The quest for CAR-T 2.0 is gaining an mRNA player, as Cambridge, Massachusetts-based Factor Bioscience sends a spinoff racing toward the clinic.
Factor drew the curtains on Exacis Biotherapeutics on Wednesday morning, with Sollis Therapeutics co-founder Gregory Fiore at the helm of a small immuno-oncology focused team built around Factor’s technology. The spinoff has the rights to 51 patents and just a bit of seed money from friends and family to get it going — but Fiore says an IND submission is on the horizon.
“We are 18 to 24 months from an IND submission, and we’ve identified our first target, which will be CD19,” Fiore told Endpoints News.
The company will be unveiling a CD19-targeted CAR-T and CAR-NK, Fiore said, with ROR1 as its next target.
The CEO says Exacis’ approach is what differentiates it from others in the crowded cell therapy field, beginning with mRNA technology in-licensed from Factor. The process starts with induced pluripotent stem cells (iPSC), which are blood or skin cells that have been engineered back into an embryonic-like stem cell state. They’re created with mRNA reprogramming, and then edited to avoid host immune surveillance, add a CAR and enhance the cells for potency against tumors.
“That iPSC is quite a robust cell. It can handle a lot of editing and the cells are able to recover from a lot of editing and manipulation,” Fiore said. And the fact that no viruses or DNA are used “significantly decreases the resource requirement for manufacturing,” he added later.
The idea of an off-the-shelf CAR-T or CAR-NK therapy — as opposed to harvesting a patient’s cells, engineering them into a cancer attack vehicle and reinjecting them — isn’t a new one. Allogene released a positive snapshot of their off-the-shelf CAR-T program at ASCO 2020, and CRISPR Therapeutics offered a glimpse at their own CAR-T success in October although it was clouded by the death of a patient given a high dose of the treatment.
Exacis’ team of four — including co-founder James Pan and former MaxiVAX CEO Dimitrios Goundis as CBO — is shooting for a Series A in the coming months to bolster its team and pipeline. The company also says it’s in talks with several potential development partners.
“We are working towards a Series A funding to be completed in Q1 of 2021, and we’ll use those funds to build out our internal team and lab, as well as further the development along the lines of differentiation into T and NK, obtaining CARs, really putting together these target cell types,” Fiore said.
While Fiore stayed mum about the the specific terms of Exacis’ licensing deal with Factor, he said that Factor has a majority ownership in exchange for the execution of the license.
The CEO, who was inspired to get into the field by his father’s battle with cancer, said Factor and Exacis’ incentives were aligned. “There’s plenty of opportunity to improve the patient experience as well as outcomes,” he said.