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.

OUR RESEARCH PROCESS

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OUR SCIENCE PARTNERS

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UMASS CHAN

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UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL

GENE REPLACEMENT THERAPY

The UMass Chan Medical School research team will develop an adeno-associated virus gene therapy to correct the UBA5 deficiency. This preclinical development will lead to an investigational new drug application to the FDA.

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BAYLOR

COLLEGE OF MEDICINE

DRUG REPURPOSING

The Bellen Lab at Baylor College of Medicine aims to model patient UBA5 mutations using the fruit fly model to screen drugs that may correct disease causing defects in UBA5 patients.

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ST. JUDE

CHILDREN'S RESEARCH HOSPITAL

TREATMENT EXPLORATION

The Mefford lab at St. Jude Children’s Research Hospital is actively investigating approaches to increase amounts of the UBA5 protein in children affected by UBA5-related disorder.

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OHSU

OREGON HEALTH AND SCIENCE UNIVERSITY

BASIC SCIENCE

The Pruneda Lab at Oregon Health & Science University will use a biochemical and protein chemistry approach to investigate how patient UBA5 mutations affect the normal function of the protein UBA5.

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GENE THERAPY IS CURRENTLY THE MOST PROMISING ROUTE FOR LIFE-CHANGING TREATMENT.

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