The Impact of your Giving
Fu/Bobo Lab Research update (Dec. 2024)
Our team continues our research efforts of developing new effective gene therapy products for treating Sanfilippo syndromes, with the goal to improve quality of life of not only the affected children, but also their families.
- Develop new effective gene therapy products. To address the critical challenges in moving gene therapy towards treating patients with rare diseases, Dr. Fu founded NeuroGT, Inc., a small start-up company. NeuroGT has recently licensed two new Sanfilippo gene therapy products that were developed in Fu/Bobo lab at UNC, for further development towards clinical application. NeuroGT has received the Orphan Drug Designation from the FDA for Sanfilippo A and B gene therapy products. We have initiated the interaction with the FDA regarding gene therapy clinical trials in patients with Sanfilippo A and B. We will submit the IND applications to the FDA for approval of Phase I/II clinical trials to test the new vectors for Sanfilippo A and B, towards eventual clinical application in patients.
- Establish novel biomarkers for assessing efficacy of Sanfilippo gene therapy. Lack of effective biomarkers has been a challenge to assessing the efficacy in Sanfilippo therapeutic development, including gene therapy. In Fu/Bobo lab, we have made efforts in Sanfilippo biomarker research targeting 3 major components of Sanfilippo disease pathology. These components are the transcription, metabolomics and extracellular vesicles (EVs). We will determine the pathological changes in the blood transcriptional, metabolomics and EV profiles in Sanfilippo mouse models and then humans, to identify the effective biomarkers with strong neurological links. The identified biomarkers may provide effective tools in the therapeutic assessment of Sanfilippo gene therapy.
- Depletion of preexisting anti-AAV antibodies for AAV gene therapy to treat all patients in need. AAV is a nonpathogenic virus widespread in humans. Therefore, there is high prevalence of anti-AAV-antibodies in human populations. This poses a critical challenge in translating AAV gene therapy to clinical application. Currently, only patients who do not have pre-existing anti-AAV-antibodies are eligible to AAV gene therapy treatments. There are no measures are currently available to overcome this challenge. To address this unmet need, we have developed a recombinant protein product that can degrade antibodies. We named it as antibody cleaver (AbC). For clinical relevance, in collaboration with NeuroGT, Fu/Bobo lab is currently testing the AbC in a large animal model (rabbits) to generate significant data to support the use of AbC pretreatment in AAV gene therapy towards clinical trials and eventual clinical application. This will make all patients in need eligible to AAV gene therapy, including the initial treatment and potential re-administration.