Biopharmaceutical firm Celyad has received FDA approval for its CAR T-cell therapy to begin human trials.

The early-stage cancer treatment, which comes under the category of immunotherapy, will use gene-editing company Horizon’s SMARTvector technology that uses shRNAs, which have a tight hairpin turn that can be used to silence target gene expression

The software equips researchers with tools for delivering and expressing genetic content in their cells of interest.

Terry Pizzie, CEO at Horizon Discovery Group, said: “We see great potential for shRNA technology in the optimisation of next-generation cell therapies.

“The success of this IND filing is testament to the strength of our relationship with Celyad, and the powerful combination of Horizon’s SMARTvector shRNA platform with Celyad’s CAR-T expertise.”

Filippo Petti, CEO at Celyad, added: “Over the past few years Celyad has made great strides in evaluating our CAR-T therapy, based on NKG2D (a transmembrane protein), for the treatment of relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

“The FDA approval for the CYAD-02 IND application will allow us to evaluate new therapies in this difficult to treat population and the inclusion of an optimised shRNA developed using Horizon’s SMARTvector technology represents the output of a strong collaboration.”


What is CAR T-cell therapy?

Called chimeric antigen receptor T-cell, CAR T-cell therapy is a type of treatment in which a patient’s T cells – a type of immune system cell – are altered in the laboratory so they can recognise and attack cancer cells specifically.

Celyad has been investigating the use of shRNAs to create these therapies since October last year.

The NHS considers the new reprogramming treatment as “highly complex and potentially risky”, but it has been shown in trials to cure some patients, even those with quite advanced cancers and where other available therapies have failed.

The “SMARTvector” platform was developed and optimised by Dharmacon, a Horizon Discovery company, who screened naturally-occurring human microRNA to select a scaffold that allows for efficient processing of the shRNA from the organism and the expression of all its components.

The Phase 1 trial evaluating the safety and clinical activity of candidates is planned for early 2020 and will involve a preconditioning chemotherapy in patients with relapse/refractory, acute myeloid leukemia or myelodysplastic syndromes.