top of page

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_Logo.png

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 Logo

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_logo_edited.jpg

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 Logo.jpeg

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.

WE ARE RACING AGAINST TIME TO DEVELOP A LIFE-SAVING TREATMENT FOR UBA5 DISORDER.

Raiden Science Foundation Logo R only-02.png
bottom of page