Creating futures through transformative science and sustainable vision.
Our “One at a Time” approach highlights NeuroGT’s commitment to precision, patient-centric care, and innovation in gene therapy. Each phase in the journey emphasizes how individual genes, patients, and solutions are the building blocks of our mission to transform genetic medicine.
Welcome to NeuroGT
At NeuroGT, we believe that every gene, every patient, and every solution counts. We are transforming the landscape of genetic medicine with advanced gene therapy technologies designed to target the root causes of rare neurogenetic diseases—offering new hope to patients and their families where none existed before.
Our therapies focus on conditions that affect the central nervous system and critical metabolic pathways, aiming to change the trajectory of diseases that impact children and their families. With a mission-driven approach, we combine cutting-edge science, patient-centric care, and innovative partnerships to make sustainable, lasting impact through every treatment we develop.
Innovation in Neurodegenerative Diseases
NeuroGT is committed to addressing critical unmet need for rare and devastating lysosomal storage disorders (LSDs)
Hurler Syndrome
Hunter Syndrome
Sanfilippo Syndrome
Explore NeuroGT: Our Journey Starts Here
We are not just developing therapies—we are pioneering new possibilities in genetic medicine. Our work is fueled by a belief that every patient matters and that innovation and collaboration can overcome the toughest challenges.
Join us on this journey.
Whether you are a patient, caregiver, investor, or partner, we invite you to explore how NeuroGT is shaping the future of gene therapy—one breakthrough at a time.
Our Technologies: Driving Innovation Forward
Our platform technologies are designed to overcome the most significant barriers to effective gene therapy.
AAV9 Gene Replacement Therapy Platform
Targeting the nervous system with precision, delivering life-changing genes across the blood-brain barrier.
Antibody Cleaver (AbC) Technology
Enabling AAV dosing and re-dosing by transiently depleting pre-existing anti-AAV antibodies, broadening patient eligibility
AAV-mediated EV-mRNA
Cargo Technology
