RARE Daily

Aro Biotherapeutics Raises $24 Million to Advance Targeted Delivery Platform

January 3, 2023

Aro Biotherapeutics, a company developing platform technology for precise tissue specific targeting of genetic medicines, has raised $24 million of a potential $100 million series B financing according to a document filed with the Securities and Exchange Commission.

The financing follows a $88 million series A round, which closed in early 2021 and was led by Northpond Ventures and Cowen Healthcare Investments.

Aro is developing a unique class of Centyrin-conjugated RNA therapies to target RNA medicines efficiently and selectively to the specific site of disease with an initial focus in rare genetic and immune disorders. The new funding will be used to advance the company’s lead therapeutic candidate, a centyrin-targeted siRNA, into clinical development for the treatment of Pompe disease, a rare inherited disease characterized by debilitating and progressive muscle weakness.

Pompe disease arises from a mutation in the enzyme called acid alpha-glucosidase (GAA) that is responsible for the breakdown of glycogen in muscle.  When there isn’t enough GAA present, glycogen accumulates, and this toxic build-up drives disease progression. Current approved enzyme replacement therapy (ERT) for Pompe aims to replace the missing enzyme with recombinant forms of GAA to reduce glycogen buildup in the muscle, and to restore normal muscle function. However, a shortcoming of these medicines is their relatively inefficient delivery to muscle tissue. Additionally, ERTs require biweekly intravenous infusions, are immunogenic in many patients, and the disease continues to progress leading to requirements for assisted ventilation, loss of mobility, and significantly reduced life expectancies. It is well recognized that there is a substantial need for new treatment options that deliver enhanced efficacy, improved safety, and better convenience for Pompe patients.

Aro is developing a novel approach in Pompe disease known as substrate reduction therapy (SRT).  ABX1100 is a first-in-class Centyrin-siRNA conjugate that is designed to reduce glycogen levels in the muscle by inhibiting the expression of the enzyme responsible for glycogen production, glycogen synthase 1 (GYS1). A mouse cross reactive Centyrin-siRNA conjugate demonstrated robust and muscle-specific inhibition of the GYS1 gene in preclinical animal studies, which has led to significant reductions of glycogen in target tissues. The company plans to advance ABX1100 to human clinical studies this year.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.