30 years from now, small molecule drugs and antibody drugs will no longer be mainstream?

The Bio International Convention is one of the largest annual events in the biotechnology and pharmaceutical industriesAffected by the new crown outbreak, local time on June 8, BIO 2020 conference opened onlineIn an online forum on the first day of the conference called "Next Generation Medicine: Cell Therapy, Gene Therapy, and others," DrJames Sabry, Roche's head of global medicine, made the "amazing" prediction that "while small molecules and antibody therapies will still exist in 30 years, their importance will diminish." Cell and gene therapy will be the mainstream of future therapies! "
there is no doubt that cell and gene therapy, as an emerging treatment model, is getting widespread attention in the industrySo what's the status quo in cell and gene therapy? What improvements need to be made to realize the future that DrSabry predicts?According to IQVIA's 2019 Research and Development Achievements (2019 R-D Achievements), the new generation of biotherapeutics (next generation Biotherapeutics, NGB), represented by cell therapy, gene therapy and nucleotide therapy, has grown rapidly in the last five yearsIn 2019, The proportion of NGB in the post-clinical research and development pipeline will be close to 12%, further increasing on the 2018 base (10%)In 2019, 99 products in research into the post-clinical research and development pipeline, will increase the number of products under development to 369, compared with 2014, the number of three timesat present, 78% of the NGB post-clinical research and development pipeline is in the stage of Phase 2 clinical developmentIt's worth noting that these NGB therapies are more likely to be approved by the FDA without a Phase 3 clinical trial than other traditional ongoing therapiesBecause the FDA believes that these therapeutic therapies are designed to treat patients with diseases with severely unmet medical needsThis means that while nGB therapies are in the lower number of Phase 3 clinical trials, they may be closer to being approved for early-onset benefit than non-NGB therapiesin NGB clinical development projects, the main research areas are oncology, gastrointestinal, hematology and ophthalmologyOncology remains the dominant area of research in the NGB post-research pipeline, with 128 products, mainly from CAR-T cell therapy, including "ready-to-use" CAR-T therapy and CAR-T therapy manufactured using CRISPR technologygene and cell therapy for the gastrointestinal tract accounted for 9 percent of the NGB's post-development pipeline This includes 21 gene therapies, which are mainly used to potentially cure infants or children with deadly genetic diseases, including mucosaclymic storage disease, Fabri disease and phenylketonuria in hematology, 21 gene therapies, or CRISPR gene-editing therapies, are in development by the end of 2019 Gene therapy for haemophilia and thalassemia is expected to be approved for sale this year inspired by The success of Luxturna, 24 gene therapies in the field of ophthalmology are currently in the late stages of development CRISPR gene editing therapy for Leber's congenital black haze has also entered the clinical development phase now, a number of large pharmaceutical companies are working with small and medium-sized biotech companies focused on cell and gene therapy development to drive further innovation in this area Roche, for example, completed its acquisition of Spark Therapeutics, a gene therapy star, last year in the development of gene therapy, many existing gene therapies use adenoviruses (AAVs) as vectors Dr Federico Mingozzi, Chief Scientific Officer of Spark Therapeutics, another guest on The Next Generation Of Medicine: Cell Therapy, Gene Therapy and others, said, "We have a lot of room to improve the specificity and effectiveness of AAV vectors by retrofitting AAV shell proteins." He believes that the next generation of gene therapy delivery platforms will allow gene therapy to treat more diseases Spark has published its latest research on Nature Medicine, using an IgG degradation enzyme called IdeS to quickly and temporarily eliminate neutralizing IgG antibodies against AAV, expanding the population of patients treated with gene therapy and possibly opening a window for repeated administration Roche's partnership with 4D Molecular Therapeutics (4DMT) also demonstrates a research and development direction for further improvement of AAV carriers Through a strategy of directional evolution, 4DMT selects AAV shells that are "tailored" for specific diseases through a library of 1 billion different AAV shell proteins Not only are they higher tissue specificity, but they are not affected by neutralizing antibodies in the patient's body and are safer in the delivery of therapy The innovative gene therapy 4D-110, developed jointly by 4DMT and Roche, can effectively deliver gene therapy to retinal tissue without being injected into the retinal tissue Not only is it safer, but it may also help patients recover their vision better Eventually, Dr Sabry says, AAV carriers may eventually be replaced by more efficient delivery systems In his vision of the future, genetic "surgery" can be as common as surgery today The third guest on the online forum was Dr Harlan Robins, co-founder and chief scientific officer of Adaptive Biotechnologies Last year, Roche and Adaptive Biotechnologies agreed a $2 billion research and development partnership to develop individualized cell therapies According to Dr Harlan Robins, the body's adaptive immune system is not only a disease diagnosis system that nature has given us, but also a powerful means of treating diseases The TruTCR technology platform of Adaptive's company enables high-throughput analysis of T cells in the immune system to identify T-cell receptors (TCR) associated with any clinical target Combining this technique with TCR cell therapy technology, TCR in cancer patients can be found for new antigens for cancer, and then develop truly individualized cell therapy Dr Sabry says that while there are doubts about the efficacy and toxic side effects of cell therapy, this is because we don't know enough about the immune system's regulatory network If we have a deeper understanding of the mechanisms by which the immune system self-regulates, we can use the immune system's self-regulating function to regulate the effectiveness of cell therapy At that time, "some of the existing concerns about the efficacy or inadequacy of cell therapy will cease to exist." 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realize these visions of cell and gene therapy require the industry to take risks and continuously promote innovation, while partnerships and mergers and acquisitions between large pharmaceutical companies and small biotech companies provide resources and opportunities to transform innovative technologies into treatments that benefit patients more quickly." we look forward to continuous innovation that will enable cell and gene therapy to usher in a better future for the benefit of more patients