08:00 Mon 08 Jul 2019
Horizon Dis Grp plc - SMARTvector platform supports Celyad IND filing
· Phase 1 trial scheduled for early 2020 will be first CAR-T cell therapy clinical trial to employ SMARTvector technology
· Horizon will receive a milestone payment for the successful IND filing of CYAD-02, autologous NKG2D-based CAR-T candidate
Celyad has been investigating the use of shRNAs to support the clinical development of its CAR-T cell platform. The FDA approved IND application involves CYAD-02, a next generation CAR-T cell therapy in which shRNA is employed to suppress two genes. Celyad has pre-clinical data indicating that this improves in vivo engraftment and efficacy of CYAD-02.
A Phase 1 dose-escalation trial evaluating the safety and clinical activity of CYAD-02 is planned for early 2020 and will involve a preconditioning chemotherapy (CyFlu) in patients with relapse/refractory (r/r) acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).
ENDS
About the use of Horizon's SMARTvector™ shRNA technology in gene therapy
RNA interference (RNAi) is a well-established technique in which RNA molecules silence gene expression by neutralizing targeted messenger RNA (mRNA) molecules, preventing the production of functional proteins. The ability of synthetic (artificial) short hairpin RNAs (shRNAs) to provide specific and prolonged gene silencing opens up the possibility of inhibiting mRNAs with key roles in disease progression: thus shRNAs can be important mediators of gene therapy. Horizon's SMARTvector shRNA technology uses a SMARTvector shRNA algorithm to rationally design target shRNA sequences with high specificity to ensure optimal gene silencing.
The SMARTvector platform originates from the product portfolio of
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Built upon more than a decade of experience in the engineering of cell lines, Horizon offers an unmatched portfolio of tools and services to help scientists gain a greater understanding of gene function, identify genetic drivers behind human disease, deliver biotherapeutics, cellular and gene therapies for precision medicine as well as develop and validate diagnostic workflows.
Horizon's solutions enable almost any gene to be altered, or its function modulated, in human and other mammalian cell lines.
The Company's customers include many of the world's foremost academic institutes, global pharmaceutical and biotechnology companies as well as clinical diagnostic laboratories. Insight into the challenges faced by these organizations enables Horizon to focus efforts on development of innovative solutions that not only differentiate the Company's offering, but also fuel development of the next wave of precision medicines.
Horizon is headquartered in
About Celyad
Celyad is a clinical-stage biopharmaceutical company focused on the development of specialized CAR-T cell-based product candidates and utilizes its expertise in cell engineering to target cancer. Celyad's CAR-T cell platform has the potential to treat a broad range of solid and hematologic tumors. The company's lead clinical candidate, CYAD-01, an autologous NKG2D-based CAR-T therapy, is currently being evaluated in several Phase 1 clinical trials to assess safety and clinical activity for the treatment of hematological malignancies, such as acute myeloid leukemia, and solid cancers, such as metastatic colorectal cancer. Celyad is also developing CYAD-101, an investigational, non-gene edited, allogeneic (donor derived) NKG2D-based CAR-T therapy, which is currently being evaluated in a Phase 1 trial for the treatment of patients with metastatic colorectal cancer. Celyad was founded in 2007 and is based in Mont-Saint-Guibert,
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