Hi families! Our last update came in May during our family meeting with Dr. Xin Chen at UTSW when we learned that his early results are very promising and he now has the treatment in the bodies of mice with ADSLD. So exciting! We also learned he required more funding, and we’ve been steadily fundraising since then. While he did give us a deadline of September 1st, this was unrealistic for us. Instead, we are going to grant funds as we raise them. This method leaves us with a

We wanted to share a summary of recent discussions about ADSL research, our fundraising efforts, and the path toward future treatments. Our goal is to keep families informed about where we are today, what we know, and what questions still remain. Where We Are Today Rare Birds Foundation is currently focused on advancing the most promising therapeutic approaches for ADSL Deficiency, with gene therapy as our primary research priority. Current evidence suggests that gene therapy

Title Slide for Rare Birds Foundation's conversation with Dr. Xin Chen of UT Southwestern regarding advancements in an AAV-9 Gene Therapy currently in development for the treatment of Adenylosuccinate Lyase Deficiency Disorder (ADSLD). June 3, 2026 This meeting focused on updates from Dr. Xin about the development of a potential gene therapy for people living with ADSL deficiency, an ultra-rare metabolic disorder affecting approximately 163 individuals worldwide. Dr. Xin shar