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Gifting Hope on Giving Tuesday
Supporting Genetic Cures for Kids’ gene therapy trials with plasmid DNA production
Founded in 2021 by Golden and Chris Whitrod, parents of now four-year-old Tallulah Moon (pictured), Genetic Cures for Kids’ (GC4K) first mission, known as Our Moon’s Mission, is to develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56), a debilitating neurodegenerative disease that robs children of the ability to walk, talk, and move independently.
“What was once a tragic diagnosis, has the potential to become a triumph, as we draw nearer to finding a cure for SPG56,” commented Golden Whitrod. “With the advancement of this groundbreaking treatment, our partnership with Charles River is bringing us closer to aiding children like Tallulah, who, until now, have faced the daily hardships of SPG56 without any glimmer of hope. We're truly excited to have the backing of this skilled team as we gear up to produce this historic treatment.
“By making this announcement on Giving Tuesday (November 28), we are giving thanks to Charles River. This symbolic occasion not only commends the generosity of [Charles River] in supporting GC4K’s quest to cure SPG56, but also to recognize the broader goal that our partnership is striving for – a brighter future for all children living with rare genetic diseases.”
What is SPG56?
SPG56 manifests as a progressive neurological disease characterized by varying degrees of spasticity and muscle weakness. Typically beginning with motor and cognitive regression in childhood, it continuously worsens through life, with additional clinical manifestations including intellectual disability, dystonia, cerebellar ataxia, subclinical peripheral neuropathy, seizures, and visual impairment.
Tragically, there is currently no treatment for this ultra-rare genetic disease, and limited access to genetic testing means diagnoses are hard to achieve, with fewer than 100 people currently diagnosed with SPG56 globally. GC4K is leading a gene therapy program that could not only cure SPG56 but create a replicable framework that paves the way to develop treatments for some of the 7,000 other genetic diseases in the world, more than 95% of which have no treatment or cure.
Commitment to Cell and Gene Therapy
Our CDMO team will support the program by providing High Quality (HQ) plasmid DNA, which combines key features of GMP manufacture with a rapid turnaround to accelerate time to clinic. As critical starting material in the development of many cell and gene therapies, in 2022 we opened a purpose-built HQ plasmid production center of excellence to address global demand and supply issues and support the growing needs of therapeutic developers, and patients, worldwide.
“The opportunity to work with Genetic Cures for Kids is exactly why we do what we do – playing a role in delivering potentially curative treatments to patients suffering with an ultra-rare disease,” remarked Kerstin Dolph, Charles River CSVP, Biologics Solutions, in a press release announcing the collaboration. “We are excited to support the team as they work through the next stage of their program development.”
For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.
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