AAVantgarde Secures $141 Million Series B to Advance Gene Therapies for Stargardt Disease and Usher 1B Syndrome
Key Insights
AAVantgarde Bio (search) closed a $141 million Series B financing round co-led by Schroders Capital (search) and Atlas Venture (search) to advance clinical programs for inherited retinal diseases (search).
The funding will support completion of clinical proof-of-concept studies for AAVB-039 targeting Stargardt disease (search) and AAVB-081 for retinitis pigmentosa (search) secondary to Usher 1B syndrome (search).
Both programs use gene augmentation therapy to address root genetic causes, with Stargardt disease (search) being the most prevalent macular dystrophy (search) in young people and Usher 1B causing progressive vision loss combined with congenital deafness.
AAVantgarde Bio (search), a clinical-stage biotechnology company developing therapies for inherited retinal diseases (search) (IRDs), has successfully closed a $141 million Series B financing round to advance its pipeline targeting devastating genetic eye conditions. The round was co-led by Schroders Capital (search) and existing investor Atlas Venture (search), with participation from multiple new and existing investors including Amgen Ventures (search), Forbion (search), and Sofinnova Partners (search).
Targeting Unmet Medical Needs in Inherited Eye Diseases
The funding will primarily support completion of clinical proof-of-concept studies for two lead programs addressing severe inherited retinal diseases (search) with no current approved treatments. AAVantgarde's AAVB-039 program targets Stargardt disease (search), the most prevalent macular dystrophy (search) in young people, caused by mutations in the ABCA4 (search) gene. The company's CELESTE study aims to demonstrate clinical efficacy through gene augmentation therapy that delivers the full-length ABCA4 protein.
"Stargardt disease (search) is the most prevalent macular dystrophy (search) in young people, yet there are currently no approved treatments," the company stated. AAVantgarde's approach addresses the root genetic cause through gene augmentation therapy, enabling treatment of any patient regardless of their specific mutation.
Dual Sensory Impairment Treatment
The second program, AAVB-081, targets retinitis pigmentosa (search) secondary to Usher syndrome 1B, a rare condition caused by mutations in the MYO7A (search) gene. This devastating disorder causes progressive vision loss combined with congenital deafness, resulting in a double sensory disability. The company's LUCE phase 1/2 clinical trial will evaluate gene augmentation therapy delivering the full-length MYO7A protein.
"Usher 1B causes progressive vision loss combined with congenital deafness, resulting in a double sensory disability," according to the company's description of the condition. The AAVB-081 program targets the root genetic defect, offering potential treatment for patients affected by this dual impairment.
Clinical Development Milestones
The Series B proceeds will specifically fund completion of the CELESTE study for Stargardt disease (search) and the associated STELLA natural history study involving more than 100 patients. Additionally, the funding will support completion of clinical proof-of-concept for the LUCE phase 1/2 trial in Usher 1B patients.
"This investment is a strong endorsement of our team, our science, and two clinical IRD programs," said Dr. Natalia Misciattelli, CEO of AAVantgarde. "Both programs address the root genetic causes of devastating conditions and offer hope of improvement to patients and families living with progressive vision loss."
Investor Confidence in Gene Therapy Platform
Harry Raikes, Head of UK Venture Investments at Schroders Capital (search), emphasized the potential of AAVantgarde's approach: "Its innovative platform could enable best-in-class treatments for inherited eye diseases like Usher 1B and Stargardt's by overcoming key barriers in current AAV technologies and reaching patients who currently have limited treatment options."
Dr. Peter Kaiser, Professor of Ophthalmology at the Cleveland Clinic Lerner College of Medicine and AAVantgarde Board member, highlighted the clinical significance: "Inherited Retinal Disorders (IRDs), such as Stargardt disease (search) and RP secondary to Usher 1B, are among the leading causes of blindness in children and young adults worldwide. 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."
Broad Investor Syndicate
The financing round attracted significant interest from both new and existing investors. New participants included Amgen Ventures (search), Athos Capital, CDP Venture Capital through its Large Ventures Fund, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors. Existing investors Longwood Fund and Sofinnova Partners (search) provided continued support alongside the co-leads.
The successful completion of this substantial Series B round positions AAVantgarde to advance its clinical programs toward potential regulatory milestones, offering hope to patients with inherited retinal diseases (search) who currently face limited treatment options.