J&J gets piggyBac from Transposagen to develop T-Cell therapies
By Dan Stanton+ Dan Stanton 26-Nov-2014 2014-11-26T00:00:00Z
Last updated on 26-Nov-2014 at 16:57 GMT 2014-11-26T16:57:43Z
There is an upsurge in biopharma request for cell and gene therapy applications, says Transposagen as it inks a deal with Janssen to develop allogeneic Chimeric Antigen Receptor (CAR) T-cells.
Autologous CAR immunotherapy involves taking T-Cells directly from a patient, engineering them so that they express receptors specific to a particular form of cancer, then reintroducing them to the patient, and is presently a prominent part in Novartis’ pipeline with its CTL019 candidate targeting lymphoblastic leukemia receiving US FDA breakthrough designation earlier this year.
In contrast, allogeneic CAR-Ts have the potential for use as off-the-shelf cancer treatments without the need of matching donor with recipient and J&J’s offshoot Janssen is looking to develop such therapies, paying up to $292m (€230m) for each allogeneic CAR-T therapeutic developed using Transposagen Biopharmaceuticals’ genome editing implements.
The deal is focused on discovery and pre-clinical development, Eric Ostertag, CEO & President of Transposagen told Biopharma-Reporter.com, with Janssen then responsible for manufacturing allogeneic CAR-T therapies discovered using the piggyBac Footprint-Free Gene Editing System.
PiggyBac Gene Delivery System
The creation of CAR-T therapies involves two main steps, Ostertag explained. Firstly a custom-designed, site-specific nuclease ties and cleaves specific sites in the DNA, resulting in a ‘knockout’ mutation, which abolishes function of the targeted gene.
“The piggyBac Gene Delivery System platform is then used to produce the CAR component stably into the genome for reliable expression on the surface of the T-cell,” he continued, adding Transposagen’s technology held a number of advantages over other therapeutic protein expression instruments in terms of safety, precision, and efficiency.
The technology is totally reversible, he said, “essentially having a built-in safety switch, which is significant for use in humans.” Furthermore, when combined with the firm’s Footprint-Free Gene Editing System, the hard can “engineer difficult cell lines, such as stem cells, and primary cell lines, such as T-cells.”
As for cost-saving, Ostertag told us the platform suggested a number of benefits. “All of our technologies are non-viral systems, resulting in cost savings and further enhancing safety. The GMP production process for the viral vectors typically used in CAR-T therapeutics is significantly more expensive and time consuming.”
The deal with Janssen is for an initial three years, but the company’s Director of Sales & Marketing Jack Crawford said the stiff is observing a general upsurge in request for its systems from Big Biopharma looking to develop personalized therapies.
“Our technologies and services have applications spanning the entire preclinical drug discovery and development R&D pipeline,” he told us, adding “other applications include engineering host systems for improved bioproduction of recombinant proteins and antibodies, for example in engineered CHO cells.”