SK pharmteco partners with NIH, Axle Informatics on rare disease gene therapies

SK pharmteco announced a strategic partnership with Axle Informatics and the National Institutes of Health (NIH) to support development of gene therapies targeting rare diseases.

Under the collaboration, SK pharmteco is serving as a subcontractor to the NIH, working alongside Axle Informatics, the agency’s primary contractor, to help advance viral vector-based therapies from academic research into clinical development.

The initial program under the partnership focuses on lentiviral vector manufacturing for rare inherited blood and metabolic disorders. SK pharmteco said it produced drug substance intended for ex vivo transduction of patients’ CD34+ hematopoietic stem cells and also managed batch-release testing, with most analytical assays conducted internally.

According to the company, the partnership is intended to provide manufacturing and technical support for rare disease programs that often face development and funding constraints. SK pharmteco said the collaboration highlights its role in supporting academic institutions, public agencies, non-profits, and emerging biotechnology companies developing advanced therapies.

The announcement follows additional recent expansion activity by SK pharmteco across both small molecule and gene therapy manufacturing. In March, the company announced a collaboration with UK-based Prozomix to expand biocatalysis capabilities across its global small molecule manufacturing network. The agreement gives SK pharmteco access to Prozomix’s library of more than 6,000 native biocatalysts to support enzyme screening and optimization for complex active pharmaceutical ingredient synthesis.

SK pharmteco has also continued investing in viral vector manufacturing infrastructure. Earlier this year, the company announced a $100 million investment to expand viral vector capabilities across facilities in the United States and France. The company also completed cGMP qualification of its Corbeil-Essonnes, France, site, which supports late-stage and commercial-scale viral vector manufacturing with capacity for up to 40 cGMP batches annually.