J&J gets piggyBac from Transposagen to develop T-Cell therapies

By Dan Stanton

- Last updated on GMT

A human T lymphocyte (also called a T cell)
A human T lymphocyte (also called a T cell)

Related tags Gene therapy Biotechnology Dna

There is an upsurge in biopharma demand 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 currently 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 tools.

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 binds 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 deliver 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 tools in terms of safety, precision, and efficiency.

The technology is completely reversible, he said, “essentially having a built-in safety switch, which is important for use in humans.” ​Furthermore, when combined with the firm’s Footprint-Free Gene Editing System, the firm 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 offered 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 firm is seeing a general upsurge in demand 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.”

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