Rare Daily Staff    

Rare Trait Hope Fund has selected VGXI, a CDMO that specializes in the production of nucleic acid-based biopharmaceuticals for DNA vaccines and gene therapy applications, to support manufacturing of Rare Trait’s novel gene therapy for the ultra-rare genetic disease, aspartylglucosaminuria.

Aspartylglucosaminuria (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 the third quarter of 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,” said Dorothy Peterson, chief operating officer of VGXI.

VGXI is a Texas subsidiary of South Korea based GeneOne Life Science, a leading contract manufacturer of DNA plasmids for use in vaccines, gene therapies, and cell therapies.

Rare Trait Hope Fund is a non-profit organization founded in 2013 with the mission to facilitate research, raise funds, and support development of treatments and a cure for AGU. Through its fundraising efforts, Rare Trait has funded development of two different investigational treatments for AGU, a chaperone therapy and a gene replacement therapy.

Photo: Dorothy Peterson, chief operating officer of VGXI

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