Liposomal formulations for treating lysosomal storage disorders

We are pleased to present a literature review on “Liposomal formulations for treating lysosomal storage disorders”, published on Advanced Drug Delivery Reviews, with contribution of authors Josep Merlo, Alba Cordoba, and Santi Sala from Nanomol Technologies, in collaboration with researchers from CIBER-BBN and ICMAB-CSIC and led by our co-founder Prof. Nora Ventosa.

Lysosomal storage disorders (LSD) are a group of rare diseases that are caused by lysosomal dysfunction, usually due to the lack of a single enzyme required for the metabolism of macromolecules, leading to a lysosomal accumulation of specific substrates, resulting in severe disease manifestations and premature death.

There is currently no definitive cure for LSD and, despite the approval of certain therapies, their efficacy is limited. Therefore, a suitable nanocarrier could help improve the efficacy of some of these therapies. Liposomes show excellent properties as drug carriers, since they can entrap therapeutically active compounds offering protection, biocompatibility and selectivity.

In this review, J. Tomsen-Melero et al. discuss the potential of liposomes for LSD treatment and a detailed analysis of promising liposomal formulations in the preclinical development stage is presented from various perspectives, including treatment strategy, manufacturing, characterization and future directions for the implementation of liposomal formulations for LSD. The review also includes recent work using our DELOS technology to manufacture robust enzyme-loaded nanoliposomes for treatment of #Fabry disease.

Judit Tomsen-Melero, Josep Merlo-Mas, Aida Carreño, Santi Sala, Alba Córdoba, Jaume Veciana, Elisabet González-Mira, Nora Ventosa. Liposomal formulations for treating lysosomal storage disorders. Advanced Drug Delivery Reviews, Volume 190, 2022, 114531. https://doi.org/10.1016/j.addr.2022.114531.