Gene Therapy R&D Market Report 2024-2034

Visiongain has published a new report entitled Gene Therapy R&D Market Report 2024-2034: Forecasts by Disease (Cancer, Rare Diseases (Oncologic, Non-oncologic), Cardiovascular Diseases, Ophthalmic Diseases, Haematology, Neurological, Diabetes Mellitus, Other)), by Vector (Viral (Retrovirus, Adenovirus, AAV, Lentivirus, Others), Non-viral (Naked DNA, Gene Gun, Electroporation, Lipofection)), by Techniques (Gene Augmentation Therapy, Gene Replacement Therapy), by Participants (Small/Medium Pharma & Biotech, Universities & Research Institutes, Hospitals, Government & Public Bodies, Big Pharma) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The gene therapy R&D market is estimated at US$3,641.7 million in 2024 and is projected to grow at a CAGR of 29.8% during the forecast period 2024-2034.

Gene Therapy: Shaping Tomorrow's Healthcare Landscape Through Innovation and Investment

Over the past five years, gene therapy has seen a remarkable resurgence fuelled by significant strides in virology, vector and capsid architecture, and manufacturing technologies. These advancements have sparked a surge in promising outcomes, attracting the attention of biotech investors and reigniting interest in the field. This renewed enthusiasm has spurred a flurry of strategic manoeuvres among biopharma firms vying for an edge in this burgeoning market.

Recent advancements in gene therapy are grounded in a deeper comprehension of viral vectors, pivotal for delivering therapeutic genes to target cells. Refinements in vector design and capsid engineering have bolstered the efficiency, specificity, and safety of gene delivery. Concurrently, progress in manufacturing processes has bolstered the scalability and consistency of gene therapy products, enabling the production of treatments for larger patient cohorts.

The market for gene and cell therapies (CGTs) is expanding rapidly, with over 1,400 companies globally focusing on CGTs and more than 3,500 therapies in preclinical and clinical development. Presently, there are over 2,000 active clinical trials, with North America leading with 964 trials, trailed by the Asia Pacific with 848, Europe with 244, and other regions with 139 active trials. Oncology and rare diseases emerge as the primary therapeutic domains targeted by CGTs, underlining the potential to address unmet medical needs and furnish innovative treatment avenues.

Investment in the CGT sector remains robust, with venture capital financing being the primary growth catalyst. The persistent inflow of capital underscores a strong belief in scientific breakthroughs and the potential to revolutionize the treatment landscape for a spectrum of diseases, encompassing both rare and common ailments.

Despite significant progress and optimism surrounding gene therapy, the field confronts several challenges, including high treatment costs, intricate regulatory requisites, and the imperative for long-term safety and efficacy data. Tackling these obstacles demands sustained collaboration among industry stakeholders, regulatory bodies, and the scientific community.

Looking ahead, the future of gene therapy holds promise. Ongoing technological advancements, coupled with substantial investment and supportive regulatory frameworks, are poised to propel further innovations and broaden the therapeutic applications of gene therapy. As the industry matures, gene therapy stands to revolutionize the treatment paradigm for a diverse array of diseases, offering newfound hope to patients who previously faced limited or no treatment options.

How has COVID-19 had a Significant Impact on the Gene Therapy R&D Market?

The COVID-19 pandemic fundamentally transformed the Gene Therapy Research and Development (R&D) market, presenting a combination of challenges and opportunities that both accelerated and impeded progress. On the positive side, the urgent demand for COVID-19 treatments and vaccines spurred technological advancements, particularly in mRNA and viral vector technologies crucial to gene therapy. This urgency drove unprecedented levels of funding, collaboration, and resource allocation, fostering innovation and expediting the translation of gene therapy concepts into clinical applications. Regulatory bodies responded by streamlining approval processes for urgent therapies, potentially benefiting future gene therapy products.

The pandemic underscored the importance of robust manufacturing and distribution networks, prompting significant investments in scaling up production capabilities for gene therapies. Conversely, the pandemic caused substantial disruptions in clinical trials, with many studies delayed or halted due to lockdowns, travel restrictions, and the prioritization of COVID-19 related research, leading to setbacks in project timelines. Supply chain interruptions also impacted the availability of critical materials and reagents, complicating R&D efforts. Despite these challenges, the pandemic highlighted gene therapy's potential to address genetic disorders and emergent viral threats, reinforcing its strategic importance. The industry's resilience and adaptability were evident in its response, suggesting that the lessons learned will strengthen future gene therapy R&D efforts.

Furthermore, the pandemic emphasized the importance of global collaboration, with international partnerships playing a crucial role in accelerating the development and distribution of new technologies. Consequently, the gene therapy market is poised for sustained growth, driven by technological advances made during the pandemic and increased recognition of the value of rapid, adaptable therapeutic development platforms. Overall, while the COVID-19 pandemic presented significant obstacles, it also served as a catalyst for innovation and structural improvements within the gene therapy R&D landscape, positioning the industry for a stronger and more agile future.

How will this Report Benefit you?

Visiongain’s 340-page report provides 156 tables and 192 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 financial analysis of the overall market and different segments including disease, vector, techniques, participants, and capture higher market share. We believe that there are strong opportunities 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 will 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?

Robust Gene Therapy Pipeline Anticipated to Boost Growth

Cell and gene therapies (CGTs) are transitioning from research labs to clinical settings, offering targeted treatments for specific diseases, unlike the broader targets of small-molecule therapies. These therapies aim to introduce healthy cells or correct genetic defects in patients, with cancer emerging as a primary focus, representing nearly half of all CGTs in clinical trials.

One promising approach is CAR-T therapy (Chimeric Antigen Receptors), which combines cell and gene therapy to combat cancer. By modifying a patient's immune cells' DNA, CAR-T therapy enhances their ability to recognize and attack cancer cells. FDA approval for two CAR-T therapies signifies their efficacy, particularly for cancer patients with limited treatment options. Continued research endeavours hold the potential to expand the availability and efficacy of these therapies across a broader spectrum of cancers.

Recent data show a notable increase in gene therapies entering Phase III trials, signalling progress and momentum in this field.

Technological Advancements Projected to Fuel Market Growth

Gene therapy, both as a cutting-edge medical technique and a burgeoning biomedical business, holds a promising future propelled by technological advancements and industry promotion. While genome editing technologies enable precise manipulation of specific genes within DNA sequences, concerns regarding off-target effects, which may inadvertently edit genes with similar sequences, pose challenges such as tumorigenicity. Despite these challenges, genome editing offers the potential for lasting genomic changes, albeit with careful consideration of safety implications.

Moreover, the market for digital bioprocessing technology is driven by the demand for reproducible, efficient, and cost-effective development of cell and gene therapies. These advancements are pivotal for realizing the commercial potential of these therapies in the coming years, enhancing market penetration, and reducing overall therapy costs. Notably, breakthroughs like the use of gold nanoparticles for gene therapy delivery by researchers at the Fred Hutchinson Cancer Research Center signify promising alternatives to conventional delivery methods, potentially revolutionizing scalability and accessibility.

CRISPR-Cas9, a transformative genome editing tool, is revolutionizing scientific research with its versatility, speed, cost-effectiveness, and accuracy compared to previous methods. Its applications span across animal research, human gene therapy, medical research, and plant science, facilitating precise gene targeting, alterations, insertions, deletions, and single base pair conversions. As breakthroughs in this field continue to unfold, the gene therapy R&D market is poised for significant growth in the forecast period.

Where are the Market Opportunities?

Artificial Intelligence Can Bring Value to Gene Therapy R&D

While fully AI-generated drug discovery hasn't yet yielded approved drugs, the number of drugs in development with AI-associated platforms is steadily increasing. In 2023 alone, at least 19 drugs attributed to AI were in clinical development, with potential advancement in the clinical pipeline anticipated for 2024.

AI holds significant potential in various stages of cell and gene therapy R&D, with McKinsey & Company identifying its greatest impact in target identification, payload design optimization, and translational and clinical development. For instance, AI and machine learning (ML) enhance target selection to optimize therapeutic success.

In viral therapeutics aiming for genome editing, algorithms predicting CRISPR target sites aid in identifying genomic locations conducive to efficient editing with minimal off-target activity. For mRNA-based vaccines, AI predicts tumour epitopes for therapeutic molecule binding. Similarly, in CAR T-cell therapies, AI facilitates antigen and binding site identification, enhancing CAR design for improved activity and reduced cytotoxicity.

AI and ML models rapidly screen numerous candidates and select designs meeting desired criteria, especially when integrated into an AI-enabled closed-loop research system. This system automatically feeds initial screening results into an ML pipeline, which learns from computational features to suggest optimized payload candidates for experimentation. Experimental data then feed back into the system, perpetuating learning and closing the research loop.

In translational and clinical development, AI and ML mitigate safety risks in clinical trials by identifying translational biomarkers indicative of success, selecting appropriate patients, estimating optimal dosing, and predicting severe adverse events based on patient profiles and real-world data from similar treatments.

Facility Expansion Anticipated to Offer Lucrative Growth Prospects

The surge in clinical-stage start-ups has led to a scarcity of viral vector manufacturing capacity among contract manufacturers, upon which new gene and cell therapy companies heavily rely for early-stage development. As these firms mature into commercial entities, they often opt for in-house manufacturing to circumvent outsourcing complexities. Consequently, biotech companies are formulating growth strategies, establishing internal teams, and seeking site consultant assistance. These experts aid in the rigorous search for suitable lab and development facilities, or, increasingly, in securing new construction sites in tight real estate markets.

These internal capabilities empower gene and cell therapy companies to swiftly scale up production from clinical batches to commercial scale, even during the research and development phase. Moreover, co-locating with drug research and development operations facilitates seamless technology transfer and minimizes disruption, especially during clinical trials.

The imperative for expedited "time-to-market" underscores the focus on existing buildings, which are increasingly scarce in established biotech hubs due to market demand. These hubs offer advantages such as tailored university programs and engagement with other gene and cell therapy companies, both as competitors and potential collaborators, creating a fertile environment for talent acquisition. While cost sensitivity is paramount for all ventures, venture-funded enterprises are particularly keen on cost reduction, necessitating efforts to minimize both upfront and ongoing cash outlays.

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, GenSight Biologics, Gilead Lifesciences, Inc., Novartis AG, Orchard Therapeutics, Oxford Biomedica, Pfizer, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Sanofi, Spark Therapeutics (Subsidiary of Roche), Takeda Pharmaceuticals, Transgene, UniQure N. V., Voyager Therapeutics, ViGeneron, GQ Bio Therapeutics GmbH, OCUGEN, INC., Taysha GTx, and Sarepta Therapeutics, Inc.. 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 launch.

Recent Developments

• On 18th April 2024, Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the U.S. Food and Drug Administration (FDA) granted fast track designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.
• On 9th April 2024, Ascend Advanced Therapies (Ascend), an end-to-end gene therapy development partner, acquired the Chemistry, Manufacturing and Controls (CMC) team and site in Alachua, Florida from Beacon Therapeutics (Beacon).
• On 18th March 2024, bluebird bio, Inc. entered into a $175 million five-year, term loan facility with Hercules Capital, Inc. (“Hercules”). The transaction strengthens the company’s balance sheet as it executes on the commercial launches for its three FDA approved gene therapies – LYFGENIA for sickle cell disease, ZYNTEGLO for beta-thalassemia and SKYSONA for cerebral adrenoleukodystrophy.

Notes for Editors

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