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2024-05-06| ASGCT 2024

Exploring Key Areas of RNA Therapeutics Development: Your Blueprint for Maximizing ASGCT Content

by Bernice Lottering
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This preview provides key information surrounding the advancements in RNA therapeutics, inclusive of various targeted treatment areas within the RNA pipeline. Moreover, rare diseases and current clinical trial activities shed light on the current playing field in the context of RNA targeted therapies. Additionally, the financial trends in gene, cell, and RNA therapeutics provide insights into industry trajectories as the significant acquisitions made in Q1 2024 are highlighted and the evolving landscape of entrepreneurial financing within the industry is scrutinised.

Exploring RNA Therapeutics Development: Advancements, Targeted Therapeutic Areas, Rare Diseases and Clinical Trial Activities

Analysis from ASGCT and Citeline reveals that, within the RNA therapeutics pipeline, messenger RNA (mRNA) and RNA interference (RNAi) remain the preferred modalities under investigation, showcasing the continued exploration and advancement of RNA-based treatments. Here we see increasing numbers of RNAi, mRNA, and antisense oligonucleotides progressing from preclinical to clinical stages. The majority of these therapeutics are still in the preclinical stage, with RNAi accounting for 73%, mRNA for 67%, and antisense oligonucleotides for 71% of their respective pipelines. 

Among the 1,072 RNA therapeutics currently in preclinical to preregistration stages, rare diseases remain the most common therapeutic area targeted, whilst non-oncology indications continue to be the most frequently targeted, accounting for 80% of the majority. Furthermore, the most commonly targeted rare neoplastic diseases include pancreatic, liver, and ovarian cancers. For non-neoplastic rare diseases, conditions such as Duchenne muscular dystrophy, amyotrophic lateral sclerosis, and Huntington’s disease are frequently focused on. 

In Q1 2024, 30 RNA trials were launched, with 90% targeting non-oncology indicators. This surge in clinical trial activities signifies the growing interest and investment in advancing RNA therapeutics towards clinical development and potential commercialization. 

Examining Financial Trends, Acquisitions, and Entrepreneurial Financing in RNA Therapeutics

In this regard, alliances, acquisitions, and financing activities continue to shape the landscape of gene, cell, and RNA therapeutics. Advanced molecules witnessed a 34% increase in deal volume in Q1 2024 compared to the previous quarter, marking the highest recorded number of deals in the past year. This reflects a growing interest from investors and industry players in the potential of advanced therapeutics. 

Then, the acquisition volume trended downward in Q1 2024, with the total deal volume being the lowest in the past year. Notable acquisitions include Novo Nordisk’s acquisition of Cardior, strengthening its cardiovascular pipeline, and Regeneron’s creation of Regeneron Cell Medicines focusing on nocel immune cell treatments and developments. Further, startup financing in gene, cell, and RNA therapeutics companies continues to decline, with both the number of deals and total value of Seed and Series A rounds decreasing in Q1 2024 compared to the previous quarter and the same quarter in 2023. This highlights the challenges faced by early-stage companies in securing funding, potentially impacting innovation and development efforts in the field.

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