16 September 2022 - - The US Food and Drug Administration granted Rare Pediatric Drug designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease, US-based biotechnology company Aro Biotherapeutics said.

Pompe disease is a rare and deadly disease that can manifest at any age of life, including infancy.

It is characterized by debilitating muscle weakness that progresses over time and arises from a mutation in the acid alpha glucosidase enzyme that is responsible for breakdown of glycogen in muscle cells.

ABX1100 is a novel approach to treating Pompe disease that aims to reduce glycogen buildup in the muscles.

The FDA awards RPD designation to drugs treating rare pediatric diseases that are serious or life-threatening and affect patients from birth to 18 years of age.

Sponsors that receive RPD from the FDA are entitled to receive a Pediatric Review Voucher upon approval in a pediatric disease indication, giving the holder access to a priority review, reduced review time and potential expedited product approval.

Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins.

The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of diseases.