Celyad, a clinical-stage biopharmaceutical company, has received investigational new drug (IND) approval from the US Food and Drug Administration (FDA) for its autologous NKG2D-based CAR-T cell therapy, CYAD-02, which uses Horizon Discovery’s SMARTvector shRNA technology.
The technology uses a SMARTvector short hairpin RNA (shRNA) algorithm to design target shRNA sequences with high specificity to ensure optimal gene silencing, according to the company.
The Phase I dose-escalation trial will involve a preconditioning chemotherapy (CyFlu) in patients with relapse/refractory (r/r) acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). It is expected to begin in early 2020.
Horizon Discovery CEO Terry Pizzie, said in a statement: “We see great potential for shRNA technology in the optimization 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.”
As per the companies’ agreement, Horizon will receive an undisclosed milestone payment for the successful IND filing.
