AAVantgarde Bio Secures $141M to Advance Retinal Gene Therapies

AAVantgarde Bio has announced the closing of a $141 million (€122 million) Series B financing round, co-led by Schroders Capital, with continued support from Atlas Venture and Forbion. The funding will accelerate the company’s two clinical-stage gene therapy programs targeting Stargardt disease and retinitis pigmentosa (RP) secondary to Usher syndrome type 1B.

Advancing Clinical Proof of Concept for Stargardt and Usher 1B

The Series B financing will support the completion of the CELESTE study, a clinical proof-of-concept (PoC) trial evaluating AAVB-039 in patients with Stargardt disease. The study assesses safety, tolerability, and preliminary efficacy across three dose levels and is being conducted in parallel with the STELLA natural history study, which remains open for recruitment.

AAVB-039 is designed to deliver the full-length ABCA4 protein, addressing the root genetic cause of Stargardt disease. This approach enables the potential treatment of any patient with ABCA4 mutations, regardless of the specific variant.

In addition, the funds will advance the ongoing LUCE Phase 1/2 clinical trial for AAVB-081, a gene therapy targeting retinitis pigmentosa secondary to Usher syndrome type 1B caused by MYO7A gene mutations.

Strategic Endorsement and Investor Support

Commenting on the announcement, Natalia Misciattelli, PhD, CEO of AAVantgarde Bio, stated: “This investment is a strong endorsement of our team, our science, and two clinical IRD programs. Both programs address the root genetic causes of devastating conditions and offer hope of improvement to patients and families living with progressive vision loss.”

Peter Kaiser, MD, a member of AAVantgarde’s Board of Directors, added: “This financing milestone represents a critical step forward in our ability to bring hope to patients affected by Usher 1B and Stargardt disease, two hereditary retinal disorders with urgent, unmet need.”

The company recently announced several regulatory achievements. AAVB-039 received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), and the company also secured Clinical Trial Authorisation (CTA) approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA), further enabling progress in both clinical programs.