OUR APPROACH
Discover. Develop. Deliver.
We are taking a holistic approach towards developing effective treatments for UBA5 with a focus on gene therapy as our first solution. We will be operating like a lean startup, meaning we will be fast, efficient and cross-functional. We know that our work will ultimately benefit countless kids suffering from rare diseases around the world.
UMASS CHAN
UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL
GENE REPLACEMENT THERAPY
Developing a gene therapy (AAV9) to correct UBA5 deficiency. This preclinical development will lead to an investigational new drug application to the FDA. Currently in the efficacy testing stages and will soon proceed to in-vivo toxicology studies.
BAYLOR
COLLEGE OF MEDICINE
DISEASE MODELING
Fly models with patient UBA5 mutations have been generated, and a forthcoming paper will detail this research. Additionally, Baylor is collaborating with the Jackson Laboratory to develop the first-ever UBA5 mouse models, which will advance translational research and used for drug screening.
ST. JUDE
CHILDREN'S RESEARCH HOSPITAL
TREATMENT EXPLORATION
Actively exploring ways to boost UBA5 protein in children with UBA5-related disorder. Also, developing UBA5 cerebral organoids for research applications.
OUR SCIENCE PARTNERS
OHSU
OREGON HEALTH AND SCIENCE UNIVERSITY
DRUG REPURPOSING
Developing a novel drug screening platform to discover potential drugs that can restore UBA5 activity. Several promising candidates have already been identified.
CORIELL
CORIELL INSTITUTE FOR MEDICAL RESEARCH
BIOBANK
Established a biobank to centralize all UBA5 sample collection for research. Please contact research@raidenscience.org for more info or if interested in submitting sample for research.
