Visiongain has published a new report entitled Gene Therapy R&D 2022-2032. It includes profiles of Gene Therapy R&D and Forecasts Market Segment by Disease {Cancer, Rare Diseases (Oncologic, Non-oncologic), Cardiovascular Diseases, Ophthalmic Diseases, Haematology, Neurological, Diabetes Mellitus, Other Diseases)}, Vector {Viral (Retrovirus, Adenovirus, AAV, Lentivirus, Others), Non-viral (Naked DNA, Gene Gun, Electroporation, Lipofection)}, Techniques (Gene Augmentation Therapy, Gene Replacement Therapy), Participants (Small/Medium Pharma & Biotech, Universities & Research Institutes, Hospitals, Government & Public Bodies, Big Pharma) PLUS COVID-19 Impact Analysis and Recovery Pattern Analysis (V-shaped, W-shaped, U-shaped, L-shaped) Profiles of Leading Companies, Region and Country.
The gene therapy R&D market was valued at US$1,653.0 million in 2021 and is projected to grow at a CAGR of 30.1% during the forecast period 2022-2032.
Gene Therapies Are Projected to Provide Potential Benefits for a Range of Rare DiseasesThere are about 7,000 rare diseases reported, but only a few hundred have therapies approved. Gene therapy is especially important for patients with rare disorders, as more than 80% of them have a documented monogenic (single-gene) cause. Rather than treating the disease, conventional small molecule medications often work by reducing symptoms. When managing a chronic condition, this may indicate that the medication or drugs used to control the condition are administered on a daily basis. Gene therapy, on the other hand, has the ability to remedy structural genetic disorders, rather than merely treating symptoms.
In October 2021, the U.S. FDA, National Institutes of Health (NIH), ten pharmaceutical companies & five non-profit groups joined forces to pace up the development of gene therapies for addressing the 30 million rare diseases patient pool across the North American region. Only two genetic disorders now have FDA-approved gene treatments, despite the fact that there are about 7,000 rare diseases. Hence, partnerships between pharmaceutical companies to tackle rare diseases is likely to fuel the demand for gene therapy in rare diseases treatment during the forecast period.
Furthermore, gene therapies provide the potential of a one-time cure for a range of rare disorders for which there are actually no clear clinical alternatives. With multiple gene therapy drugs securing FDA clearance, recent developments in genetic engineering and recombinant viral vector production have fuelled interest in the field.
The Asia Pacific Has Witnessed an Increase in Early ApprovalsThe regulatory framework for supporting fast marketing authorizations for advanced medicines to address unmet medical needs has been developed by regulatory agencies as a result of the Asia-Pacific region's rapid growth in advanced therapy research and development. With the introduction of regulatory frameworks by the authorities, the region has witnessed an increase in early approvals of new medicines. These approvals showed that regional regulators are more prepared to review and authorize cutting-edge treatments. To introduce these cutting-edge medications into Asia-Pacific, numerous pharmaceutical companies are making use of these new regulatory paths to take a competitive edge in the market.
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How has COVID-19 had a Significant Impact on the Gene Therapy R&D Market?All biopharmaceutical firms have been impacted by the COVID-19 pandemic, but many CGT companies have been hit particularly severely because of their complex manufacturing and distribution models and financial structures. Businesses' decisions will have a significant impact on both the present patients with CGT and those who stand to gain from the next wave of innovation being explored by CGT companies. The manufacturing and treatment supply of the CGT industry, as well as scientific and clinical advancement and business operations, have all been severely hampered by the COVID-19 problem. The COVID-19 impact, which has been more severe in some nations compared to others, has not affected some CGT enterprises very negatively. However, since the supply chains used to manufacture CGTs are complex and tightly regulated, CGT companies have discovered that they are especially susceptible to interruptions in regions where the new coronavirus has been widespread.
How this Report Will Benefit you?Visiongains 462 page report provides 169 tables and 228 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the gene therapy R&D market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for gene therapy R&D. Get the financial analysis of the overall market and different segments including service type, molecule type, and therapeutic area. We believe that high opportunity remains in this fast-growing gene therapy R&D market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report would help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.
What are the Current Market Drivers?
Increasing Investments Driving Market GrowthThe pandemic has highlighted the relevance of cell and gene therapies, as well as genetic medicines in specific. If the investment by venture capitalists maintains capital flows, the momentum will be maintained during the forecast years. Large biopharmaceutical firms are anticipated to invest or acquire innovative technologies & support valuation, even if the capital markets funding climate deteriorates; Visiongain anticipates that the gene therapy industry will continue to attract investor interest over the forecast period.
Even as private companies like enGene plan to go public, Generation Bio's valuation has grown to $2 billion due to non-viral gene therapy. Longer term, synthetic biology investments, such as transgene engineering, are expected. In cell-based treatment, we see more investment potential in solid tumors and off-the-shelf pluripotent stem cell technology.
Recognizing the promise of these cutting-edge developments, large pharmaceutical firms sought out partnerships with smaller, more agile biotech start-ups. Janssen (Johnson & Johnson) & Fate Therapeutics agreed to a US$100 million upfront deal to develop cell-based immunotherapies for hematologic and solid tumors. Biogen & Sangamo have agreed to a US$350 million upfront contract to create zinc finger protein-based gene regulation therapies for neurodegenerative diseases. These agreements aided in the receipt of US$3 billion in upfront fees from corporate alliances, as well as clinical and regulatory milestones worth billions more.
Technological Advancements to Fuel Market Growth Through 2032Gene therapy, both as a modern medical technique and as a biomedical business, has a bright future in terms of technology and industry promotion. Researchers may use genome editing technology to break, alter, and edit particular genes in a DNA sequence-specific manner. However, genome editing carries the possibility of unintentional editing of genes with identical DNA sequences, a phenomenon known as the off-target effect. Genome editing has the ability to create lasting changes in the genome.
Furthermore, the genome editing tool CRISPR-Cas9 is making waves in the scientific area. It has a wide range of possible uses and is quicker, less expensive, and more accurate than earlier methods of DNA editing. Animal research has been transformed by CRISPR/Cas9 technology, as has human gene therapy, medical research, and plant science study. This method has become increasingly useful in recent years for carrying out precise gene targeting and alterations, such as gene insertions and deletions, gene replacements, and single-base pair conversions. Over the forecast period, the market for gene therapy R&D is expected to grow as a result of significant breakthroughs in this field.
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Where are the Market Opportunities?
Growing Number of Clinical Trials to Offer Lucrative Growth OpportunitiesWhile 2022 will be a significant year for gene treatments that target rare diseases, we also anticipate that clinical readouts on medicines that target common diseases will garner media attention. It was one of the pivotal events when Vertex Pharmaceuticals' cell therapy effectively cured one patient's type 1 diabetes in 2021. The first gene therapy approval for a prevalent illness in the U.S. & Europe may occur within the next several years due to Phase 3 studies for indications like congestive heart failure, critical limb ischemia, diabetic peripheral neuropathy & macular degeneration. Approximately, 59% of the 2,406 clinical studies in the area focus on prevalent diseases. Additionally, 62% of academic and government-sponsored studies are against 56% for commercial trials, demonstrating the industry's greater involvement in the study of rare diseases. In addition, when compared to university and government sponsors, the industry places more emphasis on rare haematological diseases like hemophilia and sickle cell anaemia as well as rare ophthalmological conditions like retinitis pigmentosa.
Nearly two-thirds of all trials for rare diseases focus on treating rare malignancies, which continue to be the main goal. Additionally, inherited haematological conditions like SCDs & hemophilia, ophthalmological indications like retinitis choroideremia & pigmentosa, and other rare monogenic disorders like mucopolysaccharidosis, Duchenne muscular dystrophy & Wilson disease have drawn interest from cell and gene therapy developers.
Even while the proportion of trials targeting both common and rare diseases is roughly similar over phases, the prominent diseases being targeted are evolving. Phase 3 studies that target a common disease include 23% of musculoskeletal problems, but just 7% of Phase 1 trials, including bone fractures, osteoarthritis, and sports injuries. Other common disease categories targeted include viral diseases like HIV and CNS disorders such as Alzheimer's & Parkinson's disease.
There is also a change in the predominant disease category toward focusing on more complicated, polygenic diseases. We are witnessing a gradual transition within the CNS disorders, from more complex, polygenic disorders like Alzheimer's disease, autism & even treatment-resistant bipolar disorder & depression, to conditions such as spinal cord injury (SCI), traumatic brain injury (TBI), and neuropathic pain.
Facility Expansion Anticipated to Offer Lucrative Growth ProspectsContract manufacturers, on whom new gene & cell therapy businesses rely for early-stage development, are experiencing a lack of viral vector manufacturing capacity as a result of the increase in clinical-stage start-ups. When these companies reach commercial scale, they frequently prefer to maintain total control over their manufacturing in order to avoid the difficulties of outsourcing. As a result, biotech firms began to create expansion plans, set up internal teams, and/or ask for site consultant guidance. These professionals support the strenuous search for suitable research and development facilities or, increasingly, new construction sites in competitive real estate markets.
These in-house capabilities allow gene and cell therapy companies to rapidly scale up production from clinical batches to commercial scale, even when therapies are still in the research and development stage. This also allows for co-location with drug research and development operations, ensuring smooth technology transfer and minimal disruption, particularly during clinical trials.
As a result, there is a pressing need for time-to-market, so the chosen emphasis is on existing buildings, which have become increasingly difficult to come by in developed biotech hubs due to market demand. These hubs provide benefits such as tailored university programs and the involvement of other gene and cell therapy companies (both rivals and potential collaborators), all of which combine to create a target-rich environment for the talent they are all looking for. While all ventures are cost-sensitive, venture-funded businesses are more concerned with cost, and the need to reduce both upfront and ongoing cash outlay.
Competitive LandscapeThe major players operating in the gene therapy R&D market are Astellas Pharma Inc., American Gene Technologies, Applied Genetic, Bayer, Benitec BioPharma, Biogen, Bluebird Bio, Bristol Myers Squibb, Calimmune, Inc. (CSL Behiring), Cellectis, F. Hoffmann-La Roche Ltd., GeneQuine Biotherapeutics, GenSight Biologics, Gilead Lifesciences, Inc., Novartis AG, OCUGEN, INC., Orchard Therapeutics, Oxford Biomedica, Pfizer, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Sanofi, Sarepta Therapeutics, Inc., Spark Therapeutics (Subsidiary of Roche), Takeda Pharmaceuticals, Taysha GTx, Transgene, UniQure NV, Voyager Therapeutics, and ViGeneron. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launches.
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Gene therapy R&D market is projected to grow at a CAGR of 30.1% by 2032: Visiongain Reports Ltd - GlobeNewswire
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- Cell and gene therapy will be top industry trend for pharma in 2024 - The Financial Express - December 29th, 2023
- Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy - AJMC.com Managed Markets Network - December 29th, 2023
- Global Cell and Gene Therapy Manufacturing Industry is projected to surpass a valuation of US$ 240 B - PharmiWeb.com - December 29th, 2023
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- Adeno-associated Virus Gene Therapy Market is Projected to Grow at a CAGR of 43.4% from 2023-2033 - EIN News - December 29th, 2023
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- The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia - PMLiVE - October 13th, 2022
- Gene Therapy Hits Its Stride in the Clinic - Genetic Engineering & Biotechnology News - October 13th, 2022
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- Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene... - October 13th, 2022
- Health Alert for Parents: How one boy is thriving following treatment with a gene therapy after receiving an early diagnosis - PR Newswire - October 13th, 2022
- M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress - Business Wire - October 13th, 2022
- Two Cell and Gene Therapies Manufactured at Lonza Houston Reach FDA Approval - Contract Pharma - October 13th, 2022
- Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive - October 13th, 2022
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- Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead - October 13th, 2022
- What Do We Know About Gene Therapy and Cystic Fibrosis? - The Epoch Times - October 13th, 2022
- CSafe and BioLife Solutions, Inc. announce partnership to expand supply chain solutions for the cell and gene therapy market - PR Newswire APAC - PR... - October 13th, 2022
- Genomics in Cancer Care Market is estimated to be US$ 72.61 billion by 2032 with a CAGR of 16.3% during the forecast period 2032 - By PMI -... - October 13th, 2022
- After $100M deal, J&J links gene therapy to improved vision in early-phase trial - FierceBiotech - October 4th, 2022
- Bayer Head Admits COVID-19 Vaccine is Gene Therapy - October 4th, 2022
- New Gene Therapy Shows Promise for Treating Age Related Macular ... - October 4th, 2022
- BioMarin resubmits its hemophilia gene therapy to the FDA - BioPharma Dive - October 4th, 2022
- Why Philly ranks #2 among best cell and gene therapy hubs in the US - Technical.ly - October 4th, 2022
- AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire - October 4th, 2022
- bluebird's (BLUE) Gene Therapy Approvals to Drive the Top Line - Zacks Investment Research - October 4th, 2022
- Researchers Develop Potential Gene Therapy to Treat Blindness - Managed Healthcare Executive - October 4th, 2022
- Voyager Therapeutics Announces Data Presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy - GlobeNewswire - October 4th, 2022
- Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application... - October 4th, 2022
- Sanofi partners with Scribe to gain gene editing tools for cell therapy work - BioPharma Dive - October 4th, 2022
- Real Endpoints Marketplace announces collaboration with bluebird bio to help scale delivery of a first-of-its-kind value-based contract for one-time... - October 4th, 2022
- Vertex given green light to seek US approval of CRISPR-based therapy - BioPharma Dive - October 4th, 2022
- Atsena Therapeutics Announces Positive Results from Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital... - October 4th, 2022
- Meet with the OrganaBio executives in-person at The Cell & Gene Meeting on the Mesa - Business Wire - October 4th, 2022
- Ocular Gene Therapy: A Literature Review With Focus on Current Clinical Trials - Cureus - September 25th, 2022
- Will experience support use of first-ever retinal gene therapy? - Ophthalmology Times - September 25th, 2022
- Rejuvenate Bio CEO highlights ambitious approach of using gene therapy to reverse aging - MedCity News - September 25th, 2022
- Emulate Announces New Gene Therapy Application Enabling Accelerated Development of Treatments for Genetic Diseases with Organ-on-a-Chip Technology -... - September 25th, 2022
- CSL flexes gene therapy muscle with latest drug - Sydney Morning Herald - September 25th, 2022
- FDA Updates for the Week of Sept. 19, 2022 - Managed Healthcare Executive - September 25th, 2022
- AVROBIO Receives Rare Pediatric Disease Designation From FDA For First Gene Therapy In Development For Cy - Benzinga - September 25th, 2022
- Preparing Europe for a new generation of innovative therapies - Open Access Government - September 25th, 2022
- KSQ Therapeutics Announces Leadership and Board Additions - Business Wire - September 25th, 2022
- Hemophilia gene therapies from BioMarin, CSL pick up an early ICER endorsement - FiercePharma - September 16th, 2022
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- Hopkinton bioscience company forms advisory board for cell and gene therapy - Worcester Business Journal - September 16th, 2022
- Gene Therapy Restores Some Vision in Colorblind Children - Pharmacy Times - September 16th, 2022
- Coave Therapeutics partners with IMN on gene therapy - Labiotech.eu - September 16th, 2022
- MeiraGTx's Industry-Leading Gene Therapy Manufacturing Facility in Shannon, Ireland Formally Introduced by Head of Irish Government -... - September 16th, 2022
- Bluebird CFO resigns ahead of gene therapy launch - BioPharma Dive - September 16th, 2022
- Enrollment completed in STARLIGHT Phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease - Ophthalmology Times - September 16th, 2022
- Global Cancer Gene Therapy Market to Reach $11.35 Billion by 2030 at a CAGR of 23.3% - PR Newswire - September 16th, 2022
- How WhiteLab Genomics is using AI to aid gene and cell therapy development - TechCrunch - September 16th, 2022
- CAR-T Cell Therapy Market Segmentation and Forecast Analysis up to 2027 - BioSpace - September 16th, 2022
- Cluster-busters against Cancer and a Potential New Gene Therapy for LCA - BioSpace - September 16th, 2022
- Ori Biotech and CTMC team up on cell therapy delivery - BioPharma-Reporter.com - September 16th, 2022
- Adverum (ADVM) Doses First Patient in the Mid-Stage wAMD Study - Zacks Investment Research - September 16th, 2022
- Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today - September 8th, 2022
- A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review - September 8th, 2022
- Myrtelles rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European... - September 8th, 2022
- Sangamo presses ahead with Fabry disease gene therapy - BioPharma Dive - September 8th, 2022
- The gene therapy that could transform the lives of millions - ABC News - September 8th, 2022
- As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer - September 8th, 2022
- Mayflower BioVentures to Announce New Cell & Gene Therapy Companies within Months - BioSpace - September 8th, 2022
- Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire - September 8th, 2022
- AnGes : Announces Discontinuation of Development of HGF Gene Therapy Product for Additional Indication of Chronic Arterial Occlusive Disease with Rest... - September 8th, 2022