Congratulations to the team at AAVantgarde Bio on receiving clearance from the FDA on their IND for their AAVB-039 program in Stargardt's disease! AAVB-039 uses AAVantgarde’s proprietary dual AAV intein platform. The platform has achieved unprecedented preclinical data demonstrating high transduction, protein expression, and safety in multiple large animal models.
✨ We are thrilled to announce that the #FDA has cleared our IND application for AAVB-039, the best-in-class dual AAV intein gene therapy for Stargardt disease showing the highest quantified ABCA4 protein expression in relevant large animal models. This is a pivotal moment for our team and for patients living with #StargardtDisease — bringing us one step closer to delivering a much-needed treatment where none currently exists. 🎉 Congratulations and thank you to the AAVantgarde team for the hard work put into this submission! 👇 Find more about our AAVB-039 program, able to address all Stargardt patients independently of their ABCA4 mutation: https://lnkd.in/dyEvcXKZ #StargardtDisease #GeneTherapy #Ophthalmology #IRDs #RareDisease #VisionRestoration #TIGEM #FondazioneTelethon