AZ forges surprise R&D deal with virus gene therapy biotech


MedImmune, the R&D arm of AstraZeneca has teamed up with California based 4DMT, a next-generation virus-based gene therapy research house developing a treatment for chronic lung disease.

The collaboration AstraZeneca announced today is the first, and quite unexpected step of the pharma giant into the adeno-associated virus (AAV) gene therapy, vector discovery and product development.

AAV vectors are responsible for transporting genes to accessible tissues in the body for in-vivo expression and therapeutic application and target dividing and non-dividing cells without integrating genetic material into the host genome.

The genetic material transferred by AAV vectors into cells is the blueprint to produce a protein whose function targets pathological processes contributing to diseases.

AZ has been stepping into the genetic research before, but past co-operations were focused on RNA and oligonucleotide-based drugs to switch off problem genes and using CRISPR technology for gene editing.

But the newly formed collaboration is the first AZ’s direct involvement into the field of vector-based gene transfer.

Roland Kolbeck, vice president, research and development, for respiratory, inflammation and autoimmunity at MedImmune, said: “Rapid advances in AAV therapy make this a promising tool to advance innovation in chronic lung disease, particularly in areas of high unmet need.”

“This collaboration strategically pairs 4DMT’s expertise in AAV with MedImmune’s leadership in respiratory science, focused on early intervention and disease modification.”

4DMT will use its respiratory expertise in vector discovery and engineering, optimisation and process development to conduct product development starting from early clinical stages

David Kirn MD, CEO and co-founder of 4DMT, added: “This exciting collaboration may open the door to significant advancements in treatment for respiratory patients.”

“Our progress in customised AAV vectors enables us to unlock the potential of gene therapy and, with MedImmune’s expertise in protein engineering, we will continue to push boundaries in proprietary gene delivery to tissues and cells.”

The new partnership will focus on helping patients with chronic lung disease, utilising 4DMT’s novel discovery platform to generate optimised AAV vectors.

So far only three commercial gene therapies have been approved across the globe. One of which, Glybera, was developed for the ultra-rare inherited disease familial lipoprotein lipase deficiency (LPLD).

But manufacturer uniQure axed Glybera last year because, at cost of a million euros per patient, reports suggested only one patient was ever treated with it.

Piotr Wnuk

12 July, 2018