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§ A detailed review of the overall landscape of gene therapies and genome editing therapies, including information on various drug / therapy developer companies, phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others) and target disease indication(s), information on gene type, type of vector used, type of therapy (ex vivo and in vivo), mechanism of action, type of gene modification (gene augmentation, oncolytic viral therapy and others) and special drug designation (if any).
§ A discussion on the various types of viral and non-viral vectors, along with 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 engaged in developing gene therapies, and a bull's eye analysis, highlighting the distribution of clinical-stage pipeline candidates by phase of development, type of vector and type of therapy (ex vivo and in vivo).
§ A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
§ Detailed profiles of marketed and phase II/III and gene therapies, including a brief history of development, information on current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, along with information on the developer company.
§ An elaborate discussion on the various commercialization strategies that can be adopted by drug developers for use across different stages of therapy development, namely prior to drug launch, at / during drug launch and post-marketing.
§ A review of various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.
§ An in-depth analysis of the various patents that have been filed / granted related to gene therapies and genome editing therapies, since 2016. The analysis also highlights the key parameters associated with the patents, including information on patent type (granted patents, patent applications and others), publication year, regional applicability, CPC classification, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.
§ An analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019. The analysis also provides information on the key value drivers and deal multiples related to the mergers and acquisitions that we came across.
§ An analysis of the investments made at various stages of development in companies that are focused in this area, between 2014-2019, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings.
§ An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio.
§ A case study on the prevalent and emerging trends related to vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
§ A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.
§ An analysis of the various factors that are likely to influence the pricing of gene-based therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.
The report also features the likely distribution of the current and forecasted opportunity across important market segments, mentioned below:
§ Key therapeutic areas
§ Autoimmune disorders
§ Cardiovascular diseases
§ Genetic disorders
§ Hematological disorders
§ Metabolic disorders
§ Ophthalmic disorders
§ Oncological disorders
§ Type of vector
§ Adeno associated virus
§ Herpes simplex virus type 1
§ Plasmid DNA
§ Vaccinia Virus
§ Type of therapy
§ Ex vivo
§ In vivo
§ Type of gene modification
§ Gene augmentation
§ Oncolytic therapy
§ Route of administration
§ Key geographical regions
§ North America
The report includes detailed transcripts of discussions held with the following experts:
§ 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)
§ Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela)
§ Jeffrey Hung (CCO, Vigene Biosciences)
§ Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory)
§ Michael Triplett (CEO, Myonexus Therapeutics, acquired by Sarepta Therapeutics)
§ Robert Jan Lamers (CEO, Arthrogen)
§ Tom Wilton (Chief Business Officer, LogicBio Therapeutics)
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Key Questions Answered
§ Who are the leading industry players in this market?
§ How big is the development pipeline and which new indications are coming in focus? Which vectors are being used for effective delivery of the therapeutic agents?
§ Who are the key investors in the gene therapy market?
§ How is the current and future market opportunity likely to be distributed across key market segments?
§ What kind of commercialization strategies are being adopted by gene therapy developers?
§ What are the different pricing models and reimbursement strategies used for gene therapies?
§ What are the prevalent R&D trends related to gene therapies?
§ What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
§ Who are the key CMOs / CDMOs that claim to supply viral / plasmid vectors for gene therapy development?
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2. CAR-T Therapies Market (2nd Edition), 2019-2030
3. Global T-Cell (CAR-T, TCR, and TIL) Therapy Market (4th Edition), 2019 – 2030
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