Delivery Systems

Delivery and targeting are still seen as the biggest growth area for technology need or advancement in the sector. Now scale of manufacturing is third, a higher priority than it was in 2019.

This is followed by mRNA and siRNA, as RNA therapeutics have seen a boom in interest and demand since the beginning of the COVID-19 pandemic.

What strategy/ies for macromolecular drug delivery do you use?

Naked systems with lipophilic conjugates were the most common form of local delivery for macromolecular drugs, used by 46% of respondents, showing a gain in popularity than in 2019 when they were in second place. This year’s survey found local systems formulated in LNPs as second most common. Naked delivery systems without lipophilic conjugates have decreased in popularity since they were found to be the most common in 2019.

Of the respondents who said they were using systemic delivery systems for macromolecular drug delivery, half said they were using systems formulated in LNPs, while just over a third of respondents said they had systems formulated in other types of particles. This reveals a change since 2019 when naked systems with GalNac conjugates were found to be the most common form of systemic delivery.

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Therapeutic Advancements

Effective delivery is again the top-suggested major advancement for oligonucleotides over the next 5 years, as it was in our 2019 survey. Similarly, in relation to genome editing, the most common suggestion was a more mature clinical pipeline, the same top answer found relating to CRISPR therapeutics in 2019.

In the next 5 years, what major advancements do you think the following fields will see?

Oligonucleotides

Top Response: "Effective Delivery - Targeted, Systemic,etc."

"Promising treatments for neurodegenerative diseases"

"More organ specific targeting"

"Introduction of linked complex oligos"

"More approved drugs"

"This area will be more explored for niche diseased"

"Personalized treatment using a platform approach"

Peptides

Top Response: "Oral Delivery"

"Liq/Liq manufacturing platforms overtaking Solid-Liquid phase"

"Improved ADCs and T-cell antigen stimulation"

"First approval of therapeutic peptide vaccine"

"Advancement of green chemistry, more efficient processes, more conversion to innovative new processes"

"Broader applications beyond endocrine diseases, further elucidating rules for intracellular delivery"

As the next biggest advancement for peptides, the most common prediction was oral delivery systems and, perhaps not surprisingly, further uses in vaccines was the most predicted major advancement for mRNA therapeutics.

mRNA therapeutics

Top Response: "Uses in Vaccines"

"Developing orally mRNA"

"Drug targeting"

"Delivery challenges and CMC challenges will be solved"

"More commercial medicines"

"Cost & supply improvements"

"Manufacturing process scalability and analytical"

Genome editing

Top Response: "Clinical pipeline maturity"

"Delivery and more accurate editing"

"Off-target tox events will be clear"

"First regulatory approved treatment"

"Technologies other than CRISPR enter the clinic; ways to make multiple edits safely; strategies for gene insertion & gene replacement"

"Cost reduction by improving manufacturing procedure"

"Neonatal lethals"