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    Home > Medical News > Latest Medical News > 30 years from now, small molecule drugs and antibody drugs will no longer be mainstream?

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

    • Last Update: 2020-08-01
    • Source: Internet
    • Author: User
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    There is no doubt that cell and gene therapy, as an emerging treatment model, is getting wide attention from the industry.
    What is the current state of the field of cell and gene therapy? What improvements need to be made to realize the future that Dr. Sabry predicts?The status of cell and gene therapy According to IQVIA's 2019 Research and Development Achievements (2019 R and D Achievements) report, the new generation of biotherapies (next generation biotherapeutics, NGB), represented by cell therapy, gene therapy and nucleotide therapy, has grown rapidly over the past five years.
    in 2019, The proportion of NGB in the post-clinical research and development pipeline will be close to 12%, with a further increase in the 2018 base (10%).
    2019, 99 in-research products 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 times.
    at present, 78% of the NGB post-clinical research and development pipeline is in the stage of Phase 2 clinical development.
    note that these NGB therapies are more likely to be approved by the FDA without a Phase 3 clinical trial than other traditional research therapies.
    because the FDA believes these in-the-form therapies are designed to treat patients with diseases with serious unmet medical needs.
    this means that while nGB therapies are in the lower number of Phase 3 clinical trials, they may be closer to being approved for early-term benefit than non-NGB therapies.
    in NGB clinical development projects, the main research areas are oncology, gastrointestinal, hematology and ophthalmology.
    Oncology remains the leading 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 made using CRISPR technology.
    gene and cell therapy for the gastrointestinal tract accounted for 9 percent of the NGB's post-development pipeline.
    include 21 gene therapies that are used primarily to potentially cure infants or children with deadly genetic diseases, including mucosaclymic storage disease, Fabri disease and phenylketonuria.
    in the field of hematology, 21 gene therapies, or CRISPR gene-editing therapies, are under development by the end of 2019.
    , gene therapy for haemophilia and thalassemia is expected to be approved for sale this year.
    inspired by Luxturna's success, 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 clinical development.
    make gene "surgery" as common as surgery, several large pharmaceutical companies are already working with small and medium-sized biotech companies focused on cell and gene therapy to drive further innovation in the field.
    take Roche, for example, which last year completed its acquisition of Spark Therapeutics, a gene therapy star.
    in the development of gene therapy, many existing gene therapies use adeno-related viruses (AAVs) as vectors. "We have a lot of room to improve the specificity and effectiveness of AAV vectors by transforming the AAV shell protein," said Dr. Federico Mingozzi, chief scientific officer of Spark Therapeutics, another guest on the
    "Next Generation Medicine: Cell Therapy, Gene Therapy and other" online forum.
    " he believes the next generation of gene therapy delivery platforms could allow gene therapy to be used to treat more diseases.
    Spark, which recently published its latest research on Nature Medicine, uses 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 way to further improve the aAV carrier.
    4DMT selects AAV shells "tailored" for specific diseases through a library of 1 billion different AAV shell proteins through a strategy of targeted evolution.
    they not only have higher tissue specificity, but are not affected by neutralizing antibodies in the patient and safer in the delivery of therapy.
    the innovative gene therapy 4D-110, developed jointly by 4DMT and Roche, can be effectively delivered to retinal tissue without being injected into the retinal tissue.
    is not only safer, but may also help patients recover their vision better. Eventually, Dr.
    Sabry says, AAV vectors may be replaced by more efficient delivery systems.
    in his vision for the future, genetic "surgery" can be as common as surgery today. The third guest of the
    Individualized Cell Therapy Online Forum was Dr. Harlan Robins, Co-Founder and Chief Scientific Officer of Adaptive Biotechnologies.
    last year, Roche and The RapaDeion a $2 billion research and development partnership to develop individualcell therapies.
    In Dr. Harlan Robins's view, the body's adaptive immune system is not only the disease diagnosis system that nature has given us, but also a powerful way to treat diseases.
    The TruTCR technology platform of The Adaptive is able to perform high-throughput analysis of T cells in the immune system to identify T-cell receptors (TCR) associated with any clinical target.
    combined this technology with TCR cell therapy technology to identify TCR sized in cancer patients for new antigens for cancer, and then develop truly individualized cell therapies. 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-regulation function to regulate the effectiveness of cell therapy.
    by then, "some of the existing concerns about the efficacy or inadequacy of cell therapy will cease to exist."
    "Realizing these visions of cell and gene therapy requires 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.
    This article is an English version of an article which is originally in the Chinese language on and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.

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