Gene Therapy: Propelling the Future of Personalized Medicine
Roots Analysis
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According to the World Health Organization (WHO), 10 out of every 1,000 individuals are affected by genetic disorders, with over 70 million people worldwide living with such conditions. In fact, more than 40% of infant mortality globally is associated with various genetic disorders. In order to address these challenges, treatment strategies targeting the genetic cause of the aforementioned disorders has become a vital need. In this regard, gene therapies have emerged as a revolutionary approach and have proven to be a promising modality in treating a wide array of diseases.
According to the USFDA, gene therapy is defined as a novel technique of administering genetic material in order to manipulate or modify the expression of either disease-causing or suppressor genes. These therapies are used to replace a dysfunctional gene within the genome of a cell / host organism and allow genetic level changes to be made in order to eliminate the root cause of any inherited disorder. To facilitate the transfer of a therapeutic gene into the suitable cell / organ, several delivery vehicles, called vectors, have been designed; one of the most common types of carriers is viral vectors. Further, gene therapies can be administered within (in vivo) or outside (ex vivo) the body of a host. In the case of in vivo therapies, the therapeutic gene is directly delivered into the target cell within specific tissues (such as liver, skin, spleen, lung, brain, muscle and blood) inside the patient’s body. Alternatively, ex vivo therapies involve the modification of the target gene segment (within a cell / group of cells) outside the body. In this case, allogeneic (derived from donor organisms of the same species) or autologous (derived from the patient’s own body) cells are isolated and transduced / transfected with an appropriate viral / non-viral vector, which is expected to carry the therapeutic gene. These modified cells are then transferred back into the patient’s body in order to achieve the desired therapeutic response.
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Gene Therapy Market
According to Roots Analysis, the Gene therapy Market is estimated to be USD 2.4 billion in 2024 and is anticipated to grow at a CAGR of around 19.25% till 2035. In terms of type of vector, adeno-associated virus vectors are expected to capture the majority share in the gene therapy market by 2035. Additionally, in terms of therapeutic area, muscle disorders currently hold the largest share (72%) of the market. Further, amongst types of gene delivery method, the market for in vivo gene delivery method is anticipated to represent the highest share of the gene therapy market.
Gene Therapy Market Landscape
The gene therapy market landscape features more than 1,310 therapies that are either marketed or currently being evaluated in clinical trials / preclinical studies or are in the discovery stages of development. Close to 80% of the marketed and clinical stage gene therapies have received orphan drug designation. Additionally, more than 55% of the gene therapies are gene augmentation therapies that are designed to deliver a healthy version of the mutated gene into the target cell / organ of the body. Further, close to 95% of the preclinical and discovery stage gene therapies are being developed to be delivered using in vivo methods of gene delivery; notable examples (preclinical stage gene therapies, arranged in alphabetical order) include 4D-725 (4D Molecular Therapeutics), ABO-504 (Abeona Therapeutics), KT430 (Astellas Pharma and Kate Therapeutics) and KRIYA-825 (Kriya Therapeutics).
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Market Share Analysis: Adeno-associated Virus Vectors are the Most Widely Used Vectors for Delivery of Gene Therapies
Over the years, adeno-associated virus vectors have emerged as the most preferred vector for the delivery of therapeutic genes. This can be attributed to the fact that adeno-associated virus vectors serve as the most effective delivery vehicle for transfer of gene therapies. Over time these viral vectors have emerged as a promising tool owing to various advantages such as versatile tissue tropism, non-pathogenicity, and ability to produce long-term gene expression in the target tissues. Moreover, these viral vectors have a low immunogenicity profile which reduces the likelihood of unwanted immune responses, ensuring safe administration in patients. Notably, close to 45% of the gene therapies are delivered using adeno-associated virus vectors. Examples of marketed gene therapies delivered using adeno-associated virus vectors (arranged in alphabetical order) include BEQVEZ? (Pfizer), HEMGENIX? (CSL Behring), LUXTURNA? (Spark Therapeutics and Novartis) and UPSTAZA? (PTC Therapeutics).
Demand Analysis: Estimates of the Annual Demand for Gene Therapies
The demand for gene therapies is projected to reach 79,144 patients in 2035, growing at a CAGR of 11.67%. In addition, the commercial and clinical demand for gene therapies is projected to reach over 44,088 patients and 35,056 patients, respectively, in 2035. Further, in terms of type of gene delivery method, the current demand is driven by ex vivo delivery method, however in the future the demand for in vivo gene delivery is likely to grow at a faster rate.
Leading Companies in Gene Therapy Market
Examples of key companies engaged in the gene therapy domain (which have also been profiled in this market report; arranged in alphabetical order) include Amgen, Artgen Biotech, BioMarin Pharmaceutical, bluebird bio, CRISPR Therapeutics, CSL Behring, Ferring Pharmaceuticals, Kolon TissueGene, Krystal Biotech, Novartis, Orchard Therapeutics, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Shanghai Sunway Biotech, Sibiono GeneTech and Spark Therapeutics. This market report includes an easily searchable excel database of all the pharmaceutical companies engaged in the development of gene therapies for a myriad of chronic conditions.
All your Questions Related to Gene Therapy Market Answered in this Report
The Gene Therapy Market report features an extensive study of the current market landscape, market size and future opportunities for gene therapies, during the given forecast period. Further, the report highlights the efforts of several stakeholders engaged in this rapidly emerging segment of the pharmaceutical industry. The report answers many key questions related to this market. Click on the below link to know more about this emerging area of research https://www.rootsanalysis.com/reports/gene-therapies-market/268.html
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