VGXI, Inc. Partners with Rare Trait Hope Fund in Support of Gene Therapy for Rare Disease, AGU

Industry leading plasmid DNA Manufacturer VGXI, Inc. partners with Rare Trait Hope Fund in support of a gene therapy for the rare genetic disease, Aspartylglucosaminuria (AGU).

The Woodlands, TX – Contract developer and manufacturer VGXI, Inc., which specializes in production of nucleic acid-based biopharmaceuticals for DNA vaccines and gene therapy applications, announced it has been selected by Rare Trait Hope Fund to support manufacturing of Rare Trait’s novel gene therapy for the ultra-rare genetic disease, Aspartylglucosaminuria (AGU).

AGU is a lysosomal storage disorder affecting an estimated 150 individuals worldwide. Early signs of the disorder develop in childhood with speech and language delays, motor function impairment, and recurring infections. With no approved treatments available, life expectancy averages 25-35 years of age for males and 30-40 years of age for females.

Under the recently executed manufacturing agreement, VGXI will leverage its highly optimized DNA production process to supply critical plasmid DNA raw material for the AAV-based, AGU gene replacement therapy developed by researchers at the University of North Carolina and UTSW in Dallas and funded by Rare Trait. VGXI has also committed to contribute a portion of the manufacturing costs in recognition of the significant personal donations and fundraising from patient’s families worldwide that made it possible to bring the investigational gene therapy to this stage of initial clinical testing. Production at VGXI is scheduled to begin in 3Q 2021.

“Our team has extensive experience producing often challenging gene therapy plasmids, and VGXI is honored to support Rare Trait in the clinical translation of this potentially life-changing therapy,” stated VGXI COO Dorothy Peterson.

Julia Taravella, Executive Director of Rare Trait said, “Our journey towards a cure for AGU has been long and challenging. It is only possible to progress treatments for ultra-rare diseases with the support of generous and visionary people like the employees at VGXI. We are so grateful to VGXI for sharing our vision that will help us celebrate this and many more exciting milestones for our AGU kids.”


VGXI, Inc., with 20 years of experience, is a leading provider of plasmid DNA manufacturing and development services. The company has a reputation of success in manufacturing DNA products under cGMP conditions for clinical trials in the US, EU, Asia, Canada, and Australia, and its cGMP and non-GMP products have passed rigorous reviews by several international regulatory agencies. VGXI’s ability to work with unique requirements and create custom manufacturing solutions is based on its patented manufacturing process, flexible cGMP production facility, and experienced development team. VGXI, Inc. is a GeneOne company.


Rare Trait Hope Fund is a 501(c)(3) non-profit organization (EIN 46-2762646) founded in 2013. The mission of the organization is to facilitate research, raise funds and support development of treatments and a cure for Aspartylglucosaminuria (AGU). In addition, Rare Trait provides resources for doctors, scientists and families world-wide to increase awareness of rare diseases in general and Aspartylglucosaminuria in particular. Through its fundraising efforts, Rare Trait has funded development of two different investigational treatments for AGU, a chaperone therapy and a gene replacement therapy.

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GeneOne Life Science Inc. (“GeneOne” KOSPI: 011000) headquartered in Seoul, South Korea is an international biotechnology company and a leading contract manufacturer of DNA plasmids for use in vaccines, gene therapies, and cell therapies. GeneOne has recently expanded into the manufacture and development of mRNA. GeneOne has maintained a focus on vaccines against emerging infectious diseases to address global needs, including in resource challenged regions. Its small molecule portfolio of immunomodulators address diseases such as prevention of upper respiratory bacterial and viral diseases, and treatment of autoimmune and inflammatory diseases. GeneOne has three products against COVID-19 in clinical development: GLS-5310 DNA vaccine (Phase I/IIa), GLS-1200 nasal spray to prevent COVID-19 infection (Phase II), and GLS-1027 to prevent the inflammation and clinical worsening for those infected with COVID-19 (Phase II).

VGXI, Inc., a GeneOne company located in The Woodlands, Texas, is the global leading CDMO for cGMP DNA plasmid manufacture.

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