Aro Biotherapeutics Raises $41.5 Million to Advance Development of Pompe Treatment

Rare Daily Staff

Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, has secured $41.5 million in a series B funding round.

Cowen Healthcare Investments led the financing, with participation from Aro’s existing investors Johnson & Johnson Innovation – JJDC, Northpond Ventures, Healthcap and BVF Partners. In conjunction with the financing, Christopher Picariello, president, JJDC, joined Aro’s Board of Directors.

“Aro is well positioned to advance our pipeline and progress the field by leveraging our Centyrin platform to enable efficient and tissue-targeted delivery of siRNA,” said Susan Dillon, CEO of Aro. “This funding enables us to continue to pursue our mission and bring new medicines to patients who desperately need more effective treatments.”

Aro will use the funds to advance development of its lead program, ABX1100, a novel potential treatment for patients with Pompe Disease. ABX1100 is comprised of a CD71 receptor binding Centyrin conjugated to a small interfering RNA (siRNA) which specifically interferes with expression of Gys1 mRNA, thereby reducing levels and overall activity of the Gys1 enzyme in muscle tissues. ABX1100 demonstrated a favorable safety profile in GLP toxicology studies, with durable reductions in Gys1 mRNA in muscles supporting the potential for monthly or quarterly dosing. ABX1100 has received Orphan Drug designation and Rare Pediatric Disease status from the United States Food and Drug Administration.

Aro recently announced the start of a first-in-human with ABX1100, and the company anticipates that initial data from this study will be presented in 2024. In addition, the Series B funding will support new Centyrin-siRNA conjugate programs focused on the discovery of new therapies for autoimmune disorders.

Photo: Susan Dillon, CEO of Aro