Science and Technology cooperation Exploration: Global Science and Technology Project (Phase 4)

In the rapidly developing field of science and technology, innovative projects are constantly emerging. In recent years, there have been significant opportunities in all aspects surrounding cutting-edge technology. International cooperation in science and technology is of great significance in jointly solving global problems, accelerating scientific and technological progress, promoting economic development, promoting personnel training and exchanges, and building a scientific and technological community. The PAI community is committed to building a bridge of scientific and technological exchange, solving common challenges, promoting international cooperation and development, and promoting world peace and prosperity.

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* Source: China International Scienceand Technology Cooperation http://www.cistc.gov.cn/

01 / Co-construction of the Centre for Materials Science (EFAML), Siriraj Center of Interventional Radiology (SICIR)

Project summary:

Indian Institute of Science and Educational Research (IISER) Kolkata Branch is one of the seven branches of IISER, and its research and development strength ranks at the forefront of India, with outstanding research and development strength in the field of chemistry.

In recent decades, pioneering collaborative research conducted by Professor Soumyajit Roy of the Materials Science Centre (EFAML) at the Kolkata Institute of Technology in India with Chinese universities and enterprises has yielded fruitful results. In view of these achievements, the Indian side proposed the establishment of the Siriraj Center of Interventional Radiology (SICIR)

to enhance cooperation and exchange between institutions and promote further collaborative research.

02 / Stem cell and organoid research

Project summary:

The Children's Medical Research Institute of Australia (CMRI), formerly known as the Children's Medical Research Foundation, was established in 1958 and later renamed the Institute. The Institute has 132 researchers and has international cooperation with 790 institutions around the world. CMRI established Australia's first neonatal research center, established a joint venture gene therapy research center, and conducted the first gene therapy clinical trial for genetic diseases in Australia. The telomerase component was identified for the first time, and 15% of cancers were found to depend on the telomere replacement elongation (ALT) mechanism. Establishing translational vector science and vector and genome engineering facilities to advance gene therapy; Developed and implemented the first gene therapy for an inherited eye disease and the first cell therapy for vision loss; Launch of the world's first 7-year program to transform cancer diagnosis and personalized treatment.

Organoids are tissue analogues with certain spatial structure formed by three-dimensional culture of adult stem cells or pluripotent stem cells in vitro. With the emergence of induced pluripotent stem cell (iPSC) technology, IPSC-derived organoids can better reconstruct and mimic the structure and physiological mechanism of human organs in vitro, provide a unique model for human disease research, and promote the optimization, reduction and replacement of animal tests.

The CMRI Stem Cell Medicine Group (SCM) focuses on retinal, inner ear, and retina-brain organoids and is able to use human cell phenotypes to generate hundreds of human organoids for screening and omics, elucidate disease mechanisms, conduct pharmacological screening, and test new therapies. Optimization of gene therapy organoid AAV vector delivery; Using pluripotent stem cells (IPscs and embryonic stem cells) rather than adult stem cells (the latter have limited ability to differentiate); Generating retinal organoids containing transplantable photoreceptor cells for gene therapy; Innovative optimization schemes for retinal organoid proteome and single-cell RNA sequencing; High throughput microelectrode arrays (MEA) were used to evaluate excitatory cell function.

SCM now has a proprietary Stem Cell and Organoid Facility (SCOF) dedicated to enabling high-throughput automated organoid generation and evaluation research to unlock the vast potential of regenerative medicine.

The project is currently in the laboratory results stage and has applied for invention patents. The foreign party hopes to cooperate through technology investment and investment.

03 / AAV gene therapy clinical vector development and production

Project summary:

The Children's Medical Research Institute of Australia (CMRI), formerly known as the Children's Medical Research Foundation, was established in 1958 and later renamed the Institute. The Institute has 132 researchers and has international cooperation with 790 institutions around the world. CMRI established Australia's first neonatal research center, established a joint venture gene therapy research center, and conducted the first gene therapy clinical trial for genetic diseases in Australia. The telomerase component was identified for the first time, and 15% of cancers were found to depend on the telomere replacement elongation (ALT) mechanism. Establishing translational vector science and vector and genome engineering facilities to advance gene therapy; Developed and implemented the first gene therapy for an inherited eye disease and the first cell therapy for vision loss; Launch of the world's first 7-year program to transform cancer diagnosis and personalized treatment. Gene therapy refers to the introduction of foreign normal genes into target cells to correct or compensate for diseases caused by defects and abnormal genes, so as to achieve therapeutic purposes, and bring cure possibilities for some "incurable" diseases. Currently commonly used as a gene therapy tool is a generation of rAVV (recombinant adeno-associated viruses, such as AAV2, AAV8), which is characterized by natural and good transduction capabilities, tissue specificity, and safety record. However, these viruses that exist in nature have not yet evolved into vectors with gene therapy function, resulting in low clinical research efficiency and production efficiency, resulting in low clinical effectiveness, and increasing the risk of toxicity and immune response caused by increasing vector dose to achieve efficacy. CMRI has partnered with the Sydney Children's Medical Alliance (SCHN) on the gene therapy program. The program focuses on AAV-based gene delivery technology, covers the whole process from vector development to production, has applied for more than 10 patents, and realizes the development of double transplant models, which can develop customized vectors for a large number of clinical tissue targets. The joint research team has the world's leading hardware and software experimental facilities, original human tissue bank and more than 30 researchers in molecular biology, cell biology, stem cell biology, genome engineering, immunology and bioinformatics and QA, QC, CMC process developers, GMP carrier production pilot and pilot scale up to 25L and 200L. The project is currently in the laboratory results stage, has achieved small-scale trial production, has authorized invention patents, and the foreign party hopes to cooperate in technology licensing, cross-training, academic cooperation, short-term research projects, consulting services and other aspects.

04 / ProCan? Tumor proteome full type high-throughput assay analysis project

Project summary:

The Children's Medical Research Institute of Australia (CMRI), formerly known as the Children's Medical Research Foundation, was established in 1958 and later renamed the Institute. The Institute has 132 researchers and has international cooperation with 790 institutions around the world. CMRI established Australia's first neonatal research center, established a joint venture gene therapy research center, and conducted the first gene therapy clinical trial for genetic diseases in Australia. The telomerase component was identified for the first time, and 15% of cancers were found to depend on the telomere replacement elongation (ALT) mechanism. Establishing translational vector science and vector and genome engineering facilities to advance gene therapy; Developed and implemented the first gene therapy for an inherited eye disease and the first cell therapy for vision loss; Launch of the world's first 7-year program to transform cancer diagnosis and personalized treatment. ProCan? It is the world's first and only project facility to generate high-throughput, full-type human cancer proteomic data from a single platform, equipped with six high-end mass spectrometers for SWATH-MS and multiple PCT machines that support 24/7 operation and can quickly generate high-quality, repeatable proteomic data from a small number of cancer samples within 24-36 hours.

ProCan? It can be applied to existing diagnostic processes and is expected to replace most or all of the current pathologic diagnosis of cancer based on protein determination, identify proteomic features and new therapeutic targets, improve the drug development process, reduce the cost and time of clinical trials, and help oncologists develop precision medicine protocols and improve treatment outcomes. It also reduces the direct and indirect costs of patient testing and care and avoids the side effects of ineffective therapies.

The project is currently in the laboratory results stage, has achieved small-scale trial production, and authorized invention patents, the foreign party is now looking for companies engaged in early oncology trials to carry out relevant cooperation, hoping to cooperate through technology investment, investment and other ways.