Currently, 30 technology developers are presently engaged in the development of intracellular drug delivery technologies.
There has been a significant increase in the number of players in this domain post 2001. Over 45% companies were established post 2010, which clearly highlights the growing interest of stakeholders in this domain. Examples of companies which have been established in the past three years include (in alphabetical order) Genevant (2018), Pantherna Therapeutics (2017) and pHion Therapeutics (2017).
Targeting the delivery of a pharmacological substance to its intended physiological site of action ensures that adequate concentration of the therapeutic is made available to elicit the desired clinical benefit and limit off-target / systemic toxicity. Advances in cell biology and molecular probing techniques have revealed the presence of several potential therapeutic targets for a number of difficult-to-treat diseases, localized within the confines of the cell membrane. Therefore, efficient methods of facilitating intracellular drug delivery to various sub-cellular compartments are a prominent need within the modern biopharmaceutical industry.
Need for Targeted Drug Delivery
Considering the fact that many modern pharmacological interventions are being designed against specific biological targets, there is an evident need for robust drug delivery technologies. Some of the key advantages offered by targeted drug delivery systems / devices are summarized below:
- Reduction of Systemic Toxicity and Side Effects: Targeted drug delivery is particularly beneficial in the case of highly potent drugs, which include the chemotherapy drugs and biologics (such as cell therapies, gene therapies and immunotherapies), making it possible to altogether avoid off-target toxicity related concerns
Majority of the developers are based in Europe (47%), with six companies headquartered in the UK. Examples of such companies include (in alphabetical order) Evox Therapeutics, pHion Therapeutics and Silence Therapeutics. Examples of companies based in other nations in Europe include (in alphabetical order) BioCellChallenge (France), CureVac (Germany) and PCI Biotech (Norway).
For instance, there are 13 intracellular drug delivery-focused companies located in North America (43%); of these, 10 are headquartered in the US, while 3 are based in Canada. Examples of the companies based in North America include (in alphabetical order) Acuitas Therapeutics (Canada), Arcturus Therapeutics (US), Codiak BioSciences (US), and SRI International (US).
Majority of the technologies are designed for delivery of biologics (60%). Due to the large and complex structure of biopharmaceuticals (such as RNA, DNA, proteins, and antibodies), such interventions usually require facilitated entry into the cell. It is worth highlighting that nearly 37% technologies enable cell internalization of both small molecules and biologics. Examples of such technologies include (in alphabetical order) CriPec technology platform (by Cristal Therapeutics), DeliverEX platform (by Evox Therapeutics), engEx Platform (by Codaik BioSciences), Ligand Conjugated Antisense (LICA) technology (by Ionis Pharmaceuticals) and QTsome technology (by Zhejiang Haichang Biotechnology).
26 technologies are available for the delivery of RNA. Examples of companies offering such technologies include (in alphabetical order) AOC platform (by Avidity Biosciences), Eligen (by Emisphere technologies), and GalNAc-siRNA Platform technology (by Silence Therapeutics).
Intracellular drug delivery technologies use vehicles that encapsulate (52%) the drug payloads and facilitate them into the cytosol of the cell. Examples of technologies which utilize encapsulation includes (in alphabetical order) DeliverEX platform (Evox Therapeutics), Exosome Platform (ReNeuron), LUNAR (Arcturus Therapeutics) and SNIM RNA platform (ethris).
Majority of the intracellular drug delivery technologies (67%) use nanoparticles for delivering the payloads directly to the target cell type. It is worth highlighting that 13 technologies have deployed lipid nanoparticles as vehicles in their intracellular delivery platforms. Examples of such technologies include (in alphabetical order) DNAbilize (by Bio-Path Holdings), Fusogenix Platform (by Entos Pharmaceuticals) and MRT platform (by Translate Bio).
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