Charles River Laboratories Partners With Non-Profit in an Effort to Cure a Rare Disease

On September 6, Charles River Laboratories International and Cure AP-4 announced a manufacturing collaboration. Charles River will supply plasmid DNA for Cure AP-4’s Phase I/II gene therapy trials against the rare disease AP-4 HSP.

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What is AP-4 HSP?

AP-4-associated hereditary spastic paraplegia (AP-4-HSP) is a group of four rare diseases. They include the subtypes SPG47, SPG50, SPG51, and SPG52. A mutation causes them in an autosomal recessive gene. As a result, children born with one of these defective genes have stunted muscle growth, delayed motor development, poor or absent speech development, and moderate to severe intellectual disability, among other symptoms.

AP-4-HSP cases are quite rare. Since its discovery in 2011, doctors have identified approximately only 249 cases of the neurodegenerative disorder at the time of writing

Despite the extreme rarity of these diseases, an organization focused on curing them sprung up. Founded in 2016 by the families of two SPG47 patients, Cure AP-4 seeks a cure for all four subtypes of the disease.  

Following suit, Charles River Laboratories announced a partnership with Cure AP-4 in support of their mission for the cure.

Cure AP-4’s Most Recent Partnership

In 2020, a clinical trial facilitated by Cure AP-4 began. The phase 1/2 trial’s goal is the documentation of clinical presentation and history of AP-4-HSP and the facilitation of early diagnosis, counseling, and anticipatory guidance for affected families. In addition, researchers seek to establish endpoints for future interventional traits.

To further the progress of the clinical trial, Charles River Laboratories entered an agreement with Cure AP-4 to supply high-quality DNA plasmids. These plasmids are a critical component for many cell and gene therapies. 

On the partnership, Birgit Girshick, Corporate Vice President and Chief Operating Officer of Charles River Laboratories, said, “The opportunity to work with a group as driven as Cure AP-4 is exactly why we do the work we do. Playing a small but critical role in delivering a potentially transformative therapy to a severely underserved patient population gives us an incredible sense of pride, and we are excited for the Cure AP-4 team to achieve this next stage.

Outside of Cure AP-4, other institutions and organizations have taken note of AP-4-HSP. For example, LifeArc granted Dr. Mimoun Azzouz, University of Sheffield, around $600,000 for his efforts in researching a cure for SPG47. Another example is The Manton Center for Orphan Disease Research giving Dr. Darius Ebrahimi-Fakhari a pilot grant for his “Functional Genomic Screen in a Neuronal Model of Hereditary Spastic Paraplegia”. The project seeks the identification of novel modulators of intracellular protein trafficking for the treatment of AP-4-HSP.

As for Cure AP-4, their trial’s end date is not until April 2025. However, collaborations such as Charles River Laboratories’ make the path to progress much more tangible. 

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Author

Max Heirich