By GREGORY ZELLER //
Marking a big step in the war against cancer, a Stony Brook-based startup has initiated pre-clinical development of a non-viral, plasmid-free chimeric antigen receptor modified T-cell manufacturing platform.
In English: A novel platform designed to develop and manufacture new pharmaceutical treatments, including treatments for different forms of cancer.
Noting “a very important announcement for the future of cancer treatment,” Applied DNA President and CEO James Hayward said Tuesday the new DNA-manufacturing platform will pioneer a new generation of faster, more affordable chimeric antigen receptor modified T-cell (CAR T) therapeutics – starting with an exclusive North American licensing and research-services agreement LineaRX has signed with Stony Brook-based iCell Gene Therapeutics, also announced Tuesday.
iCell Gene Therapeutics, which works closely with China-based researchers on the development of chimeric antigen receptor-engineered cells for therapeutic purposes, will collaborate with LineaRX on the development and commercialization of LinCART19, a non-viral, plasmid-free anti-CD19 CAR T drug candidate.
The B-lymphocyte CD19 is a protein found on the surface of B-cells (a type of white blood cell) and serves as both a biomarker for lymphoma (a cancer of the lymphatic system) and an increasingly promising target for leukemia immunotherapies.
The science is dense but the bottom line is clear, according to Hayward, who notes that since they were approved by the FDA in 2017, CAR T therapies “have shown unrivaled efficacy against several forms of cancer” – although manufacturing them is “extremely complex” and “often cost-prohibitive.”
Enter the new CAR T manufacturing platform, which aims to alter the dynamics of that critically important playing field.
“With our expertise in the large-scale [polymerase chain reaction]-based production and modification of DNA constructs, we believe we are in a unique position to offer a disruptive, inexpensive and safer alternative to established CAR T manufacturing platforms that rely on viral vectors and plasmids,” Hayward said.
Despite the limitations of those existing manufacturing platforms, which are both costly and inefficient, the CAR T market is already experiencing rapid growth and capital investment. Industry watchdog Bioinformant reports that 2018 has been a busy year for CAR T-related IPOs and investments, with market capitalization already exceeding $20 billion.
Into those warming waters wades LineaRX, which according to Stephen Hughes, Applied DNA’s director of DNA programs, has the breakthrough tech to reset the entire industry.
“CAR T therapies are changing the treatment of cancer, but they must be affordable and accessible to all patients,” Hughes noted. “We believe the approach of the LineaRx team will alter the manufacturing of these life-changing therapies, increasing the speed and safety of production while lowering the costs.”
LineaRX also hopes to race ahead of the burgeoning field by leveraging iCell Gene Therapeutics’ experience in engineering novel, first-in-class adoptive-cell therapies. The company has already commenced Phase I clinical trials on a new generation of chimeric antigen receptors that, in tandem with a “tumor vaccine,” aim to create a “whole body defense” against multiple myeloma and B-cell malignancies.
The circa-2014 clinical-stage R&D company – which collaborates with Stony Brook Medicine, Chengdu Military General Hospital in China and the National Institutes of Health, among others – is also conducting Phase I trials of new combinations of multiple CARs designed to prevent tumor relapses associated with leukemia and other disorders.
That sort of aggressive, ready-to-rock end-user is precisely whom LineaRX has in mind as it rolls out its new CAR T manufacturing platform, according to Hayward.
“We are excited to have begun pre-clinical development of our [non-viral, plasmid-free] CAR T manufacturing platform to enable a new generation of cell therapies,” the CEO said. “We believe our NVPF approach to generate important biotherapeutics without using viruses and plasmids … can redefine ‘bench to bedside’ therapy.”