Background of Nanocarrier-Based Delivery Therapy for LSDs
Lysosomal storage disorders (LSDs) are a group of rare life-threatening diseases caused by lysosomal dysfunction, usually due to the accumulation of undegraded substrates as a result of the deficiency or dysfunction of a single enzyme required to metabolize macromolecules such as lipids, glycoproteins and mucopolysaccharides. Multiple therapies for LSDs are currently approved, but their effectiveness is limited. Advances in nanotechnology for biomedical applications have facilitated the development of targeted drug delivery. In this system, a biomolecule or drug of interest is affixed, complexed, or encapsulated (incorporated) into a nanocarrier and delivered to the site of action by active or passive mechanisms. This process could open the door to a plethora of alternative strategies for drug transport and delivery, overcoming the constraints of traditional drug administration. Scientists have devised several types of nanovacule-based delivery technologies to translocate therapeutic agents to the central nervous system for the treatment of LSDs.
Fig. 1. Liposomal formulations for treating lysosomal storage disorders. (Tomsen-Melero J, et al., 2022)
Solutions
Enzyme replacement therapies (ERT) for LSDs are unable to effectively deliver recombinant lysosomal enzymes to all organs affected in LSDs. One strategy to overcome some of the limitations of current ERT is to encapsulate the recombinant enzyme into a nanocarrier. CD BioSciences is dedicated to the preclinical development of biomaterials and nanotechnology to improve delivery of therapeutic agents targeting LSDs. We design robust enzyme-delivery nanoparticles as drug carriers, including but not limited to:
❖ Lipid-based nanoparticles, such as liposomes, non-liposomal vesicles, micelles, solid lipid nanoparticles, and exosomes.
❖ Polymer-based nanocarriers, such as polymer nanoparticles, polymer micelles, dendrimers, and nanogels
❖ Protein-based nanoparticles, such as protein complexes, virus-like particles
❖ Inorganic-based nanocarriers, such as metal, magnetic and silica nanoparticles, quantum dots, and carbon nanotubes
❖ Other types of materials, such as metal-organic skeleton composites.
Liposomes are attractive nanocarriers due to their biocompatibility and biodegradability, as well as the possibility of mass production of liposomes. Notably, our nanoliposomes are highly appreciated by our customers. The liposome formulations we are developing operate under two different therapeutic strategies:
(1) ERT strategy, where liposomes are used as nanocarriers to capture and deliver defective enzymes.
(2) Genetic strategy, where liposomes are used to capture genetic material with genetic information for further translation into desired proteins by cellular machinery.
Technology for the Preparation of Nanoliposomes
The innovative methods being developed by our team of experts for the production of liposome formulations allow to prepare these supramolecular nanostructures in a well-controlled manner and to obtain systems with proven properties including multifunctionality.
- Thin Film Hydration Technology
- De-Scaling Agent Removal
- Solvent Injection Method
- Reverse Phase Evaporation
- Microfluidics
- Compressed Fluid-Based Technique
Sequences encoding transgenes of interest, selected promoters and other regulatory elements.
Advantages of Nanocarrier-Based Delivery Therapeutic Solutions for LSDs
- Improving bioavailability of therapeutic compounds or enzymes
- Protecting the compound or enzyme from degradation
- Controlling the cycle time and release rate of the compound or enzyme
- Providing site-specific targeting to specific sites, collectively improving therapeutic outcomes while minimizing side effects
CD BioSciences' mission is to provide preclinical nanocarriers-based delivery therapeutic strategies for lysosomal storage diseases that provide new ideas for drug discovery. We look forward to collaborating with you in the discovery of innovative LSD therapeutic strategies. If you are interested in our solutions, please feel free to contact us.
Reference
- Tomsen-Melero J, Merlo-Mas J, Carreño A, et al. (2022) Liposomal formulations for treating lysosomal storage disorders[J]. Advanced Drug Delivery Reviews. 114531.