ClearPoint Neuro, Inc. congratulates its partner PTC Therapeutics on completion of its BLA submission to the U.S. Food and Drug Administration (FDA) for the approval of Upstaza?(eladocagene exuparvovec), an investigational treatment for AADC Deficiency. If approved, Upstaza? would become the first therapy to treat AADC Deficiency in the United States.

AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life ? physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping.

The lives of affected children are severely impacted and shortened. Ongoing physical, occupational and speech therapy, and interventions, including surgery, also are often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems. With over 7,000 cannulas sold to date, SmartFlow is the only co-labeled device to gain approval by a regulatory agency for delivery of an approved gene therapy to the brain. The cannula is used by many of ClearPoint Neuro?s 50+ pharmaceutical, academic, and biotech partners to bypass the blood brain barrier and deliver therapeutics to regions of interest using Convection Enhanced Delivery (CED) under direct image guidance.

The SmartFlow Cannula has 510(k) clearance from the FDA for use in the United States for the aspiration of cerebrospinal fluid or injection of the chemotherapy drug Cytarabine into the ventricles. It has also been CE marked to deliver approved fluids into the brain and for aspiration of cerebrospinal fluid, and holds regulatory clearance for clinical use in Israel and Brazil. SmartFlow is utilized in approved clinical and preclinical studies for various research and drug trials.