LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
Combining Sarepta Therapeutics Inc.’s gene editing technology and Genedit Inc.’s Nanogalaxy platform to treat neuromuscular disorders shows promising potential, the companies reported. A year into the research collaboration, Genedit’s polymer nanoparticles have demonstrated the ability to deliver therapeutic cargo to specific muscle tissue following system administration of targeted genetic medicines.