SK pharmteco leverages its viral vector expertise in gene therapy partnership
Team-up with NIH and Axle Informatics called an 'important step' for rare disease
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SK pharmteco is partnering with Axle Informatics and the National Institutes of Health (NIH) to advance gene therapy programs for rare diseases.
The collaboration, which was funded in part by the NIH, focuses on developing viral vectors that use modified viruses to deliver genetic material into cells to treat rare inherited blood and metabolic disorders, such as aromatic L-amino acid decarboxylase (AADC) deficiency.
“Collaborating with the NIH and Axle Informatics marks an important step in our efforts to support patients with rare diseases,” John Lee, global head of viral vector at SK pharmteco, said in a company press release. “Rare disease programs often operate under significant development and funding constraints, making reliable technical execution and flexible partnerships especially important.”
AADC deficiency is caused by mutations in the DDC gene, which provides instructions for making the AADC enzyme. This enzyme is needed to produce dopamine and serotonin, two chemical messengers that nerve cells use to communicate.
Focus on lentiviral vectors
Gene therapies are designed to address the underlying genetic cause of disease. In AADC deficiency, the one-time gene therapy Kebilidi (eladocagene exuparvovec) has been approved since 2024 and is delivered directly into the brain.
Many rare disease gene therapy programs depend on viral vectors, which act as delivery vehicles for genetic material. SK pharmteco said its new partnership will pair its manufacturing capabilities with NIH investigators working to move academic research closer to clinical testing. The company is working as a subcontractor to the NIH, with Axle Informatics serving as primary contractor. The goal is to help accelerate the translation of academic research into clinical practice for rare diseases with significant unmet needs.
The first program under the partnership focuses on producing lentiviral vectors for rare inherited blood and metabolic disorders.
“By working closely with Axle Informatics and NIH investigators, we are applying our lentiviral manufacturing and analytical expertise to help advance these programs toward the clinic for patients with serious unmet needs,” Lee said.
As part of the work, SK pharmteco has manufactured a drug substance intended for use in the lab to genetically modify patients’ blood-forming stem cells, which are then infused back into the patient. The company also completed batch release testing, which confirms that a manufactured therapy meets quality and safety standards before it can move forward.
“This effort illustrates how integrated partnerships can accelerate the advancement of translational research into clinical outcomes,” said Gary Mays, chief operating officer at Axle Informatics. “Axle is focused on connecting capabilities across the NIH environment, and our collaboration with SK pharmteco adds essential manufacturing strength to help NIH advance promising gene therapies for patients in need.”
By providing viral vector development, manufacturing, and analytical support, SK pharmteco said it is positioning itself as a partner for academic investigators, public institutions, nonprofits, and emerging biotech companies working to bring promising therapies into the clinic.
