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Ben Johnson

More Than 10 Approved Gene Therapies and Over 465 Product Candidates are Being Evaluated for the Treatment of a Variety of Disease Indications

Currently, there are 11 gene therapies that have already received approval / conditional approval. In addition, there are more than 20 molecules in the advanced stages of clinical development (phase II/III and above) that are anticipated to soon enter the market. Further, we came across 213 gene therapy products that were either approved or are being developed in clinical stages and over 271 early stage (preclinical / discovery) candidates that are being investigated for various disorders. It is worth mentioning that more than two third of the gene therapies are in the early stages of clinical development (phase I/II and phase I), indicating that this domain has significant opportunity in the mid-long term.



Most of the pipeline drugs (36%) use AAV vectors as their preferred delivery vehicle. This can be attributed to the fact that these vectors offer various advantages, such as high titer, mild immunogenic response, the ability to infect a wide spectrum of cells and safety over other potential vectors. Other key vectors being used for the development of gene therapies include (in decreasing order of their popularity) non-viral vectors (20%), adenovirus vectors (18%) and lentivirus vectors (13%). In addition, 33% of the clinical candidates are being developed to target various oncological disorders. Unlike the clinical pipeline, within the preclinical / discovery pipeline, metabolic disorders (19%) have emerged as the most prominent therapeutic area.

In terms of modification, over 50% of the gene therapy candidates are modified through gene augmentation. This technique involves the addition of a functional copy of the gene in order to produce the missing proteins. Further, close to one-fourth of the therapies are immunotherapies, while about 17% of the candidates are oncolytic therapies. It is worth noting that immunotherapies are the therapies that stimulate the immune system to attack various disease-causing agents. On the other hand, oncolytic therapies use genetically modified viruses that selectively target and kill cancer cells. In case of gene therapies, these viruses act as vehicles to deliver the therapeutic agent to the cancer site.

In terms of route of administration, the intravenous route emerged as the most popular mode of injection for gene therapies; close to 30% therapy candidates are currently being developed to be delivered through this route of administration. Other prominent approaches being adopted by developers for the delivery of gene therapies include subretinal (11%), intramuscular (11%) and intratumoral (9%). It is important to mention that the subretinal route of administration is primarily adopted by gene therapies intended for the treatment of ophthalmic disorders.



Over the past few years, big pharmaceutical players, such as Amgen, Novartis and Pfizer, have either launched their gene therapy products or have advanced their clinical candidates towards later stages of development; however, the current market landscape is led by start-ups and mid-sized companies that have multiple gene therapy candidates in their pipeline.

Majority (57%) of the companies in this domain are small-sized firms (less than 50 employees); examples of such players include (in alphabetical order, established in 2017 and 2018) Actus Therapeutics, Apic Bio, Aruvant Sciences, Lacerta Therapeutics, Neurogene, Passage Bio, Pattern BioSciences, Prevail Therapeutics, Urovant Sciences, Verve Therapeutics and Wyvern Pharmaceuticals. In terms of geography, 63% of the companies developing gene therapies are headquartered in North America; of these, majority are based in the US. This is followed by companies (43) located in Europe. 

https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html