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.