Gene Therapy Market Overview The current market landscape of gene therapy market features over 1,150 gene therapy candidates being investigated by more than 250 developers across different stages of development for the treatment of a myriad of disease indications; till date, 11 gene therapies have been commercialized.

The gene therapy market is estimated to be worth $2.6 billion in 2023 and is expected to grow at compounded annual growth rate (CAGR) of 18.0% during the forecast period. It is worth mentioning that three gene therapies received approval in 2020 across different regions; these include (in reverse chronological order of the month of approval) Luxturna (in Canada; developed by Sparks Therapeutics) Libmeldy (in Europe; developed by Orchard Therapeutics) and Zolgensma (in Japan; developed by Novartis).

Most of the therapies (70%) are currently in the early stages of development (preclinical / discovery), indicating that this domain has significant opportunity in the mid-long term. The growing pipeline and the increasing demand for effective treatment using gene therapies, has spurred the establishment of many companies in the last decades.

Gene Therapy Market Segmentation

Gene Therapy Market includes various segments :Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions. Further is worth mentioning most of the companies engaged in the development of gene therapies were established post 2015 (42%). In fact, more than 40 gene therapy developers have been established, during the period 2019-2021.

Notable examples of companies established in 2021 include (in alphabetical order) AAVantgarde Bio, Axovia Therapeutics, Nuntius Therapeutics and Opus Genetics. Also, the gene therapy market is currently dominated by the presence of small players (60%), followed by mid-sized firms (30%). Examples of well-established players in this domain include (firms established prior to 1990, more than 5,000 employees) Allergan, Amgen, Astellas Pharma, Bayer, Biogen, Boehringer Ingelheim, CSL Behring, Eli Lilly, Intas Pharmaceuticals, Janssen Pharmaceuticals, Pfizer, Roche, Takeda Pharmaceutical and UCB.There are about 339 gene therapy products that have either been approved or being developed in clinical stages for the treatment of various diseases.

Moreover, 53% of the gene therapies currently use AAV vectors as a preferred delivery vehicle. In addition, 65% of such therapies are presently modified using the gene augmentation approach, followed by those based on oncolytic immunotherapy (18%). It is worth mentioning that oncolytic therapies use genetically modified viruses that are capable of selectively targeting and killing cancer cells. In case of gene therapies, these viruses act as vehicles responsible for delivering the therapeutic agent to the cancer site. Moreover, most of the gene therapies have received orphan drug designation (168), followed by those awarded with fast track designation (70), and rare pediatric disease designation (59).

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