The collaboration – financial terms of which have not been disclosed – will see the Swiss contractor develop an adeno-associated virus (AAV) production process for Benitec’s candidate gene therapy at its site in Houston, Texas in the US.
Benitec's candidate - TT-034 – is designed to prevent the expression – or silence – hepatitis C virus genes by binding the mRNA sequences produced when they are transcribed. This creates double stranded sections on the mRNA transcript that cannot be translated into protein, therby preventing viral replication.
What differentiates TT-034 from RNA-based interference therapies is that it is a double stranded DNA molecule introduced directly into the cell nucleus where it acts as a template for the production of many copies of the silencing sequence.
Benitec – which bought the TT-34 programme along with developer Tacere Therapeutics in 2012 after its former partner, Pfizer, pulled out - claims its so-called "DNA directed" approach bypasses the delivery problems that hinder RNA-based silencing therapies.
Lonza’s role will be to create a scalable production process for the adeno associated viruses (AAV) that are used to deliver the double stranded DNA constructs.
Ryan Scanlon, Lonza’s Director of Commercial Development for Viral Therapy, told us: “The process will produce AAV vectors containing the gene of interest.”
He added that Lonza is hopes it will win the commercvial supply contract if the drug is approved.
Scanlon said: “Lonza is looking forward to building a strong, long-lasting relationship with Benitec beginning with this agreement. As Benitec’s AAV pipeline progresses, we hope to help Benitec leverage Lonza’s industry-leading biologics track record in process technology.”
AVV history
Benitec will hope its drug does not suffer the same fate as Celladon’s Mydicar (AAV1/SERCA2a), the last AAV-based drug for which Lonza developed a production process at the Houston site.
The US biotech ceased development of the drug – a gene therapy for heart failure – in April after it failed to meet primary and secondary endpoints in a Phase IIb trial.
Lonza – which completed development of the Mydicar manufacturing process and production of a demonstration batch in December – had been poised to build Celladon a facility and produce supplies of the drug for trials.
Celladon has said since that it does not intend to exercise its option to have Lonza build a facility. The US biotech – which also cancelled an active pharmaceutical ingredient (API) deal it had with Novasep – is hunting for a buyer.