Moligo Technologies has entered into a collaboration with Nationwide Children’s Hospital to provide long, single-stranded DNA (ssDNA) for research into gene therapies for cystic fibrosis (CF) and other airway diseases.
Moligo’s innovative enzymatic DNA production technology produces ultrapure, long ssDNA at an industrial scale. This technology is especially promising as it can introduce chemical modifications, making it a potential non-viral delivery method for cell and gene therapies.
Gene therapy for airway diseases, especially cystic fibrosis, is still in its infancy. The primary focus has been on replacing the mutated CFTR gene in CF with a correct version using an adeno-associated virus (AAV) as a delivery vehicle.
However, the CFTR gene’s length poses challenges for AAV delivery due to its limited genome size. Sriram Vaidyanthathan, Ph.D., from Nationwide Children’s Hospital, highlighted the potential of ssDNA to insert large genes like CFTR into airways, noting the unique characteristics of Moligo’s ssDNA, such as its length, scalability, and chemical modifiability.
Vaidyanthathan’s approach involves transplanting corrected airway stem cells into the airways to treat CF and other airway diseases. While double-stranded DNA (dsDNA) is another non-viral gene delivery option, it has a higher risk of triggering an immune response that could destroy the replacement gene. Vaidyanthathan plans to conduct an in vitro study using ssDNA in the coming year, followed by a subsequent study if successful.
Moligo CEO, Cosimo Ducani, Ph.D., expressed his enthusiasm about the collaboration, emphasizing the potential of gene therapy to offer a permanent cure for cystic fibrosis, especially for patients with rare genetic mutations.
He also highlighted the advantages of ssDNA over current gene therapy methods, especially when the gene size exceeds the AAV’s packaging capacity.
Moligo’s proprietary technology produces ultrapure DNA strands longer than 10,000 bases, 100% sequence-verified, and at scale. This process overcomes the limitations of traditional chemical methods, which produce shorter fragments with higher error rates and lower quantities.
About Moligo Technologies:
Founded in 2019, Moligo Technologies specializes in producing long, ultrapure, complex DNA at an industrial scale. Their patent-pending, PCR-free “injection-molding” platform can produce large quantities of DNA, aiding in the development of safer and more effective gene-based therapeutics, vaccines, and diagnostics.