RNA - Genetherapys

ShanghaiTech University Welcomes National Center President Rosario Rizzuto

Exploring opportunities for international collaborations, the President of the National Center for Gene Therapy and Drugs based on RNA Technology Foundation, Prof Rosario Rizzuto arrives in China to meet with delegates and faculty members from ShanghaiTech University and distinguished leaders across related fields.

Sharing the same National Center goals of enriching research and innovation, the visit offers an opportunity for Prof Rizzuto to tour the Shanghai Institute for Advanced Immunochemical Studies (SIAIS), which focuses on the basic structure and design of biological molecules. This state-of-the-art research institute aims to improve the quality of human life through basic research and fundamental science by focusing on four core research areas; immuno-chemistry, cell biology, computational biology & artificial intelligence, and structural biochemistry.

Prof Rizzuto then visits the ShanghaiTech University School of Biomedical Engineering (BME), where faculty members offer an educational platform to inspire a new generation that applies the principles and problem-solving techniques of engineering to biology and medicine.  Continuing to the Shanghai Clinical Research and Trial Center (SCRTC), Prof Rizzuto receives insight into how the truly small-scale university, with less than 2000 undergraduate students and under 4000 graduate students, prepares them to become professionals in clinical and medical research that is uniquely integrated alongside a broad range of Chinese healthcare services.  To achieve this level of comprehensive patient care, the Research Hospital of ShanghaiTech University established six specifically focused diagnosis and treatment centers covering Cancer, Neurological diseases, Pan-vascular diseases, Respiratory system diseases, Rheumatic, Immune & Musculoskeletal diseases, and Sensory system diseases.

Meeting with the Founder and CEO of the Chinese biopharmaceutical startup company, Prof Rizzuto witnesses how InnoStellar Biotherapeutics focuses on improving the lives of those with genetic and chronic diseases through the development and manufacturing of innovative gene therapy drugs. As the Chinese startup has successfully begun clinical trials in the treatment of hereditary retinal dystrophy, their meeting draws particular interest from researchers within Spoke 1 concentrating on Work Package 1.3 who aim at developing gene therapy and RNA-based therapeutics for rare eye diseases.

Touring the facilities of CorrectSequence Therapeutics (Correctseq) allows Prof Rizzuto to view five of the Chinese biotech company base editing systems, including enhanced Base Editor (eBE), dCpf1 Base Editor (dCpf1-BE), human APOBEC3A Base Editor (hA3A-BE), BEACON, and its transformer base editor (tBE). Capable of inducing efficient editing with no observable genome-wide or transcriptome-wide off-target mutations in human cells or animal models that only become active at on-target sites, which may greatly expand the scope of tBE’s clinical application.

While RongCan Biotech focuses on the development and industrialization of nucleic acid-based therapeutics, the visit by Prof Rizzuto gives the National Center insight into a company striving to become a one-stop shop covering the design and synthesis of mRNA sequencing, LNP-based delivery systems, to final mRNA-based pharmaceutical products.  Open to collaborations with hospitals, prevention and control units of infectious diseases, scientific research institutions, and organizations eager to offer scientific, clinical, and business continuity for clinical trial sponsorship, the visit could lead to joint research collaborations and international partnerships.

Between visits to academic facilities, research hospitals, and innovative startups, Prof Rizzuto returns to Italy with a fresh perspective on optimizing, exploring, designing, and developing gene therapy and drugs based on RNA technology.

Foundation staff writer Ellen Jane Corcoran

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