Gene therapy can be described as the transfer of genetic material mostly in a vector or carrier while cell therapy includes the transfer of cells with the applicable function into the patient’s body. In the broader sense, gene therapy involves introduction, withdrawal, or modification in genetic code with an aim of curing or treating a disease. Gene therapy can be utilized to minimize the level of a disease-causing form of a protein and enhance the production of disease-fighting proteins, or to produce modified proteins. Cell therapy is the transfer of live, intact cells into a patient’s body in order to cure or treat a disease. The introduced cells can be from patient’s own body or from the donor. The cells utilized in cell therapy can be categorized by their possibilities to transform into different cell types. Cell and gene therapies are widely used to treat severe diseases such as cancer as well as rare diseases such as sickle cell disease, muscular dystrophy, and progeria.
The U.K. cell and gene therapy research challenges market is estimated to be valued at US$ 848 million in 2021 and is expected to exhibit a CAGR of 23.6% over the forecast period (2021-2028).
Figure 1. U.K. Cell and Gene Therapy Research Challenges Market Share (%), By Therapy Type, 2021
The increasing product launches and approvals are expected to drive the market growth over the forecast period.
For instance, in October 2018, Bluebird bio, Inc. a biotechnology company, received European Medicines Agency (EMA) acceptance for the company’s authorization application (MAA) for its investigational ‘LentiGlobin gene therapy’. This therapy is used for treating adults and adolescents suffering from transfusion-dependent β-thalassemia (TDT). LentiGlobin gene therapy being studied as a possible treatment to address the significant genetic cause of TDT which may reduce or eliminate the requirement for blood transfusions.
| Report Coverage | Details | ||
|---|---|---|---|
| Base Year: | 2020 | Market Size in 2021: | US$ 848 Mn |
| Historical Data for: | 2017 to 2020 | Forecast Period: | 2021 to 2028 |
| Forecast Period 2021 to 2028 CAGR: | 23.6% | 2028 Value Projection: | US$ 2525 Mn |
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| Companies covered: |
Novartis International AG, Pfizer Inc., Sanofi S.A., Amgen, Inc., PTC Therapeutics, Inc., Orchard Therapeutics Plc., Biomarin Pharmaceutical Inc., Regeneron, Bluebird Bio, Inc. (Celgene Corporation), Freeline Therapeutics Ltd, MeiraGTx Limited, Kolon TissueGene, Inc., Kite Pharma, Inc. (Gilead Sciences, Inc.), Biogen INC, and Bristol-Myers Squibb Company |
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Figure 2. U.K. Cell and Gene Therapy Research Challenges Market Share (%), By Indication, 2021
The increasing adoption of inorganic growth strategies such as mergers, collaborations, partnerships, and acquisitions by key players in order to strengthen their position in the U.K. market is expected to drive the market growth over the forecast period.
For instance, in March 2019, Biogen Inc., pioneers in neuroscience entered into an agreement to acquire Nightstar Therapeutics, a clinical-stage gene therapy company, to set up a clinical pipeline of gene therapy candidates in the field of ophthalmology.
U.K. Cell and Gene Therapy Research Challenges Market – Impact of Coronavirus (COVID-19) Pandemic
The COVID-19 pandemic and lockdown across the globe has negatively impacted the financial status of businesses across all sectors. The COVID-19 pandemic has impacted the entire supply chain of the healthcare industry mainly due to strict lockdown in several regions. Likewise, COVID-19 pandemic has made significant impact on the UK cell and gene therapy market.
The cell and gene therapy development activities had slowed down in pandemic as companies had switched focus to vaccine production. The regulatory guidelines for advanced therapy medicinal products (ATMPs) is complex, with complexities in reimbursement environment, regulatory regime, and supply chain. There are issues in conduction of trials for small patient population suffering through rare diseases. These complications have increased due to COVID-19 pandemic.
U.K. Cell and Gene Therapy Research Challenges Market - Restraint
Unfavourable reimbursement policies and manufacturing issues limiting the availability of products at treatment centres are expected to hinder growth of the U.K. cell and gene therapy research challenges market over the forecast period. However, cell and gene therapy has witnessed multiple setbacks from regulatory authorities in charge of reimbursement policy, which is expected to inhibit growth of the market.
For instance, in August 2018, the National Institute for Health and Care Excellence (NICE), U.K. concluded that Yescarta is a very expensive therapy and hence cannot be considered as a cost-effective use of National Health System (NHS) resources.
Key Players
Major players operating in the U.K. cell and gene therapy research challenges market include Novartis International AG, Pfizer, Inc., Sanofi S.A., Amgen, Inc., PTC Therapeutics, Inc., Orchard Therapeutics Plc., Biomarin Pharmaceutical Inc., Regeneron, Bluebird Bio, Inc. (Celgene Corporation), Freeline Therapeutics Ltd, MeiraGTx Limited, Kolon TissueGene, Inc., Kite Pharma, Inc. (Gilead Sciences, Inc.), Biogen INC., and Bristol-Myers Squibb Company.
Cell therapy can be defined as the transfer of new cells into a patient’s body to repair or replace damaged tissue or cells to treat a disease. The introduced cells can be from patient’s own body or from the donor. Different types of cells can be utilized in cell therapy such as lymphocytes, pancreatic islet cells, stem cells, and dendritic cells. This technology of cell therapy enables the development of various types of specific human cells required for therapeutic purposes. While gene therapy involves introduction or modification of genes into a patient’s body with an objective of providing treatment, prevention or to cure a disease.
The gene therapy alters a person’s genes to cure or treat disease. This process involves various mechanisms such as introduction of modified or new gene into the patient’s body in order to treat a disease, replacement of a disease causative gene by a healthy gene copy, or inactivation of a disease causing gene which exhibits improper functioning. Gene and cell therapies may provide long lasting impact than traditional medicines.
Market Dynamics
Rising research and development activities for the development of novel technology, increasing approvals and launches of novel gene therapies for treatment of various rare diseases and robust pipeline of products which are expected to gain approval over the forecast period. Additionally, increasing adoption of inorganic growth strategies such as mergers, acquisitions, partnerships, and collaborations by key players operating in the market are the major factors that are expected to drive growth of the U.K. cell and gene therapy research challenges market over the forecast period.
For instance, in August 2018, Novartis International AG, a global healthcare company, received European Commission’s (EC) approval for ‘Kymriah’. It is approved for the indication of treatment of patients up to 25 years of age with refractory or relapse B-cell acute lymphoblastic leukemia and for treating adult patients with refractory or relapsed diffuse large B-cell lymphoma after two or more lines of systemic therapy.
Key features of the study:
Detailed Segmentation:
“*” marked represents similar segmentation in other categories in the respective section.
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