How are the oligonucleotides and peptides markets shifting? What are the biggest challenges that currently need to be overcome? What impact will the emergence of mRNA therapeutics have? What can we expect to see in the near future? We asked two experts in the field for their insights on these questions, trends and discoveries. Here's what they had to say.
Renata Szyszka, PhD, Owner, Entrepreneurship & Innovation for Leber's Congenital Amaurosis (E&ILCA)
Where do you see as potential opportunities and challenges for the therapeutic oligos field over the next five years?
RS: Opportunities start with the patient. Patient-industry partnerships provide an excellent opportunity for both sides to learn about the history of the disease. They provide a required context for the drug long-term performance. Future modifications and necessary improvements are based on patient positive experiences. Patient-industry close collaboration and patients’ involvement throughout the entire new drug development process help determine the new drug efficacy and safety.
The positive benefits that outweigh potential risks become a drive for the expansion of therapeutics from small, rare disease populations into large non-orphan cohorts.
Challenges for AONs or any other new therapeutic will be associated with multiple-site, international clinical trials, regulatory harmonization and potential discrepancies between EMA, FDA, and Japanese regulatory authorities.
To dive down from a high-level overview into specifics of the AON field, one might pay close attention to the stereochemical control of AON molecules, their enhanced stability associated with the types of chirality on the AON backbone, and their target specificity. AON editing capabilities cover new areas of therapeutic unmet needs.
Challenging may be AON supply after the molecules obtain access to the market and will be in demand in multiple regions across the globe. When moved from the rare disease patients counted in hundreds or couple of thousands of patients towards large scale of more common diseases, the supply and manufacturing may certainly require expansion of knowledge and young, vibrant, and fresh expertise.
Dr. Marc M. Lemaitre, PhD, Principal, ML_consulting and Member of the Board of Directors and Treasurer, Oligonucleotide Therapeutics Society
What impact do you think the emergence of mRNA therapeutics will have on the industry?
ML: I personally think that emergence of mRNA will not have a significant impact on the oligo and peptide industry, at least in the short- and mid-term because there are quite some challenges - delivery is one, stability and duration of expression is another - that need to be resolved. After that, it is quite speculative.
Do you see any major industry shifts or particular growth areas in the oligonucleotide therapeutic market?
ML: I do not see any real shift in the oligonucleotide therapeutic market anytime soon.
The main players in terms of developers of oligonucleotide therapeutics will still be Ionis, Alnylam, Wave, Arrowhead, Dicerna, miRagen, Biogen, Sanofi Genzyme, Roche and a few others in the near future. Depending on the approval of more products currently in Phase III we may see a couple of Big Pharma entering more publicly.
As for the CMO market, I do not see any big new player coming in the foreseeable future.
Technologically speaking, I also do not see much coming in the immediate future. A delivery method besides GalNAc that may allow targeting to other organs or body parts as well as solid tumors will be very welcome. Avidity is an interesting company to follow.
The growth of the market will all be driven and is 100% dependent on the success of more projects in phase III. Projects like Geron/Janssen’s Imetelstat are important as it would be an important first oligonucleotide success against cancer.
CRISPR technology is still far from a real big project in clinical development I think and mRNA therapeutics are completely dependent on the ability for the developers to come with good, efficacious delivery systems in patients. In my opinion, it will take at least another five years before we have a clear view on the future of those technologies.
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