Impact of Therapeutic Oligos on Biotech growth in 2022
Therapeutic oligonucleotides are small size polymers like siRNA, RNAi, miRNA or ASOs with the potential to treat a wide range of diseases using a different mode of action to moderate gene expression. In 2020, the therapeutic applications segment accounted for the largest share of the oligonucleotide synthesis market. The increasing applications of oligos as therapeutic agents (such as antisense oligos (ASOs) and siRNA) used in treating neurological, infectious, and rare genetic disorders are expected to drive market growth.
As of January 2020, ten oligonucleotide drugs have received regulatory approval from the FDA, whereby in October 2020 Ionis Pharmaceuticals and Alnylam Pharmaceuticals launched 5 candidates (each) in Phase II/III. Beginning of 2021 a total number of 229 clinical trials investigating drugs (ASO/RNA-based) in oncology are registered on ClinicalTrials.gov. Of these, 195 are using ASOs as intervention, 17 trials for siRNAs, 7 trials for miRNAs, and 9 trials for aptamers. This may reflect my market investigation into creating a survey on Oligo-producing CMOs asking: “what your major projects with large companies are”, see Fig. 1. Participating in 2022 in the RNA leader conference and Biologics UK there is still a strong focus of Big Pharma on therapeutic ASOs, with all their disadvantages applying to humans. The newly investigated siRNA, RNAi, miRNA or sgRNA technologies by young Therapeutic Biotech companies cover the lack of knowledge or investigation, that’s why Big Pharma is strongly looking for tech transfer (Eli Lilly, AstraZeneca, Sanofi, and Roche to in-license RNA-based candidate for several hundred million dollars per API).
Fig. 1: Business model for Oligo-producing CMOs. Key requirements are lead time, GMP capability, pricing, and R&D capacity for new chemical modalities.
Venture capital has discovered these companies and is driving the market, I found just in America, which is the major market, 200 companies were investigating RNA-based therapeutic technologies. Besides BioNTech and Moderna, emerging Therapeutic Biotech companies like Regulus Therapeutics, MiNA Therapeutics, Stoke Therapeutics, Sarepta Therapeutics and Silence Therapeutics collect immense funding to develop their innovative RNA-based drug platforms and established their pipeline.
Therapeutic CMO Market Scale (ASOs, siRNA, RNAi, RNAa)
Tab.1: Project types and sizes
Customer Characteristics:
R&D discovery projects of Therapeutic Companies (lead time, high purity and complexity are not required)
GMP Outsourcing by Big Pharma & Therapeutic Biotech (high purity and supply chain are required)
Unique: new chemical developments (incl. IP rights)
Project timelines in Therapeutics
Fig. 2: Time lines in the CDMO production process
Fig. 3: A timeline of RNA-based technological advances, drug approvals and other important events are highlighted. Rapid development in the RNA-targeting field has been applied to treat rare and common diseases. ASOs antisense oligonucleotides, RNAi RNA interference, RISC RNA-induced silencing complex, siRNA small interfering RNA, saRNA small activating RNAs, ssRNA single-stranded RNA. (Cell Death & Disease (Cell Death Dis) ISSN 2041-4889 (online))