Horizon Discovery’s SMARTvector shRNA technology deployed in Celyad’s successful IND filing for next-generation CAR-T cell therapy, CYAD-02

Cambridge, UK, 8 July 2019: Horizon Discovery Group plc (LSE: HZD) (“Horizon”), a company driving the application of gene editing and gene modulation within the global life science market, today announced that Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, received FDA Acceptance of Investigational New Drug (IND) application for the autologous NKG2D based CAR-T cell therapy CYAD-02 that deploys Horizon’s optimized SMARTvector™ shRNA technology. The Phase 1 trial will be the first CAR-T cell therapy to employ the SMARTvector platform. Horizon will receive an undisclosed milestone payment for the successful IND filing.


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).


Terry Pizzie, Chief Executive Officer, Horizon Discovery Group plc, commented: “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.”


Filippo Petti, Chief Executive Officer, Celyad, said: Over the past few years Celyad has made great strides in evaluating our NKG2D-based CAR-T therapy 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 optimized shRNA developed using Horizon’s SMARTvector technology represents the output of a strong collaboration. We look forward to a continued successful relationship.”




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 Dharmacon - a company that is now part of Horizon Discovery.


For further information from Horizon Discovery Group plc, please contact:


Horizon Discovery Group plc

Terry Pizzie, Chief Executive Officer

Jayesh Pankhania, Chief Financial Officer

Jon Davies, Head of Investor Relations

Tel: +44 (0) 1223 655 580


Zyme Communications (Trade and Regional Media)

Katie Odgaard

Tel: +44 (0)7787 502 947

Email: katie.odgaard@zymecommunications.com


Consilium Strategic Communications (International Financial PR)

Mary-Jane Elliott, Matthew Neal

Tel: +44 (0)203 709 5700

Email: horizon@consilium-comms.com


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About Horizon Discovery Group plc www.horizondiscovery.com

Horizon Discovery Group plc (LSE: HZD) ("Horizon") drives the application of gene editing and gene modulation within the global life science market – supporting scientists on the path from research to therapy.


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 Cambridge, UK with offices in USA and Japan.  The Group is listed on the London Stock Exchange's AIM market under the ticker HZD.


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, Belgium, and New York, NY. Celyad’s ordinary shares are listed on the Euronext Brussels and Euronext Paris exchanges, and its American Depository Shares are listed on the Nasdaq Global Market, all under the ticker symbol CYAD.