Gene therapy R&D market is projected to grow at a CAGR of 30.1% by 2032: Visiongain Reports Ltd

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 Diseases
There 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 Approvals
The 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?
Visiongain’s 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 Growth
The 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 2032
Gene 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 Opportunities
While 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 Prospects
Contract 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 Landscape
The 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.

Recent Developments

  • In June 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending conditional marketing authorization (CMA) to BioMarin Pharmaceutical Inc. for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The one-time infusion is planned to be marketed under the brand name ROCTAVIAN™ (valoctocogene roxaparvovec).
  • In May 2022, ASC Therapeutics joined as a full partner with the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), managed by the Foundation for the National Institutes of Health (FNIH).
  • In May 2022, Ultragenyx Pharmaceutical Inc. & Abeona Therapeutics Inc. entered into a license agreement for adeno-associated virus gene therapy ABO-102 (UX111) for the treatment of Sanfilippo syndrome type A.
  • In April 2022, Pfizer Inc. announced the launch of its U.S. sites in the Phase 3 study evaluating the investigational fordadistrogene movaparvovec & mini-dystrophin gene therapy in patients with Duchenne muscular dystrophy.
  • In April 2022, the U.S. FDA granted commercial approval to Novartis for its Durham, N.C. site, which is a gene therapy manufacturing facility. This authorization enables the cutting-edge, 170,000 –square-foot facility to manufacture, test, and commercially market Zolgensma as well as gene therapy products for ongoing and upcoming clinical trials. The achievement of this milestone allows Novartis Gene Therapies to open a second commercially-licensed manufacturing plant, complementing the Libertyville, Illinois, location that received approval for the production and distribution of Zolgensma® (onasemnogene abeparvovec) in 2019.  

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Find more Visiongain research reports on Pharma sector click on the following links:

  • Rare Diseases Market Report 2021-2031
  • Advanced Therapy Medicinal Products (ATMP) Market Report 2021-2031
  • Viral Vectors & Plasmid DNA Manufacturing Market Report 2021-2031

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About Visiongain

Visiongain is one of the fastest-growing and most innovative independent market intelligence around, the company publishes hundreds of market research reports which it adds to its extensive portfolio each year. These reports offer in-depth analysis across 18 industries worldwide. The reports cover 10-year forecasts and are hundreds of pages long, with in-depth market analysis and valuable competitive intelligence data. Visiongain works across a range of vertical markets, which currently can influence one another, these markets include automotive, aviation, chemicals, cyber, defense, energy, food & drink, materials, packaging, pharmaceutical, and utility sectors. Our customized and syndicated market research reports mean that you can have a bespoke piece of market intelligence customized to your very own business needs.

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PR at Visiongain Reports Limited
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