Market Overview

Gene Therapy Market, 2018-2030 - Around 300 Product Candidates Under Various Stages of Development for a Diverse Range of Indications

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Dublin, Aug. 13, 2018 (GLOBE NEWSWIRE) -- The "Gene Therapy Market (2nd Edition), 2018-2030" report has been added to ResearchAndMarkets.com's offering.

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.

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), 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) and Michael Triplett (CEO, Myonexus Therapeutics). 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.

Companies Mentioned

  • 4BIO Capital
  • 5AM Ventures
  • Abbott
  • Abby Grace Foundation
  • Abeona Therapeutics
  • Abingworth
  • ABL Europe
  • Ablynx
  • Abraxis BioScience
  • Acceleron Pharma
  • Acucela (Subsidiary of Kubota Pharmaceutical)
  • Adage Capital Management
  • Aduro Biotech
  • Advanced BioScience Laboratories (ABL)
  • Advanced Biotherapeutics Consulting
  • Advantagene
  • Advaxis Immunotherapies
  • Adverum Biotechnologies
  • Agent Capital
  • Agilis Biotherapeutics
  • Ajinomoto Althea
  • AJU IB Investment
  • Aldevron
  • Alexandria Venture Investments
  • Allele Biotechnology and Pharmaceuticals
  • Ally Bridge Group (ABG)
  • Alpha-1 Foundation
  • AlphaVax
  • Amgen
  • AMSBIO
  • Anaeropharma Science
  • Anemocyte (previously known as Areta International)
  • AnGes
  • Angionetics
  • Apic Bio
  • Applied Genetic Technologies Corporation (AGTC)
  • Applied Viromics
  • ARCH Venture Partners
  • Arthrogen
  • AstraZeneca
  • Athena Vision
  • Atlas Venture
  • aTyr Pharma
  • Audentes Therapeutics
  • AveXis
  • AVROBIO
  • Baillie Gifford
  • Bamboo Therapeutics (acquired by Pfizer)
  • Batavia Biosciences
  • Bayer
  • Beckman Research Institute
  • Beijing Capital Grand (previously known as Beijing Capital Juda)
  • Benitec Biopharma
  • BioCanCell Therapeutic
  • Biogen
  • BioMarin Pharmaceutical
  • Biomay
  • Biomiga
  • BioMotiv
  • BioNTech Innovative Manufacturing Services (previously known as EUFETS)
  • BioReliance / SAFC
  • Bioverativ
  • Biovian
  • BioViva
  • Blue Sky BioServices
  • bluebird bio
  • BlueWater Angels
  • Boehringer Ingelheim BioXcellence
  • Boehringer Ingelheim Venture Fund
  • Bpifrance
  • Brammer Bio
  • Bristol-Myers Squibb
  • Broad Institute
  • Brookside Capital Partners Fund
  • California Institute for Regenerative Medicine (CIRM)
  • Cambridge Enterprise
  • Cancer Prevention and Research Institute of Texas (CPRIT)
  • CapDecisif Management
  • Caribou Biosciences
  • Casdin Capital
  • Celgene
  • Cell and Gene Therapy Catapult
  • Cellectis
  • CellGenTech
  • Celsion
  • Center for the Development of Industrial Technology (CDTI)
  • Centre for Process Innovation
  • Centre national de la recherche scientifique (CNRS)
  • CEVEC Pharmaceuticals
  • CHDI Foundation
  • Chiesi Farmaceutici
  • Children's Medical Research Foundation
  • Children's Hospital of Philadelphia
  • Children's Medical Research Foundation
  • Clarus Ventures
  • Clough Capital Partners
  • Cobra Biologics and Pharmaceutical Services
  • Cold Genesys
  • Columbus Venture Partners
  • Commercial & External Partnership, Industrial Affairs (CEPiA)
  • Copernicus Therapeutics
  • Cormorant Asset Management
  • Cowen
  • Creative Biogene
  • CRISPR Therapeutics
  • Cure Sanfilippo Foundation
  • CureVac
  • Cystic Fibrosis Foundation Therapeutics (CFFT)
  • Deerfield Management Company
  • Dimension Therapeutics (acquired by Ultragenyx Pharmaceutical)
  • DNAtrix Therapeutics
  • Easton Capital
  • EcoR1 Capital
  • Editas Medicine
  • Elk OrthoBiologics
  • Emergent BioSolutions
  • Eminent Venture Capital
  • enGenes
  • Ensysce Biosciences
  • Epeius Biotechnologies
  • Errant Gene Therapeutics
  • Esteve
  • Etubics
  • Eurofins Genomics
  • Eurogentec
  • European Investment Bank (EIB)
  • Expression Therapeutics
  • ExSight Capital
  • Eyevensys
  • Federal Holding and Investment Company (SFPI-FPIM)
  • Fibrocell Science
  • Fireman Capital Partners
  • FKD Therapies
  • Flagship Pioneering (previously known as Flagship Ventures)
  • Fondacin Genoma Espaa
  • Fondation Sanfilippo Suisse
  • Fondazione Telethon and Ospedale San Raffaele
  • Forbion Capital Partners
  • Foresite Capital Management
  • FoxKiser
  • F-Prime Capital Partners (previously known as Fidelity Biosciences)
  • Franklin Advisers
  • Franklin Templeton Investments
  • Fred Hutchinson Cancer Research Center
  • Freeline Therapeutics
  • Friedreich's Ataxia Research Alliance (FARA)
  • FUJIFILM Diosynth Biotechnologies
  • Fund+
  • Fundacin Sanfilippo B
  • Fundacin Stop Sanfilippo
  • Further Lane Securities
  • Gene Medicine Japan (GMJ) / Kobe Biomedical Accelerator
  • GeneCure Biotechnologies
  • GeneDetect
  • GeneImmune Biotechnology
  • Genelux
  • Genentech
  • GeneOne Life Science
  • Genethon
  • Genexine
  • GenIbet Biopharmaceuticals
  • Genprex
  • GenScript Biotech
  • GenSight Biologics
  • GenVec
  • GlaxoSmithKline
  • Global BioTherapeutics
  • GlobeImmune
  • GO Capital Amorage
  • Google Ventures (GV)
  • Gradalis
  • Green Cross Holdings
  • Guangzhou Double Bio-products
  • Harvard University
  • HealthCap
  • Hemera Biosciences
  • Herantis Pharma
  • Hercules Capital
  • Hoffmann-La Roche
  • HORAMA
  • Horizon Discovery
  • Horizon Pharma
  • Huapont Life Sciences
  • Human Stem Cells Institute
  • I2BF Global Ventures
  • Ichor Therapeutics
  • Ichor Systems
  • ID Pharma
  • Idinvest Partners
  • Immune Technology
  • Imperial Innovations
  • Inception Capital Management
  • Index Ventures
  • Innovation Network Corporation of Japan (INCJ)
  • Innovative Genomics Institute (IGI)
  • Inovio Pharmaceuticals
  • Inserm
  • Intellia Therapeutics
  • Intrexon
  • Invesco Perpetual
  • InvivoGen
  • Ion Channel Innovations
  • Iowa State University
  • Israel Innovation Authority
  • Janssen
  • Janus Capital Management
  • Jennison Associates
  • Johns Hopkins University
  • Johnson & Johnson Development Corporation
  • JumpStart Ventures
  • Juventas Therapeutics
  • Kaneka Eurogentec
  • Khosla Ventures
  • Kiwoom Securities
  • Knight Therapeutics
  • KOLON LIFE SCIENCE
  • Krystal Group
  • Kubota Pharmaceutical
  • Kurma Partners
  • Leerink Transformation Partners
  • Leland Stanford Junior University
  • Lentigen Technology (wholly owned subsidiary of Miltenyi Biotec)
  • Leukon Investments
  • Lime Asset Management
  • Lonza
  • Louisiana State University (LSU)
  • Luminous BioSciences
  • Lundbeckfonden Ventures
  • Lysogene
  • Marsala Biotech
  • Maryland Technology Development Corporation (TEDCO)
  • Massachusetts General Hospital
  • Massachusetts Institute of Technology
  • MassBiologics
  • Medarex
  • Medigen Biotechnology
  • MedImmune
  • Medtronic
  • MeiraGTx
  • Mercia Technologies
  • Merck
  • Mercury Fund
  • Meridian Life Science
  • MERITZ Securities
  • MidCap Financial
  • Milo Biotechnology
  • Miltenyi Biotec
  • Mita Securities
  • Mitsubishi Tanabe Pharma
  • Mitsubishi UFJ Capital
  • Moderna Therapeutics
  • Molecular Diagnostic Services(MDS)
  • MolMed
  • MOLOGEN
  • Momotaro-Gene
  • Morningside Group
  • Movember Foundation
  • Myonexus Therapeutics
  • NanoCor Therapeutics
  • NanoDimension Venture Firm
  • NantCell
  • Nantes Gene Therapy Institute
  • National Cancer Institute (NCI)
  • National Eye Institute (NEI)
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • National Institute on Drug Abuse (NIDA)
  • National Institutes of Health (NIH)
  • National MPS Society
  • National Securities Corporation (NSC)
  • Nature Technology
  • Neuralgene
  • Neurotech Pharmaceuticals
  • New Energy and Industrial Technology Development Organization (NEDO)
  • New Enterprise Associates (NEA)
  • New Leaf Venture Partners
  • New River Management
  • NewVa Capital Partners
  • Nightstar Therapeutics
  • Nohla therapeutics
  • Novartis
  • Novartis Venture Fund
  • Novasep
  • Novo Seeds
  • Oberland Capital
  • Okairos
  • Omega Fund Management
  • Omnes Capital
  • Omnia Biologics
  • Oncolys BioPharma
  • OncoSec Medical
  • OrbiMed Advisors
  • ORCA Therapeutics
  • Orchard Therapeutics
  • ORI Capital
  • Osage University Partners
  • Oxford BioMedica
  • Oxford Finance
  • Oxford Genetics
  • PacificGMP (a part of Abzena Group)
  • Pangu BioPharma
  • Panmure Gordon
  • Paragon Bioservices
  • Partners Innovation Fund
  • Pavilion Capital Partners
  • PeriphaGen
  • Pfizer
  • PhaseRx
  • PlasmidFactory
  • PNP Therapeutics
  • Polaris Partners
  • Pontifax
  • Posco Capital Partners
  • Poseida Therapeutics
  • Precision BioSciences
  • Profectus BioSciences
  • Promethera Biosciences
  • PsiOxus Therapeutics
  • Puresyn
  • Quethera
  • RA Capital Management
  • RBV Capital
  • Red Sanfilippo Foundation
  • Redmile Group
  • Regeneron Pharmaceuticals
  • REGENXBIO
  • Relieve Genetics
  • Renova Therapeutics
  • RetroSense Therapeutics (acquired by Allergan)
  • Rev1 Ventures (previously known as TechColumbus)
  • REYON Pharmaceutical
  • Richter-Helm BioLogics
  • Ridgeback Capital Management
  • Roche
  • Roche Venture Fund
  • Rock Springs Capital Management
  • Rocket Pharma
  • RTW Investments
  • Rusnano
  • SAB Technology
  • Sabby Capital
  • San Raffaele Telethon Institute for Gene Therapy (SR-TIGET)
  • SanBio
  • Sanfilippo Children's Foundation
  • Sanfilippo Children's Research Foundation (SCRF)
  • Sanfilippo Research Foundation (SRF)
  • Sangamo Therapeutics
  • Sanofi
  • Sanofi Genzyme
  • Sanofi-Genzyme BioVentures
  • Santen Pharmaceutical
  • Sarepta Therapeutics
  • Scancell
  • Sectoral Asset Management
  • Selecta Biosciences
  • Seventure Partners' Health for Life Capital fund
  • Sham Innovation Sant
  • Shanghai Sunway Biotech
  • Shenzhen Qianhai Taxus Industry Capital Management
  • Shenzhen Sibiono GeneTech
  • Shinhan Capital
  • Shinsei Corporate Investment
  • Shire
  • Sigma-Aldrich (now a part of Merck)
  • SignaGen Laboratories
  • Sino Biological
  • SIRION Biotech
  • Skolkovo Foundation
  • SMS Investments
  • Sofinnova Ventures
  • Song Hong Fang
  • Spark Therapeutics
  • Sphera Global Health Care
  • SR One (investment arm of GlaxoSmithKline)
  • Stratophase
  • StrideBio
  • SV Life Sciences Investors
  • Syncona Partners (a subsidiary of the Wellcome Trust)
  • Synpromics
  • Synthace
  • System Biosciences
  • T. Rowe Price
  • Takeda Pharmaceutical
  • Target ALS Foundation
  • Targeted Technology Fund
  • Targovax
  • Team Sanfilippo
  • Tekes
  • Temasek Holdings
  • Tethys Ventures
  • The Finnish Funding Agency for Technology and Innovation
  • The French Muscular Dystrophy Association (AFM-Tlthon)
  • The Jagen Group
  • The Scripps Research Institute
  • TheraBiologics
  • TheraVectys
  • Thermo Fisher Scientific
  • Third Rock Ventures
  • Third Security
  • TissueGene
  • Tocagen
  • Transgene
  • Tufts Financial Group (TFG)
  • Turenne Sant
  • TVM Capital
  • Two Blades Foundation
  • UB Securities
  • UCL Technology Fund
  • UK Innovation & Science Seed Fund (UKI2S, previously known as The Rainbow Seed Fund)
  • uniQure
  • University of California
  • University of Florida
  • University of Minnesota
  • University of Pennsylvania
  • University of Texas
  • University of Washington
  • Valneva
  • Vascular Biologics (also known as VBL Therapeutics)
  • VCN Biosciences
  • Vectalys
  • Vector Biolabs
  • Vecura (Karolinska University Hospital)
  • Venrock
  • Versant Ventures
  • Vertex Pharmaceuticals
  • VESSL (previously known as MultiGene Vascular Systems )
  • Vibalogics
  • Vical
  • Vigene Biosciences
  • Viking Global Investors
  • Virginia Biosciences Health Research Corporation (VBHRC)
  • ViroMed
  • Virovek
  • Virttu Biologics (acquired by Sorrento Therapeutics)
  • VIVEbiotech
  • Vivet Therapeutics
  • Vivo Capital
  • Voyager Therapeutics
  • Waisman Biomanufacturing
  • Wellcome Trust
  • Wellington Management Company
  • Wellstat Ophthalmics
  • Whitesun Healthcare Ventures
  • WI Harper Group
  • Woodford Investment Management
  • WuXi AppTec
  • XyloCor Therapeutics
  • Ysios Capital
  • Yuhan
  • ZIOPHARM Oncology

For more information about this report visit https://www.researchandmarkets.com/research/x97328/gene_therapy?w=12

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Related Topics: Genomics

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