Precision BioSciences bags $110M to take off-the-shelf CAR-T into humans

Precision BioSciences has raised $110 million. The series B round sets the genome editing specialist up to take its lead, off-the-shelf CAR-T therapy into clinical trials.

North Carolina-based Precision is built around Arcus, a genome editing platform that uses a synthetic mimic of the homing endonucleases found in nature to make insertions, deletions or other edits to DNA. Precision thinks its approach is more specific than competing gene-editing technologies, cutting the risk of off-target activity and opening new opportunities in oncology and gene therapies.

The startup has persuaded some big names to support its pursuit of these opportunities. Having tapped investors including venBio and the VC wings of Amgen and Baxter for $26 million in 2015, Precision has now put together a series B led by ArrowMark Partners with assists from Gilead and a clutch of other financiers.

Precision has put together the syndicate to bankroll the next phase of its evolution, in which it will start testing an off-the-shelf CAR-T in humans. The plan is to use Arcus’ ability to make DNA deletions and insertions to create a CAR-T therapy from T cells donated by healthy donors, not cancer patients. Other biotechs, notably Cellectis and Arie Belldegrun’s Allogene Therapeutics, are working on similar therapies. But Precision thinks there is room in the sector for its cells, which it foresees modifying to zero in on tumors and avoid immune attacks.

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In parallel, Precision is working to get a gene therapy into IND-enabling studies. While the first wave of gene therapies are seeking to deliver missing or defective genes, Precision plans to send its genome editing technology into the body to modify the patient’s source code in a variety of ways. This approach could enable Precision to correct or delete mutated genes, or remove whole sections of disease-causing DNA.

Precision thinks its approach to gene therapy will expand the range of addressable diseases and ensure treatments permanently address the underlying problem. There are unanswered questions about the ability of gene therapies currently in development to trigger such durable effects.

As a preclinical company, there are even more unanswered questions about Precision and its Arcus platform. But with investor commitments totaling $110 million, Precision is now positioned to start validating its potential.