ClearPoint Neuro has achieved a historic milestone in neurological medicine with the successful delivery of the first commercial gene therapy treatments directly to the brain in the United States. The groundbreaking procedures utilized the company's SmartFlow Neuro Cannula to administer KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy approved for pediatric and adult patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
The treatments were performed by physician teams at hospitals in Texas and Boston, led by neurosurgeons from the IGNITE (Image-Guided Biologic Therapies: Neurosurgeons Innovating Treatment Excellence) working group. KEBILIDI is delivered directly to the putamen region of the brain through a stereotactic neurosurgical procedure, with the SmartFlow Neuro Cannula serving as the only FDA-authorized device indicated for this specific administration.
Clinical Significance and Expert Perspectives
"The future of cell and gene therapy is not coming…it is already here," commented Joe Burnett, President and CEO at ClearPoint Neuro. "The pivotal milestone of the first commercial gene therapy treatments in the US ever delivered to the brain should serve as both a sign of hope to patients, and as a clear headline to hospitals around the world that it is time to get ready for these transformational new medicines."
Dr. Daniel J. Curry, Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children's Hospital and Professor of Neurosurgery and Surgery at Baylor College of Medicine, emphasized the transformational nature of this approach. "Direct delivery of cell and gene therapy into the brain represents such a transformation in care, in that we can finally potentially restore the defects responsible for devastating pediatric neurological disease in this condition and eventually many other conditions that were previously relegated to comfort care. We can now treat the disease, not just the symptoms, and improve quality of life and life expectancy, as we've seen in children with AADC deficiency."
Dr. Scellig S. D. Stone, Director of Stereotactic and Functional Neurosurgery at Boston Children's Hospital, noted the broader implications: "While AADC deficiency is a rare childhood genetic disorder, we believe that this is the first of many new restorative therapies. Days like today make all our training and preparation truly worthwhile."
Record Clinical Activity and Regulatory Progress
ClearPoint Neuro reported unprecedented clinical activity in July, with 17 global patients treated across 11 different drug platforms, representing either commercial treatments or registered clinical trials. This record demonstrates the continued progression of the company's more than 60 active partners through the regulatory approval process.
The company's expanding portfolio has led to two additional biologics and drug delivery partners being selected for expedited review by the FDA, bringing the total number of company partners accepted into expedited review programs to nine. PTC Therapeutics was the first partner to receive approval through this pathway.
Technology Platform and Market Position
The SmartFlow Neuro Cannula has established itself as a critical component in the emerging field of direct brain therapy delivery. With over 9,000 cannulas sold to date, SmartFlow 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.
The device uses Convection Enhanced Delivery (CED) under direct image guidance to bypass the blood-brain barrier and deliver therapeutics to specific brain regions. Beyond its approval for KEBILIDI administration, the SmartFlow cannula has also received 510(k) clearance from the FDA for cerebrospinal fluid aspiration and injection of the chemotherapy drug Cytarabine into the ventricles.
Infrastructure Development
Supporting the commercial expansion of brain-delivered therapies, the ICD-10 Coordination & Maintenance committee has approved new, neuro infusion-specific ICD-10-PCS codes that will become effective October 1, 2025. These codes, for which ClearPoint Neuro submitted the application, will assist in tracking commercial use of the SmartFlow cannula and other direct brain delivery technologies.
Burnett highlighted the company's comprehensive approach: "Our complete ecosystem of drug delivery solutions including pre-planning software, peri-procedural neuro navigation hardware, cannula-based routes of administration, A.I.-informed quality control documentation, and expert clinical support are being used preclinically and in clinical trials for more than 60 active biopharma partners."
Future Implications
The successful commercial launch of direct brain gene therapy delivery represents a significant advancement in treating previously intractable neurological conditions. AADC deficiency, a rare, life-threatening neurological disorder, exemplifies the type of devastating pediatric conditions that may now be addressable through direct therapeutic intervention rather than symptomatic management alone.
As Burnett noted, "If ClearPoint technology continues to be included in the labeling itself as a combination product, it is hard to imagine a situation where ClearPoint is not an essential part of any hospital that wants to participate meaningfully in the neuro cell and gene therapy market."
