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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Gene Replacement Therapy

University of Massachusetts

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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.

 

NEW! Translational Institute for Molecular Therapeutics
Learn about the Gray-Edwards Lab

Learn about the Sena-Esteves Lab

Meet Dr. Guangping Gao, A Pioneer in AAV Gene Therapy

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Drug Repurposing

Baylor College of Medicine 

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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.

 

Learn about the Bellen Lab

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Treatment Exploration

St Jude Children's Research Hospital

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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.

Learn about the Mefford Lab

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Basic Science

Oregon Health and Science University

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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.

 

Learn more the Pruneda Lab

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

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