This Report Analyzes Emerging Market Forces, Highlights Industry Partnerships and Deal-Making, and Captures the Progression of Gene Therapy Products
Gene therapy is a novel area of medicine that involves the insertion of functional genes into cells to replace faulty or missing genes in order to treat genetic diseases. This report reviews key developments from across the gene therapy sector, evaluating the industry's commercial successes and failures, regulatory issues across key global markets, manufacturing and scale-up challenges, pricing and reimbursement considerations, and requirements for long-term technology adoption. It evaluates trends in scientific publications, clinical trials, and patent activity, as well as presents market size determinations with 5-year projections.
Representing a “new frontier of medicine,” gene therapy made its grand arrival in the U.S. in 2017 through the FDA approval of chimeric antigen receptor (CAR) T-cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), as well as the gene therapy Luxturna (voretigene neparvovec). Kymriah and Yescarta represent gene-modified cell therapies in which the T cells of the patient are genetically modified ex vivo by the addition of a gene and infused back into the patient's body to seek out and destroy cancer cells. Luxturna is a gene therapy product consisting of modified viral particles loaded with a therapeutic gene that is injected into the cornea of a patient to improve functional vision. These products have revolutionized the gene therapy industry, giving hope to thousands of patients suffering from incurable diseases and expanding interest in gene therapy technology adoption on a global basis. Thus far, China, Australia, New Zealand, South Korea, India, Japan, Europe, Canada, and the US. have approved gene therapy products, indicating worldwide adoption is underway.
Since 2012, hundreds of clinical trials have confirmed the efficacy and safety of gene therapy for a range of indications, including blindness, hemophilia, immunodeficiencies, inherited anemia and cancer immunotherapy with CAR-T cells. Because some of these clinical trials have successfully reached Phase III, it is anticipated that the gene therapy industry will see up to 50 or 60 approved products by 2030. The handful of currently approved gene therapies and anticipated future approvals will likely be used to treat more than a half million patients around the world by 2030.
The successful launch of gene therapy products has also lured substantial investment into the industry and attracted a growing number of market competitors, ranging from spin-offs to large conglomerates. Pharmaceutical companies are showing an appetite for gene therapy technologies, engaging in licensing agreements, R&D collaborations, and M&A deal making aimed to bring new gene therapies to market. Ranging from small to large, gene therapy companies are emerging in major healthcare markets worldwide.
The main objectives of this report are to provide the reader with the following details:
- Approved gene therapy products in the global market.
- Number of gene therapy products to gain FDA approval by 2024.
- Market value of the forthcoming gene therapy products.
- Leading gene therapy startups.
- Select anticipated clinical data and events in gene therapy space.
- Gene therapy products approved in Australia, New Zealand, China, South Korea, India, Japan, Europe, Canada and the U.S.
- Gene therapy products with RMAT designation.
- Pricing of gene therapy products.
- Manufacturing cost of gene therapy products in manual, partially-automated and fully-automated facilities.
- Pharma companies having in-house capability for gene therapy manufacture.
- Big and Mid-Pharma companies relying on outsourced manufacturing of gene therapies.
- Gene therapy manufacturing capacities in the U.S. and Europe.
- Select CMOs/CDMOs in gene therapy space.
- Projected outsourcing market for the manufacture of gene therapy products, 2018-2025.
- Global market for gene therapy products by therapy type and geography, 2018-2025.
- Profiles of companies that are developing products for gene therapy.
In summary, this report analyzes emerging market forces, highlights industry partnerships and deal-making, and captures the progression of gene therapy products across various states of development. Because this market is rapidly expanding, this report is your guide to understanding gene therapy research advances, clinical breakthroughs, and future directions.
Claim this report to:
1. Identify the preclinical and clinical product pipeline for gene therapy products globally.
2. Reveal the identities of competitors developing gene therapies and supporting technologies.
3. Determine company strategy or purse external funding from investors for gene therapy technologies.
4. Access gene therapy market size determinations, market segmentation, and 5-year projections through 2024.
5. Make smarter decisions, faster.
The publisher of this report has a unique understanding of the rapidly evolving Gene Therapy (GT) Industry. The author has collected more than decade of historical data on the industry, providing it with a robust historical database of data on which to make future market projections.
Table of Contents
Companies Mentioned
- 4D Molecular Therapeutics, LLC
- Abeona Therapeutics, Inc.
- Adaptimmune Therapeutics, plc
- Advanced Bioscience Laboratories, Inc
- Adverum Biotechnologies, Inc
- Akouos, Inc
- Aldevron, LLC
- Allogene Therapeutics, Inc
- Ambys Medicines, Inc.
- American Gene Technologies International, Inc
- Amgen, Inc
- Amicus Therapeutics, Inc.
- AskBio
- Audentes Therapeutics, Inc.
- AveXis, Inc.
- AVROBIO, INC.
- Benitec Biopharma Ltd
- Bioverativ, Inc.
- bluebird bio, Inc
- BlueRock Therapeutics
- Brammer Bio, LLC
- Carina Biotech
- CARsgen Therapeutics, Ltd
- Casebia Therapeutics
- Celyad SA
- Cevec Pharmaceuticals GmbH
- CombiGene AB
- CRISPR Therapeutics AG
- Delphi Genetics S.A
- DNAlite Therapeutics, Inc.
- Editas Medicine, Inc
- Elixirgen Therapeutics, LLC
- Fibrocell Technologies, Inc.
- FinVector Vision Therapies Oy
- Freeline Therapeutics, Ltd.
- GEG Tech
- Gemini Therapeutics, Inc
- Generation Bio Co.
- Genethon
- Homology Medicines, Inc
- Horama SAS
- Intellia Therapeutics Inc
- Kite Pharma, Inc.
- Kolon Lifesciences, Inc.
- Krystal Biotech, Inc.
- LakePharma
- Lonza Group, Ltd.
- Lysogene S.A
- MeiraGTx Holdings plc
- MolMed S.p.A
- Mustang Bio, Inc.
- Myonexus Therapeutics, Inc
- Nightstar Therapeutics plc
- Novartis AG
- Orchard Therapeutics plc
- Oxford Genetics, Ltd.
- OxfordBioMedica plc
- Paragon Bioservices, Inc.
- Posedia Therapeutics, Inc.
- Precision BioSciences, Inc.
- Prevail Therapeutics, Inc.
- PTC Therapeutics, Inc.
- Quethera, Ltd.
- REGENXBIO, INC.
- Rocket Pharmaceuticals, Inc.
- Sangamo Therapeutics, Inc
- Sarepta Therapeutics, Inc
- Sibiono GeneTech Co. Ltd.
- Sirion Biotech, GmbH
- Solid Biosciences, Inc
- Spark Therapeutics, Inc
- Synpromics, Ltd.
- Takara Bio, Inc
- The Cell and Gene Therapy Catapult
- Translate Bio, Inc.
- Ultragenyx Pharmaceutical, Inc.
- uniQure N.V.
- Vigene Biosciences
- Virovek, Inc.
- Vivebiotech SL
- Vivet Therapeutics
- Voyager Therapeutics, Inc
- Vybion, Inc.
Methodology
The content and statistics contained within the publisher's reports are compiled using a broad range of sources, as described below.
Input Sources
- Clinical Trial Databases (ClinicalTrials.gov, International Clinical Trials Registry Platform, European Union Clinical Trials Register, Chinese Clinical Trial Registry, Others)
- Scientific Publication Databases (PubMed, Highwire Press, Google Scholar)
- Patent Databases (United States Patent and Trade Office, World Intellectual Property Organization, Google Patent Search)
- Grant Funding Databases (RePORT Database, CIRM, MRC, Wellcome Trust - UK, Others)
- Product Launch Announcements (Trade Journals, Google News)
- Industry Events (Google News, Google Alerts, Press Releases)
- Company News (SEC Filings, Investor Publications, Historical Performance)
- Social Analytics (Google Adwords, Google Trends, Twitter, Topsy.com, Hashtagify.me, BuzzSumo.com)
- Interviews with Stem Cell Industry Leaders
Research & Analysis Methodologies
The publisher employs the following techniques for deriving its market research:
- Historical Databases: As the first and only market research firm to specialize in the stem cell industry, the publisher has 13+ years of historical data on each segment of the stem cell the industry. This provides an extremely rare and robust database for establishing market size determinations, as well as making future market predictions.
- Prolific Interviews with Industry Leaders: As the global leader in stem cell industry data, the publisher has interviewed hundreds of leaders from across the stem cell industry, including the CEO of FUJIFILM CDI, FUJIFILM Irvine Scientific, Pluristem Therapies, Celularity, and many others.
- Industry Relationships: The research team and its President/Founder, Cade Hildreth, Chair and present at a wide range of stem cell industry events, including Phacilitate's Advanced Therapies Week, World Stem Cell Summit (WSCS), Perinatal Stem Cell Society Congress, AABB's International Cord Blood Symposium (ICBS), and other events hosted within the U.S. and worldwide.
- Global Integrated Feedback: Because the publisher maintains the world's largest stem cell industry news site that is read by nearly a million unique readers per year and the company has large social media audiences (25.7K+ followers on Linked, 21.2K+ followers on Twitter, and 4.3K+ followers on Facebook), the publisher is able to publish content relevant to the industry and receive immediate feedback/input from a global community of readers. In short, the publisher's data is crowd-sourced from market participants worldwide, including those in diverse geographic regions.
- Preliminary Research: In addition to the interviews described above, the publisher conducts market surveys, executes social media polls, and aggregates market data from stem cell industry announcements, press releases, and corporate filings/presentations.
- Secondary Research: The publisher summarizes, collects and synthesizes existing market research that is relevant to the market area of interest.
- Future Projections: Using the resources described above, the publisher is uniquely positioned to make future projections about market size, market growth by segment, market trends, technology evolution, funding activities (financing rounds, M&A, and IPOs), and importantly, market leadership (market share by company).
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