Please login to the form below

Not currently logged in

Precision BioSciences to raise $100m for off the shelf CAR-Ts

Joins next wave of cell therapy competitors


Precision Biosciences is aiming to further develop its allogeneic (off the shelf) CAR-T immunotherapy candidate PBCAR0191, and it’s filed a $100m IPO to fund this.

The gene-editing start-up says the funding will allow the candidate to be brought into a phase 1/2 programme, initially assessing the drug in two cancer indications - acute lymphoblastic leukaemia and non-Hodgkin lymphoma (NHL).

This is Precision’s first clinical-stage candidate, but it targets a familiar protein – CD19, which is also the focus of already approved CAR-Ts Novartis’ Kymriah and Gilead’s Yescarta.

However, Precision, which has partnered up with French drugmaker Servier on the drug, says that its technique is backed up by its in-house tech ARCUS, which is derived from the natural genome editing enzyme homing endonuclease.

These endonucleases are site-specific DNA-cutting enzymes that are non-destructive in nature, and their main mechanism is to trigger gene modification events such as inserting and deleting DNA.

The company says the enzyme’s ability to target a single DNA break in a complex genome to achieve gene modification without random off-targeting makes these enzymes particularly attractive.

ARCUS, and the synthetic enzyme ARC nuclease, is built around this mechanism but it is “significantly improved to be the starting point for the first therapeutic-grade genome editing platform”.

ARC nucleases are small for varied delivery strategies, can be customised to recognise a DNA sequence within any target gene, and the company says that it doesn’t miss a target.

It’s a bold claim, but if the drug operates as claimed, then the company could offset some of the issues associated with first generation CAR-T therapies.

Current CAR-Ts available on the market are manufactured on a patient-by-patient basis, unique to each individual. Despite the success rate, the drug is very expensive and time consuming.

Unlike typical CAR-Ts, Precision BioSciences’ one-step genomic platform uses cells from healthy donors - reducing graft versus host reactions - which will translate to saving time and cutting costs.

Precision isn't short of competitors, however, with Gilead being one notable rival in the field, having entered a $445m deal last year to develop new hepatitis B candidates.

Article by
Gemma Jones

5th March 2019

From: Sales



COVID-19 Updates and Daily News

Featured jobs


Add my company

Langland, a Publicis Health company, is the only health communications agency that thinks further across Clinical Trial Experience, Medical Strategy...

Latest intelligence

Leveraging Real-World Evidence for Cell and Gene Therapies
Regulators and reimbursement bodies are increasingly turning to real-world evidence (RWE) to understand the long-term value of drugs, in particular novel therapies. Here, Mariam Bibi, Senior Director, Global RWE at...
Food Allergy – Is avoidance the only option?
Following on from Allergy Awareness Week 2022, we’re continuing the discussion about food allergies and the experiences of those who live with them. In this blog, we discuss the current...
Serious about sustainability?
Get to know our new Sustainability Officer here at Cuttsy+Cuttsy...