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ClearPoint Neuro Congratulates Partner PTC Therapeutics on Receiving Positive CHMP Opinion for Gene Therapy to Treat AADC Deficiency
First Gene Therapy Directly Infused into the Brain to be Administered with ClearPoint’s SmartFlow® Cannula SOLANA BEACH, Calif., May 20, 2022 (GLOBE NEWSWIRE)
About this update from Clearpoint Neuro Inc.
[{"type":"text","content":"First Gene Therapy Directly Infused into the Brain to be Administered with ClearPoint’s SmartFlow® Cannula\nSOLANA BEACH, Calif., May 20, 2022 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global therapy-enabling platform company providing navigation and delivery to the brain, today congratulates partner PTC Therapeutics for receiving a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending its gene therapy treatment Upstaza™ (eladocagene exuparvovec), formerly PTC-AADC, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The use of ClearPoint Neuro’s proprietary CE Marked SmartFlow® Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy is included in the submission for administration of Upstaza. “This is a significant milestone for PTC Therapeutics, for ClearPoint and for biologics researchers everywhere as it marks the very first positive CHMP recommendation for direct injection to the brain of a gene therapy to treat a severe, highly morbid and fatal neurological disorder,” commented Joe Burnett, President and CEO of ClearPoint Neuro. “This milestone is incredibly important for a couple of reasons. First, it provides a potential path for other therapies to follow and sets an important precedent for regulatory approval of direct administered gene therapies to the brain. And second, we see this as a validation of ClearPoint’s biologics partnership strategy and our rigorous bench, preclinical and clinical experience. We believe this announcement will give our 45 existing and future pharmaceutical, academic and biotech partners the confidence that SmartFlow is able to pass through the necessary regulatory scrutiny and can de-risk the commercialization efforts of many different drugs to multiple targets in the brain.” About aromatic L-amino acid decarboxylase (AADC) deficiency 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, which can happen daily and last for hours, causing the eyes to roll up in the head, fr...