With backups at academic manufacturing centers beginning to hamper translation of early stage gene therapies, pushing those who can afford it to CMOs, at least one non-profit has stepped in with a model to deliver the services on the cheap. But without more, the field may be slow to benefit from the next generation of gene therapies coming through.
Gene therapies are notorious for scale up issues that can hinder late stage trials or commercialization. However, manufacturing difficulties also extend to preclinical development, in particular in the transition from animal proof of concept to IND-enabling studies. The former does not require GMP-like vector, but the latter does.
Academic researchers typically have two options for obtaining high enough quality vector, and sufficient quantities, for IND-enabling work: use a specialized vector production core at a large academic institution, of which there are few; or compete with drug companies that are capable of paying more for limited manufacturing slots at CMOs.
With FDA’s first-ever approval of an in vivo gene therapy in December, Spark Therapeutics Inc.’s Luxturna voretigene neparvovec, the field is on the rise. Academic and industry researchers alike are pushing the modality’s horizons, moving gene therapies beyond the small volume compartment of the eye, where Luxturna is delivered, into larger organs and into indications that require systemic administration, which necessitates orders of magnitude more vector.
University production cores now have long lines that are pushing some researchers to seek more expensive CMO services, which in most cases requires dipping into grant money that isn’t allocated for that purpose.
NIH is still backing the search for new