy, Key Developments and Clinical Results
8.9. GSK2696275: Overview of Therapy, Key Developments and Clinical Results
8.10. ImmunoPulse®: Overview of Therapy, Key Developments and Clinical Results
8.11. Instiladrin®: Overview of Therapy, Key Developments and Clinical Results
8.12. Lenti-D™: Overview of Therapy, Key Developments and Clinical Trials
8.13. LentiGlobin® BB305: Overview of Therapy, Key Developments and Clinical Trials
8.14. Ofranergene Obadenovec (VB-111): Overview of Therapy, Key Developments and Clinical Results
8.15. OTL-101: Overview of Therapy, Key Developments and Clinical Results
8.16. Pexastimogene Devacirepvec (PEXA-VEC): Overview of Therapy, Key Developments and Clinical Results
8.17. ProstAtak®: Overview of Therapy, Key Developments and Clinical Trials
8.18. Valoctocogene Roxaparvovec (BMN 270): Overview of Therapy, Key Developments and Clinical Results
8.19. Vigil®: Overview of Therapy, Key Developments and Clinical Results
8.20. VGX-3100: Overview of Therapy, Key Developments and Clinical Results
8.21. Vocimagene Amiretrorepvec (Toca-511): Overview of Therapy, Key Developments and Clinical Results
9. EMERGING TECHNOLOGIES
9.1. Chapter Overview
9.2. Genome Editing Technologies
9.2.1. Overview
9.2.2. Applications
9.3. Emerging Genome Editing Platforms
9.3.1. CRISPR / Cas9 System
9.3.2. TALENs
9.3.3. megaTAL
9.3.4. Zinc Finger Nuclease
9.4. Gene Expression Regulation Technologies
9.5. Technology Platforms for Developing / Delivering Gene Therapy
9.5.1. AGTC’s Proprietary Technology
9.5.2. Gene Mediated Cytotoxic Immunotherapy Technology
9.5.3. in situ Delivery and Production System (i-DPS)
9.5.4. LentiVector® platform
9.5.5. NAV® Technology Platform
9.5.6. uniQure’s Proprietary Delivery Technology
10. PROMISING THERAPEUTICS AREAS
10.1. Chapter Overview
10.2. Cardiovascular Diseases
10.3. Hematological Disorders
10.4. Inflammatory & Infectious (I&I) Diseases
10.5. Metabolic Disorders
10.6. Muscular Disorders
10.7. Neurological Disorders
10.8. Ocular Diseases
10.9. Oncology
11. FUNDING AND INVESTMENT ANALYSIS
11.1. Chapter Overview
11.2. Types of Funding
11.3. Gene Therapy: Funding and Investment Analysis
11.3.1. Analysis by Number of Funding Instances
11.3.2. Analysis by Amount Invested
11.3.3. Analysis by Type of Funding
11.3.4. Analysis by Most Active Players
11.3.5. Analysis by Key Investors
12. COST PRICE ANALYSIS
12.1. Chapter Overview
12.2. Gene Therapy Market: Factors Contributing to Price Tags
12.3. Gene Therapy Market: Pricing Models
12.3.1. On the Basis of Associated Costs
12.3.2. On the Basis of Competition
12.3.3. On the Basis of Patient Segment
12.3.4. On the Basis of Opinions of Experts in the Industry
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Scope and Methodology
13.3. Gene Therapy Patents: Distribution by Filing Year
13.4. Gene Therapy Patents: Distribution by Geographical Location of Patent Offices
13.5. Gene Therapy Patents: Distribution by CPC Classification
13.6. Gene Therapy Patents: Emerging Areas
13.7. Gene Therapy Patents: Leading Players
13.8. Gene Therapy Patents: Competitive Benchmarking
13.8.1. Analysis by Patent Characteristics
13.8.2. Analysis by Geographical Distribution
13.9. Gene Therapy Patents: Valuation Analysis
13.10. Gene Therapy Patents: Leading Citations
14. MARKET FORECAST AND OPPORTUNITY ANALYSIS
14.1. Chapter Overview
14.2. Forecast Methodology
14.3. Overall Gene Therapy Market, 2018-2030
14.3.1. Gene Therapy Market: Distribution by Therapeutic Area
14.3.2. Gene Therapy Market: Distribution by Vector Type
14.3.3. Gene Therapy Market: Distribution by Therapy Type
14.3.4. Gene Therapy Market: Distribution by Geography
14.4. Gene Therapy Market: Product-wise Sales Forecasts
14.4.1. Gendicine®
14.4.1.1. Target Patient Population
14.4.1.2. Sales Forecast
14.4.2. Oncorine®
14.4.2.1. Target Patient Population
14.4.2.2. Sales Forecast
14.4.3. Rexin-G®
14.4.3.1. Target Patient Population
14.4.3.2. Sales Forecast
14.4.4. Neovasculgen®
14.4.4.1. Target Patient Population
14.4.4.2. Sales Forecast
14.4.5. Strimvelis®
14.4.5.1. Target Patient Population
14.4.5.2. Sales Forecast
14.4.6. Imlygic®
14.4.6.1. Target Patient Population
14.4.6.2. Sales Forecast
14.4.7. Invossa™
14.4.7.1. Target Patient Population
14.4.7.2. Sales Forecast
14.4.8. Luxturna™
14.4.8.1. Target Patient Population
14.4.8.2. Sales Forecast
14.4.9. Axalimogene Filolisbac
14.4.9.1. Target Patient Population
14.4.9.2. Sales Forecast
14.4.10. AVXS-101
14.4.10.1. Target Patient Population
14.4.10.2. Sales Forecast
14.4.11. Collategene® (Beperminogene Perplasmid)
14.4.11.1. Target Patient Population
14.4.11.2. Sales Forecast
14.4.12. Donaperminogene Seltoplasmid (VM202)
14.4.12.1. Target Patient Population
14.4.12.2. Sales Forecast
14.4.13. E10A
14.4.13.1. Target Patient Population
14.4.13.2. Sales Forecast
14.4.14. GS010
14.4.14.1. Target Patient Population
14.4.14.2. Sales Forecast
14.4.15. GSK2696274
14.4.15.1. Target Patient Population
14.4.15.2. Sales Forecast
14.4.16. GSK2696275
14.4.16.1. Target Patient Population
14.4.16.2. Sales Forecast
14.4.17. ImmunoPulse®
14.4.17.1. Target Patient Population
14.4.17.2. Sales Forecast
14.4.18. Instiladrin®
14.4.18.1. Target Patient Population
14.4.18.2. Sales Forecast
14.4.19. Lenti-D™
14.4.19.1. Target Patient Population
14.4.19.2. Sales Forecast
14.4.20. LentiGlobin® BB305
14.4.20.1. Target Patient Population
14.4.20.2. Sales Forecast
14.4.21. Ofraneregene Obadenovec (VB-111)
14.4.21.1. Target Patient Population
14.4.21.2. Sales Forecast
14.4.22. OTL-101
14.4.22.1. Target Patient Population
14.4.22.2. Sales Forecast
14.4.23. Pexastimogene Devacirepvec (PEXA-VEC)
14.4.23.1. Target Patient Population
14.4.23.2. Sales Forecast
14.4.24. ProstAtak®
14.4.24.1. Target Patient Population
14.4.24.2. Sales Forecast
14.4.25. Valoctocogene Roxaparvovec (BMN 270)
14.4.25.1. Target Patient Population
14.4.25.2. Sales Forecast
14.4.26. Vigil®
14.4.26.1. Target Patient Population
14.4.26.2. Sales Forecast
14.4.27. VGX-3100
14.4.27.1. Target Patient Population
14.4.27.2. Sales Forecast
14.4.28. Vocimagene Amiretrorepvec (Toca 511, Toca FC)
14.4.28.1. Target Patient Population
14.4.28.2. Sales Forecast
15. CONCLUSION
15.1. Gene Therapy Market is Characterized by a Robust Pipeline of Candidates Being Developed Using Various Types of Vectors
15.2. Although Product Candidates are Being Developed to Treat a Number of Clinical Conditions, the Primary Focus is on Oncology
15.3. Currently, the Market is Led by Big Pharma Players; However, Many Start-ups, Supported by Both Private and Public Investors, are Spearheading the Innovation in this Domain
15.4. CMOs are Expected to Continue to Play a Key Role in Driving Manufacturing Efforts in the Industry
15.5. Given the Advancements in Technology Platforms and the Approval of Late Stage Product Candidates, the Market is poised to Grow Significantly in the Foreseen Future
16. INTERVIEW TRANSCRIPTS
16.1. Chapter Overview
16.2. Adam Rogers, CEO, Hemera Biosciences (Q1, 2018)
16.3. Al Hawkins, CEO, Milo Biotechnology (Q4 2014)
16.4. Buel Dan Rodgers, Founder & CEO, AAVogen (Q1, 2018)
16.5. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics (Q4 2017)
16.6. Christopher Reinhard, CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics) (Q4 2014)
16.7. Jeffrey HunG, COO, Vigene Biosciences (Q4 2017)
16.8. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory (Q4 2017)
16.9. Michael Tripletti, CEO, Myonexus Therapeutics (Q1 2018)
16.10. Robert Jan Lamers, CEO, Arthrogen (Q1, 2018)
17. APPENDIX 1: TABULATED DATA
18. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
LIST OF FIGURES
Figure 3.1 History of Evolution of Gene Therapies
Figure 3.2 Ex Vivo Gene Therapy
Figure 3.3 In Vivo Gene Therapy
Figure 3.4 Mechanism of Action of Gene Therapies
Figure 3.5 Advantages and Disadvantages of Gene Therapies
Figure 3.6 China: Manufacturing Requirements for Gene Therapy Products
Figure 3.7 China: Key Elements of Quality Control
Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
Figure 6.1 Viral Vectors: Manufacturing Steps
Figure 6.1 Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
Figure 6.2 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Areas
Figure 6.3 Gene Therapies, Clinical Pipeline: Distribution by Indications
Figure 6.4 Gene Therapies, Clinical Pipeline: Distribution by Vector Type
Figure 6.5 Gene Therapies, Clinical Pipeline: Distribution by Gene Type
Figure 6.6 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
Figure 6.7 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
Figure 6.8 Gene Therapies, Preclinical Pipeline: Distribution by Therapeutic Areas
Figure 6.9 Gene Therapies, Preclinical Pipeline: Distribution by Indications
Figure 6.10 Gene Therapies, Preclinical Pipeline: Distribution by Vector Type
Figure 6.11 Gene Therapies, Preclinical Pipeline: Distribution by Gene Type
Figure 6.12 Gene Therapies, Preclinical Pipeline: Distribution by Type of Modification
Figure 6.13 Gene Therapies, Preclinical Pipeline: Distribution by Type of Gene Therapy
Figure 6.14 Gene Therapies: Key Players
Figure 6.15 Gene Therapies: Prominent Hubs
Figure 6.16 Gene Therapy Developers: North America
Figure 6.17 Gene Therapy Developers: Europe
Figure 6.18 Gene Therapy Developers: Asia Pacific
Figure 7.1 Gendicine®: Development Timeline
Figure 7.2 Oncorine®: Development Timeline
Figure 7.3 Oncorine®: Mechanism of Action
Figure 7.4 Rexin-G®: Development Timeline
Figure 7.5 Neovasculgen®: Development Timeline
Figure 7.6 Imlygic®: Development Timeline
Figure 7.7 Imlygic®: Mechanism of Action
Figure 7.8 Strimvelis®: Development Timeline
Figure 7.9 Invossa™: Development Timeline
Figure 7.10 Invossa™: Mechanism of Action
Figure 7.11 Luxturna™: Development Timeline
Figure 9.1 Genome Editing Technologies: Applications
Figure 9.2 Genome Editing: Emerging Technology Platforms
Figure 10.1 Gene Therapies for Cardiovascular Disorders: Distribution by Indication and Phase of Development
Figure 10.2 Gene Therapies for Cardiovascular Diseases: Distribution by Vector Type
Figure 10.3 Gene Therapies for Hematological Disorders: Distribution by Indication and Phase of Development
Figure 10.4 Gene Therapies for Hematological Disorders: Distribution by Vector Type
Figure 10.5 Gene Therapies for I&I Disorders: Distribution by Indication and Phase of Development
Figure 10.6 Gene Therapies for I&I Disorders: Distribution by Vector Type
Figure 10.7 Gene Therapies for Metabolic Disorders: Distribution by Indication and Phase of Development
Figure 10.8 Gene Therapies for Metabolic Disorders: Distribution by Vector Type
Figure 10.9 Gene Therapies for Muscular Disorders: Distribution by Indication and Phase of Development
Figure 10.10 Gene Therapies for Muscular Disorders: Distribution by Vector Type
Figure 10.11 Gene Therapies for Neurological Disorders: Distribution by Indication and Phase of Development
Figure 10.12 Gene Therapies for Neurological Disorders: Distribution by Vector Type
Figure 10.13 Gene Therapies for Ocular Disorders: Distribution by Indication and Phase of Development
Figure 10.14 Gene Therapies for Ocular Disorders: Distribution by Type of Vector
Figure 10.15 Gene Therapies for Oncology: Distribution by Indication and Phase of Development
Figure 10.16 Gene Therapies for Oncology: Distribution by Vector Type
Figure 11.1 Gene Therapy Market: Cumulative Number of Funding Instances, Pre-2013-2018
Figure 11.2 Gene Therapy Market: Cumulative Amount Invested, Pre-2013-2018 (USD Billion)
Figure 11.3 Gene Therapy Market: Distribution by Type of Funding, Pre-2013-2018
Figure 11.4 Gene Therapy Market: Amount Invested under each Type of Funding, Pre-2013 – 2018 (USD Million)
Figure 11.5 Gene Therapy Market: Funding and Investment Summary
Figure 11.6 Gene Therapy Market: Most Active Players
Figure 11.7 Gene Therapy: Key Investors
Figure 12.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 13.1 Gene Therapy Patents: Distribution by Types of Patent
Figure 13.2 Gene Therapy Patents: Distribution (Cumulative) by Publication Year
Figure 13.3 Gene Therapy Patents: Distribution by Patent Offices
Figure 13.4 Gene Therapy Patents: Cumulative Distribution by CPC Classification Symbol
Figure 13.5 Gene Therapy Patents: Emerging Areas
Figure 13.6 Gene Therapy Patents: Leading Industry and Non-Industry Players
Figure 13.7 Gene Therapy Patents: Distribution by Industry Players
Figure 13.8 Gene Therapy Patents: Distribution by Non-Industry Players
Figure 13.9 Gene Therapy Patents: Geographical Distribution of Leading Players
Figure 13.10 Gene Therapy Patents (Industry Players): Benchmarking by Patent Characteristics
Figure 13.11 Gene Therapy Patents (Non-Industry Players): Benchmarking by Patent Characteristics
Figure 13.12 Gene Therapy Patents (Industry Players): Benchmarking by Geography
Figure 13.13 Gene Therapy Patents (Industry Players): Benchmarking by International Patents
Figure 13.14 Gene Therapy Patents (Non-Industry Players): Benchmarking by Geography
Figure 13.15 Gene Therapy (Non-Industry) Patents: Benchmarking by International Patents
Figure 13.16 Gene Therapy Patents: Distribution by Age of Patents (January 2013-October 2017)
Figure 13.17 Gene Therapy Patents: Patent Valuation
Figure 14.1 Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
Figure 14.2 Gene Therapy Market: Distribution by Therapeutic Area, 2018, 2024 and 2030
Figure 14.3 Gene Therapy Market: Distribution by Vector Type, 2018, 2024 and 2030
Figure 14.4 Gene Therapy Market: Distribution by Therapy Type, 2018, 2024 and 2030
Figure 14.5 Gene Therapy Market: Distribution by Geography, 2018, 2024 and 2030
Figure 14.6 Gene Therapy Market: Distribution by Geography (RoW), 2018, 2024 and 2030
Figure 14.7 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.8 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.9 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.10 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.11 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.12 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.13 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.14 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.15 Axalimogene filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.16 AVXS-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.17 Beperminogene Perplasmid (Collategene) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.18 Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.19 E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.20 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.21 GSK2696274 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.22 GSK2696275 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.23 ImmunoPulse® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.24 Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.25 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.26 LentiGlobin® BB305 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.27 Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.28 OTL-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.29 Pexastimogene Devacirepvec (Pexa-Vec) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.30 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.31 Valoctocogene Roxaparvovec (BMN-270) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.32 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.33 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.34 Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 15.1 Gene Therapy: Distribution of Clinical Stage Product Candidates by Type of Vector and Type of Therapy (Ex vivo, In Vivo)
Figure 15.2 Gene Therapy: Key Therapeutic Areas
Figure 15.3 Gene Therapy Market: Conservative, Base and Optimistic Forecast Scenarios, 2018, 2024 and 2030 (USD Billion) 
LIST OF TABLES
Table 3.1 Differences between Ex Vivo and In Vivo Techniques
Table 3.2 Administration Routes for Targeted Gene Delivery to Specific Organs / Tissues
Table 3.3 Vectors used for Targeted Gene Delivery to Tissues / Organs
Table 3.4 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 3.5 Prices of Marketed Gene Therapies
Table 3.6 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 4.1 Viral Vectors: Key Features
Table 5.1 Small Scale Cell Culture Systems
Table 5.2 Contract Manufacturing Services for Viral Vectors and Plasmid DNA
Table 5.3 Gene Therapy Manufacturing Partnerships
Table 6.1 Gene Therapies: Clinical Pipeline
Table 6.2 Gene Therapies: Preclinical Pipeline
Table 7.1 Gene Therapies: Marketed and Approved Products
Table 7.2 Company Overview: Shenzhen Sibiono GeneTech
Table 7.3 Gendicine®: Patent Portfolio
Table 7.4 Gendicine®: Status of Development
Table 7.5 Company Overview: Shanghai Sunway Biotech
Table 7.6 Oncorine®: Status of Development
Table 7.7 Company Overview: Epeius Biotechnologies
Table 7.8 Rexin-G®: Status of Development
Table 7.9 Company Overview: Human Stem Cell Institute
Table 7.10 Neovasculgen®: Status of Development
Table 7.11 Company Overview: Amgen
Table 7.12 Imlygic®: Status of Development
Table 7.13 Imlygic®: Recommended Dose and Schedule
Table 7.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
Table 7.15 Company Overview: GSK
Table 7.16 Strimvelis®: Status of Development
Table 7.17 Company Overview: TissueGene
Table 7.18 Invossa™: Status of Development
Table 7.19 Company Overview: Spark Therapeutics
Table 7.20 Luxturna™: Status of Development
Table 8.1 Gene Therapies: Phase III Drugs
Table 8.2 Drug Profile: Axalimogene filolisbac
Table 8.3 Drug Profile: AVXS-101
Table 8.4 Drug Profile: Collategene® (Beperminogene Perplasmid)
Table 8.5 Drug Profile: Donaperminogene Seltoplasmid (VM202)
Table 8.6 Drug Profile: E10A
Table 8.7 Drug Profile: GS-010
Table 8.8 Drug Profile: GSK2696274
Table 8.9 Drug Profile: GSK2696275
Table 8.10 Drug Profile: ImmunoPulse®
Table 8.11 Drug Profile: Instiladrin®
Table 8.12 Drug Profile: Lenti-D™
Table 8.13 Drug Profile: LentiGlobin® BB305
Table 8.14 Drug Profile: Ofranergene Obadenovec (VB-111)
Table 8.15 Drug Profile: OTL-101
Table 8.16 Drug Profile: Pexastimogene Devacirepvec (PEXA-VEC)
Table 8.17 Drug Profile: ProstAtak®
Table 8.18 Drug Profile: Valoctocogene roxaparvovec (BMN 270)
Table 8.19 Drug Profile: Vigil®
Table 8.20 Drug Profile: VGX-3100
Table 8.21 Drug Profile: Vocimagene Amiretrorepvec (Toca-511)
Table 9.1 Gene Editing Technology Platforms
Table 9.2 Editas Medicine: CRISPR / Cas9 Technology, Research Publications
Table 9.3 Editas Medicine: Funding Instances
Table 9.4 Editas Medicine: Collaborations
Table 9.5 Intellia Therapeutics: CRISPR / Cas9 Technology, Research Publications
Table 9.6 Intellia Therapeutics: Funding Instances
Table 9.7 Intellia Therapeutics: Collaborations
Table 9.8 CRISPR Therapeutics: CRIPSR / Cas9 Technology, Research Publications
Table 9.9 CRISPR Therapeutics: Funding Instances
Table 9.10 CRISPR Therapeutics: Collaborations
Table 9.11 bluebird bio: megaTAL Technology, Research Publications
Table 9.12 bluebird bio: Funding Instances
Table 9.13 bluebird bio: Collaborations
Table 9.14 Cellectis: Funding Instances
Table 9.15 Cellectis: Collaborations
Table 9.16 Sangamo Therapeutics: Funding Instances
Table 9.17 Sangamo Therapeutics: Collaborations
Table 9.18 Gene Switch Technology Platforms
Table 9.19 Intrexon: Funding Instances
Table 9.20 Intrexon: Collaborations
Table 9.21 MeiraGTx: Funding Instances
Table 9.22 MeiraGTx: Collaborations
Table 9.23 Gene Therapy: Technology Platforms
Table 10.1 Gene Therapies for Cardiovascular Disorders Cardiovascular Disorders
Table 10.2 Gene Therapies for Hematological Disorders
Table 10.3 Gene Therapies for I&I Disorders
Table 10.4 Gene Therapies for Metabolic Disorders
Table 10.5 Gene Therapies for Muscular Disorders
Table 10.6 Gene Therapies for Neurological Disorders
Table 10.7 Gene Therapies for Ocular Disorders
Table 10.8 Gene Therapies for Oncology
Table 11.1 Gene Therapy Market: Funding and Investment Analysis
Table 11.2 Gene Therapy: Summary of Investments
Table 12.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 12.2 Pricing Model: Price of Marketed Targeted Drugs
Table 12.3 Pricing Model: Opinions of Experts / Other Analysts
Table 13.1 Gene Therapy Patents: CPC Symbol Definitions
Table 13.2 Gene Therapy Patents: Most Popular CPC Symbols
Table 12.3 Gene Therapy Patents: List of Top CPC Classifications
Table 13.4 Gene Therapy Patents: List of Top Leading Citations
Table 14.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 14.2 Gendicine®: Target Patient Population
Table 14.3 Oncorine®: Target Patient Population
Table 14.4 Rexin-G®: Target Patient Population
Table 14.5 Neovasculgen®: Target Patient Population
Table 14.6 Strimvelis®: Target Patient Population
Table 14.7 Imlygic®: Target Patient Population
Table 14.8 Invossa™: Target Patient Population
Table 14.9 Luxturna™: Target Patient Population
Table 14.10 Axalimogene Filolisbac: Target Patient Population
Table 14.11 AVXS-101: Target Patient Population
Table 14.12 Beperminogene Perplasmid (Collategene): Target Patient Population
Table 14.13 Donaperminogene Seltoplasmid (VM202): Target Patient Population
Table 14.14 E10A: Target Patient Population
Table 14.15 GS010: Target Patient Population
Table 14.16 GSK2696274: Target Patient Population
Table 14.17 GSK2696275: Target Patient Population
Table 14.18 ImmunoPulse®: Target Patient Population
Table 14.19 Instiladrin®: Target Patient Population
Table 14.20 Lenti-D™: Target Patient Population
Table 14.21 LentiGlobin® BB305: Target Patient Population
Table 14.22 Ofraneregene Obadenovec (VB-111): Target Patient Population
Table 14.23 OTL-101: Target Patient Population
Table 14.24 Pexastimogene Devacirepvec (Pexa-Vec): Target Patient Population
Table 14.25 ProstAtak®: Target Patient Population
Table 14.26 Valoctocogene Roxaparvovec (BMN-270): Target Patient Population
Table 14.27 Vigil®: Target Patient Population
Table 14.28 VGX-3100: Target Patient Population
Table 14.29 Vocimagene Amiretrorepvec (Toca-511): Target Patient Population
Table 17.1 Gene Therapy, Clinical Pipeline: Distribution by Phase of Development
Table 17.2 Gene Therapy, Clinical Pipeline: Distribution by Therapeutic Areas
Table 17.3 Gene Therapy, Clinical Pipeline: Distribution by Indications
Table 17.4 Gene Therapy, Clinical Pipeline: Distribution by Vector Type
Table 17.5 Gene Therapy, Clinical Pipeline: Distribution by Gene Type
Table 17.6 Gene Therapy, Clinical Pipeline: Distribution by Type of Modification
Table 17.7 Gene Therapy, Clinical Pipeline: Distribution by Type of Gene Therapy
Table 17.8 Gene Therapy, Preclinical Pipeline: Distribution by Therapeutic Areas
Table 17.9 Gene Therapy, Preclinical Pipeline: Distribution by Indications
Table 17.10 Gene Therapy, Preclinical Pipeline: Distribution by Vector Type
Table 17.11 Gene Therapy, Preclinical Pipeline: Distribution by Gene Type
Table 17.12 Gene Therapy, Preclinical Pipeline: Distribution by Type of Modification
Table 17.13 Gene Therapy, Preclinical Pipeline: Distribution by Type of Gene Therapy
Table 17.14 Gene Therapy: Key Players
Table 17.15 Gene Therapies for Cardiovascular Disorders: Distribution by Indication and Phase of Development
Table 17.16 Gene Therapies for Cardiovascular Disorders: Distribution by Vector Type
Table 17.17 Gene Therapies for Hematological Disorders: Distribution by Indication and Phase of Development
Table 17.18 Gene Therapies for Hematological Disorders: Distribution by Vector Type
Table 17.19 Gene Therapies for I&I Disorders: Distribution by Indication and Phase of Development
Table 17.20 Gene Therapies for I&I Disorders: Distribution by Vector Type
Table 17.21 Gene Therapies for Metabolic Disorders: Distribution by Indication and Phase of Development
Table 17.22 Gene Therapies for Metabolic Disorders: Distribution by Vector Type
Table 17.23 Gene Therapies for Muscular Disorders: Distribution by Indication
Table 17.24 Gene Therapies for Muscular Disorders: Distribution by Vector Type
Table 17.25 Gene Therapies for Neurological Disorders: Distribution by Indication and Phase of Development
Table 17.26 Gene Therapies for Neurological Disorders: Distribution by Vector Type
Table 17.27 Gene Therapies for Ocular Disorders: Distribution by Indication and Phase of Development
Table 17.28 Gene Therapies for Ocular Disorders: Distribution by Type of Vector
Table 17.29 Gene Therapies for Oncology: Distribution by Indication and Phase of Development
Table 17.30 Gene Therapies for Oncology: Distribution by Vector Type
Table 17.31 Gene Therapy Patents: Distribution by Types of Patent
Table 17.32 Gene Therapy Market: Cumulative Amount Invested, Pre-2013-2018 (USD Billion)
Table 17.33 Gene Therapy Market: Distribution by Type of Funding, Pre-2013-2018 (USD Billion)
Table 17.34 Gene Therapy Market: Amount Invested under each Type of Funding, Pre-2013-2018 (USD Million)
Table 17.35 Gene Therapy Market: Amount Invested under Venture Funding, Pre-2013 – 2018 (USD Million)
Table 17.36 Gene Therapy Market: Most Active Players
Table 17.37 Gene Therapy: Key Investors
Table 17.38 Gene Therapy Patents: Distribution (Cumulative) by Publication Year
Table 17.39 Gene Therapy Patents: Distribution by Patent Offices
Table 17.40 Gene Therapy Patents: Distribution by Industry Players
Table 17.41 Gene Therapy Patents: Distribution by Non-Industry Players
Table 17.42 Gene Therapy Patents (Industry Players): Benchmarking by Patent Characteristics
Table 17.43 Gene Therapy Patents (Non-Industry Players): Benchmarking by Patent Characteristics
Table 17.44 Gene Therapy Patents: Distribution by Age of Patents
Table 17.45 Overall Gene Therapy Market: Conservative, Base and Optimistic Scenario, 2018-2030 (USD Million)
Table 17.46 Gene Therapy Market: Distribution by Vector Type, 2018, 2024 and 2030
Table 17.47 Gene Therapy Market: Distribution by Therapy Type, 2018, 2024 and 2030
Table 17.48 Gene Therapy Market: Distribution by Geography, 2018, 2024 and 2030
Table 17.49 Gene Therapy Market: Distribution by Geography (RoW), 2018, 2024 and 2030
Table 17.50 Genedicine® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.51 Oncorine® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.52 Rexin-G® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.53 Neovasculgen® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.54 Strimvelis® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.55 Imlygic® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.56 Invossa™ Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.57 Luxturna™ Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.58 Axalimogene Filolisbac Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.59 AVXS-101 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.60 Beperminogene Perplasmid (Collategene®) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.61 Donaperminogene Seltoplasmid (VM202) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.62 E10A Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.63 GS010 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.64 GSK2696274 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.65 GSK2696275 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.66 ImmunoPulse® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.67 Instiladrin® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.68 Lenti-D™ Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.69 LentiGlobin® BB305 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.70 Ofranergene Obadenovec (VB-111) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.71 OTL-101 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.72 Pexastimogene Devacirepvec (PEXA-VEC) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.73 ProstAtak® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.74 Valoctocogene Roxaparvovec (BMN-270) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.75 Vigil® Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.76 VGX-3100 Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.77 Vocimagene Amiretrorepvec (Toca-511) Market (Till 2030): Conservative, Base and Optimistic Scenario (USD Million)
Table 17.78 Overall Gene Therapy Market: Conservative, Base and Optimistic Scenario, 2018, 2024, 2030 (USD Billion)

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