Exploring the Possibilities of RNA Therapeutics

Fuelled by the successful utilisation of the mRNA COVID-19 vaccines, RNA therapeutics are expected to make larger strides in the near future. Let’s look at some of the drugs in late stage trials and the trends that will shape the RNA therapies landscape in 2022 and beyond:

- Ayesha Siddiqui

The success of the mRNA vaccine against COVID-19 has skyrocketed the development of RNA therapies. In 2021, more than 600 therapies were in development (from preclinical to pre-registration stage), with 73 per cent of therapies in preclinical development; 20 therapies were in Phase III clinical studies (including COVID-19 vaccine). Trials evaluating vaccines for COVID-19 infections predominated but there are also trials initiated for dyslipidemia and hepatitis B infection. The top therapeutic areas being targeted by RNA therapeutics are for rare diseases and oncology, according to a report from Informa Pharma Intelligence.

‘‘The biotechnology industry is in agreement as to the power and benefit of RNA therapeutics. There has been a lot of work done in delivering RNA within various nanoparticles directly into the body. However there’s an untapped, and potentially larger, opportunity in using the cell (instead of a nanoparticle) as the vehicle to deliver, and the factory to produce, a combination of RNA therapeutics right at the site of disease: that’s the power of RNA cell therapy. In contrast to conventional nanoparticle-based RNA therapies, RNA cell therapy may have minimal immunogenicity with repeat dosing. It can also be easily engineered with tissue-specific homing proteins. Compared to conventional DNA-based cell therapies, RNA cell therapy has a predictable half-life and avoids the risk of genomic integration: two attributes that are expected to make it safer. RNA Cell Therapy is also less expensive and enables true combination therapy without vector cargo limits,’’ said Dr Murat Kalayoglu, President and CEO of Cartesian Therapeutics, USA.