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SMARTvector Technology Deployed in Successful IND Filing for Next-Generation CAR-T Cell Therapy

Phase 1 trial scheduled for early 2020 will be first CAR-T cell therapy clinical trial to employ SMARTvector technology

10 Jul 2019
Jay Leech
Marketing / Sales

Industry news

Horizon Discovery Group plc (LSE: HZD) (“Horizon”), a company driving the application of gene editing and gene modulation within the global life science market, have 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.”

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Biopharmaceutical AdvancesBiopharmaceutical advances follow the development of pharmaceuticals derived from biotechnology, also known as biotechnology medicines. Biopharmaceuticals may be produced from cell lines, plants, or microbial cells. Important considerations of biopharmaceutical use include application, cost, production process and purification.Clinical TrialsClinical Trials, an essential part of drug discovery process, assess the safety and effectiveness of a new medication or device in the pharmaceutical industry. Clinical Trials are a phased process (Phase 0, Phase I, Phase II, Phase III and Phase IV) which begins after initial preclinical testing.LeukemiaBiopharmaceuticalsBiopharmaceuticals are proteins and other compounds (such as nucleic acids) produced by living organisms that have uses as therapeutics or for in vivo diagnostics. The most well known example of a biopharmaceutical product, and the first to be approved for therapeutic use, was recombinant human insulin.Cell TherapyCell therapy involves using living cells to treat diseases, often by replacing damaged cells or stimulating regeneration. Stem cell therapy and CAR-T cell therapy are examples of cutting-edge treatments in regenerative medicine and cancer immunotherapy. Browse our peer-reviewed product directory to find the best cell therapy tools, compare products, check reviews, and get pricing directly from manufacturers.Gene EditingGene editing is the precise alteration to an organism's DNA, with CRISPR being the most well-known technique. It is used in a wide range of applications, including the development of disease models and gene therapy, and crop improvement. Browse our peer-reviewed product directory to find the best gene editing solutions, compare products, check reviews, and get pricing directly from manufacturers.ChemotherapyCancer ResearchAlthough cancer is often referred to as a single condition, it actually consists of more than 100 different diseases. Microscopy, mass spectrometry, high throughput sequencing and flow cytometry are some of the most common techniques employed in cancer research labs.

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