2.1.1 There are many types of naturally occurring RNA, so it naturally follows that there are many types of therapeutic modalities in development. To undertake a survey of clinical trials, it is helpful to first review the modalities in development. A few common modalities are summarized as follows (see also Table 1).
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2.2.1 Since the emergency use authorization (EUA) of mRNA-based COVID-19 vaccines, many other mRNA-based candidates have rapidly progressed through the clinic. As of July 28, 2023, 316 RNA treatments were actively being investigated by 190 companies, according to Clarivate’s Cortellis Competitive Intelligence Database, with 57% at the discovery and preclinical stage, 39% undergoing clinical study, 1.9% at the pre-registration stage, and 1.6% having received approvals. Of the 125 candidates in the clinic, approximately two-thirds were vaccines and one‑third therapeutics. Most of the later-stage candidates and products are COVID-19 vaccines.
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2.3.1 Since the first patent was granted for an RNA in 1970, there has been a steady increase in patents granted, culminating in a record 11,077 patents related to therapeutic RNA in 2020 (Figure 1). The number of patents continues to rise as well at an average of 2.5% year-over-year (YoY) from 2010 to 2023. Interestingly, the number of patents granted during the pandemic decreased. This is reflective of a decrease in the overall number of patents being granted and not specific to RNA-based therapeutics. If one discards the effects of the pandemic and considers only the period from 2013 to 2020, the YoY growth rate was 5.9%. Key topics include delivery technologies and mRNA design (conventional, self-amplifying, circular, double-stranded), but stability, immunogenicity, translation efficiency, and targeted delivery also appear in many of the applications.[40]
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2.4.1 The first FDA-approved RNA therapies were landmark modalities in 1998 and 2004 but have often been forgotten in the RNA therapeutic story. Fomivirsen (brand name Vitravene) is an antisense RNA indicated for CMV infections in immunocompromised patients, especially those with HIV.[44] It was later removed from the market by the FDA when effective antivirals became more widely used to control the infection. Pegaptanib was a treatment for neovascular (wet) age-related macular degeneration. Pegaptanib (brand name Macugen) was approved in December 2004. It is an RNA aptamer that blocks vascular endothelial growth factor (VEGF), a now well-established target for the disease. Sales of the therapy peaked in 2010 but declined after other antibody-based treatments were approved for the same indication. It has since been withdrawn from the market by the manufacturer.[45]
2.4.2 As of February 2024, there are 13 FDA-approved RNA therapies on the market.
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2.5.1 GlobalData estimates 747 RNA-based modalities are in preclinical development, with another 477 in the discovery phase. Throughout the pipeline, mRNA modalities represent approximately 50% of the RNA-based therapies. As reported above, there are 273 active trials investigating both infectious (Figure 10) and noninfectious (Figure 18) diseases. This represents over 60 different therapeutics. The drugs in phase III trials are listed in Table 4 along with global sales forecasts from GlobalData. The relatively large number of therapeutics that are nearing phase III completion, the breadth of their modalities and indications, and the sales forecasts all indicate that the RNA market will remain healthy and continue to spur investment into the foreseeable future.
2.5.2 Very few studies have been undertaken to study long-term effects, which are especially of interest for genetic therapies. In the coming decade we can expect a rise in phase IV trials for the curative treatments that are currently in the clinic or recently approved. The data from long-term effects will likely influence a new generation of therapies yet to be developed.
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