ClearPoint Neuro's Breakthrough: FDA Approves SmartFlow Cannula for Gene Therapy Delivery

ClearPoint Neuro, a global therapy-enabling platform company, has made a significant stride in the field of neuro gene therapy with the U.S. Food and Drug Administration (FDA) granting marketing authorization for its SmartFlow Neuro Cannula using the De Novo pathway. This approval marks the first-ever FDA marketing authorization of a device used to deliver gene therapy directly to regions of interest within the brain. The SmartFlow Neuro Cannula is intended for intraputaminal administration of PTC Therapeutics' gene therapy KEBILIDI™ (eladocagene exuparvovec-tneq) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

The SmartFlow Neuro Cannula's unique design enables low-volume priming, minimizes reflux, and enhances gene therapy delivery. Its single silica inner lumen extends throughout its length, allowing for low-volume priming and preventing leaks. The cannula body is rigid for precise placement and control, while the proprietary, non-PVC, latex-free, DEHP-free extension tubing with a female luer connector facilitates easy connection to syringes or pumps. The stepped distal tip and lumen sizes of 0.2 or 0.5mm reduce reflux, minimize priming/dead volume, and ensure precision. This design allows for accurate dosing to regions of interest in the brain, enabling intra-procedural adjustments to improve distribution and comparison of patient outcomes to infusion parameters.

The FDA's De Novo classification of the SmartFlow Neuro Cannula has significant implications for the future of neuro gene therapy treatments in the United States. This approval opens doors for treating AADC deficiency and holds promise for many other conditions, transforming the standard of care for children with these disorders and offering hope for improved outcomes and quality of life. With over 8,000 cannulas sold to date, ClearPoint Neuro's SmartFlow Neuro Cannula is the only co-labeled device to gain approval by regulatory bodies in both the U.S. and EU for delivery of an approved gene therapy to the brain.



ClearPoint Neuro's success in co-developing the SmartFlow Cannula with PTC Therapeutics highlights the importance of strategic partnerships in the gene therapy and delivery device sectors. The company's ability to attract new partners and offer a complete medical device solution, including surgical strategy, benchtop testing, compatibility assessments, and clinical trial support, gives confidence to current and future partners. This approval positions ClearPoint Neuro to lead the way in treating other neurological disorders, expanding its market reach, and transforming the standard of care.

In conclusion, the FDA's De Novo marketing authorization of ClearPoint Neuro's SmartFlow Cannula for direct delivery of gene therapy to the brain is a significant milestone in the field of neuro gene therapy. This approval opens new avenues for treating neurological disorders, validates ClearPoint Neuro's technology and expertise, and signals a growing market potential for gene therapy and delivery device stocks. Investors should consider ClearPoint Neuro as a strategic play in the growing neuro gene therapy market, with the potential for long-term growth and sustainability.