Preclinical development of a nanomedicine candidate for Fabry rare disease treatment to enter clinical phase

Introduction

Despite the development of new orphan medicines in recent years, there are still limited treatment options available for rare diseases. The Nano4Rare team has developed a patent-protected nanoencapsulation KET for the development of cost-effective therapies for rare diseases, based on biomolecules.

Development of nanoGLA

The Nano4Rare team has successfully used this technology for the development of a new patent-protected medicinal product candidate, named nanoGLA, for the treatment of Fabry disease, which is one of the most devastating LSD rare diseases.

Regulatory Designation

nanoGLA has been designated, in January 2021, as Orphan Drug by the European Medicine Agency (EMA) and the European Commission.

Preclinical Development

With the EU H2020 Smart4Fabry, nanoGLA was brought to an advanced stage of preclinical development, including the first GLP-toxicology studies in rats.

Objectives of the Phoenix EU Project

Under the Phoenix EU Project (#953110) (2021-2025), the production of nanoGLA will be scaled up and brought to GMP conditions.

1. First Objective: The Nano4Rare project will use nanoGLA engineered batches produced in the frame of the Phoenix project to complete the preclinical phase and generate sufficient quality data on safety, efficacy, and quality in order to get approval by regulators to proceed with the clinical phase.

2. Second Objective: A new spin-off company will be created for the advancement of nanoGLA towards the market and the commercialization of the patent-protected nanoencapsulation platform to generate new product candidates for rare disease treatments.