The European biotechnology company, AAVantgarde Bio, has announced the successful closure of a $141 million (€122 million) Series B financing round.
The funds will be used to accelerate the company’s clinical programs targeting Stargardt disease and Usher syndrome type 1B, both inherited retinal diseases (IRDs). The round was co-led by Schroders Capital, Atlas Venture, and Forbion, with participation from Amgen Ventures, Athos Capital, CDP Venture Capital, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors. Existing investors Longwood Fund and Sofinnova Partners also reaffirmed their support.
Funding to Drive Dual Gene Therapy Programs
The Series B proceeds will primarily fund two pivotal studies:
- Phase I/II CELESTE study (NCT07161544), evaluating AAVB-039 in Stargardt disease, a genetic eye disorder caused by mutations in the ABCA4 gene. The trial aims to establish clinical proof of concept and complete the STELLA natural history study involving more than 100 patients.
- Phase I/II LUCE trial (NCT06591793), investigating AAVB-081 for retinitis pigmentosa (RP) secondary to Usher syndrome type 1B, driven by mutations in the MYO7A gene.
Both programs are gene augmentation therapies that use adeno-associated virus (AAV) vectors to deliver full-length versions of the defective proteins (ABCA4 and MYO7A) to target the underlying genetic causes rather than just the symptoms. This approach could open the door to treating a broader patient population across diverse mutations.
Stargardt disease is the most common macular dystrophy in young people, leading to progressive central vision loss. Usher 1B syndrome, on the other hand, causes both retinal degeneration and congenital deafness, resulting in a dual sensory impairment that profoundly impacts quality of life.
“With this investment, we can accelerate our mission to bring transformative therapies to patients who currently have no treatment options,” said Dr. Natalia Misciattelli, CEO of AAVantgarde. “Both programs directly target the root genetic causes of devastating conditions and offer real hope to families affected by progressive vision loss.”
Dr. Peter Kaiser, Professor of Ophthalmology at the Cleveland Clinic Lerner College of Medicine and AAVantgarde board member, emphasized the broader implications of the financing: “This milestone represents a critical step forward in our ability to bring hope to patients affected by Usher 1B and Stargardt disease. These are among the leading causes of blindness in children and young adults worldwide.”
