About Us:
The Pediatric Advanced Neurotherapeutics (PANTher) Program was developed to coordinate basic science, translational and clinical research efforts relevant to the treatment of childhood neurological diseases. Its goals are to:
- foster collaborations between basic scientists and clinicians at UCSF to develop new therapeutics for pediatric neurological disorders
- translate basic science discoveries from UCSF researchers to clinical trials
- engage with industry partners to conduct clinical trials of promising novel therapeutics for neurological diseases at UCSF
We are particularly interested in treatments for rare diseases, including gene therapy and medications with novel therapeutic mechanisms.
Working with Scientists
- Discussion of basic science approaches for therapy development for rare disease
- Discussion of feasibility of clinical trials for rare diseases
- Defining outcome measures for trials
- Natural history studies to lay ground work for treatment trials
- Working with IRB and Scientific Review to Launch Trials
Working with Industry
- Identification of UCSF experts in particular diseases to partner for clinical trials
- Natural history studies as precursor to treatment trials
- Development of clinical outcome measures for trials
- Clinical trial sites in San Francisco (Mission Bay) and Oakland already engaged in gene therapy trials
- Experienced investigators, experts in rare diseases, and research coordinators
Examples of Recent and Ongoing Clinical Trials
- A Study of AAV9 Gene Therapy in Participants With Canavan Disease
- A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
- CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
- Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency by MR-Guided Direct Delivery of AAV2-AADC to Midbrain Dopaminergic Neurons. (Investigator-Initiated)
- Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II) (REGENXBIO)
- Phase 1/2 Study with ETV:IDS (DNL310) in Patients with Hunter Syndrome (MPS II) (Denali)
- A Prospective Open-Label Study to Assess Efficacy and Safety of RT001 in Subjects with Infantile Neuroaxonal Dystrophy. (Retrotope).
- An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) (PTHS-001)
- An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) (PWS-001)
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