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    Home > Active Ingredient News > Drugs Articles > Small molecule breakthrough double anti-ADC outbreak Bio-phase change can achieve the next Nobel Prize

    Small molecule breakthrough double anti-ADC outbreak Bio-phase change can achieve the next Nobel Prize

    • Last Update: 2021-03-22
    • Source: Internet
    • Author: User
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    No matter which form of medicine has its own characteristics, it is not a question of who should eliminate it, and each has its own advantages.


    01 Small molecules break through the "unable to make drugs" target

    01 Small molecules break through the "unable to make drugs" target

    For the first time in the past two years, small molecules have broken through the "unable to drug" target KRAS.


    There is also PROTAC (targeted protein degradation) technology, the principle is very simple.


    This technology can overcome drug resistance at the application level.


    In the field of small molecules, there are also some breakthroughs in basic research, such as MicroED technology.


    Of course, whether any technology can bring application breakthroughs depends on the follow-up development, and how long it will take to bring real and substantial breakthroughs.


    02 The field of macromolecules: monoclonal antibodies are gradually entering the mature stage, and double antibodies and ADCs have exploded

    02 The field of macromolecules: monoclonal antibodies are gradually entering the mature stage, and double antibodies and ADCs have exploded

    In the field of macromolecules, the monoclonal antibody technology itself is slowly entering a mature stage.


    We can see a very rapid increase in the clinical number of bi-antibodies this year, and almost all antibody companies are trying to develop bi-specific antibodies.


    The other is the ADC field.


    There are already ten ADC drugs on the market, but why does 2020 suddenly become so hot? I think the biggest catalyst is the revolutionary success of Daiichi Sankyo’s DS-8201.


    Daiichi Sankyo’s DS-8201 is the first to prove to everyone that toxin molecules do not require PM-level activity, and can be compensated by connecting multiple toxin molecules and bystander effects to a single monoclonal antibody, which brings innovation in the ADC field.


    03 The results of cell therapy and gene therapy appear

    The third area is cell therapy.


    While CAR-T exerts a good effect, there are also some shortcomings.


    Gene therapy has attracted attention since 2008.


    In 2018, two breakthrough landmarks were achieved in the field of gene therapy.


    In fact, gene therapy started with rare diseases.


    But as a worker engaged in technical research, the state's payment for orphan drugs is actually an indirect exploration of science and technology.
    In fact, both nucleic acid drugs and gene editing technologies start with orphan drugs.
    Therefore, we still hope that the country can vigorously support the development of orphan drugs.

    04 RNA drugs "Return of the King"

    RNA drugs have been studied since the 1950s and 1960s, and have experienced several ups and downs in the development of half a century.
    Only in 2018 has it truly been "return of the king".
    According to statistics, there are currently more than 200 RNA drugs under research worldwide, mainly ASO and siRNA.
    The market size is expected to exceed US$10 billion in 2025.
    This is mainly affected by the maturity of drug delivery technology, and it is expected that RNA drugs will become the third largest drug in the next ten years.
    Due to the lack of technological precipitation, especially delivery technology, there are not many RNA drugs under research in China at present, and there are still opportunities to fill the gap.
    Whether it is investment or entrepreneurship, it is a good direction.

    However, the current indications of RNA drugs are still dominated by rare diseases and will soon enter the field of chronic diseases.
    Inclisiran is the world's first small interference nucleic acid drug for lowering low-density lipoprotein cholesterol (LDL-C).
    Two injections a year can reduce blood lipids by an average of 52.
    3%.
    The side effects are similar to placebo, and it is very likely to slowly expand.
    To the field of chronic diseases.
    If safety is verified in the real world, it can even be used to prevent the emergence of cardiovascular diseases.
    If it is really used for prevention, it will inevitably bring revolutionary changes to the cardiovascular field.
    This possibility is very high.
    In addition, RNA drugs have also achieved good results in the treatment of hepatitis B in clinical phase 2.

    The launch of Moderna's mRNA vaccine in 2020 is not only a milestone for RNA technology, but also a milestone for vaccines.
    Because it is difficult for you to imagine a new virus appearing before this, it only takes less than a year to develop a vaccine.
    For example, we have developed an HIV vaccine for 40 years, and it has not been successful yet.
    However, the new crown vaccine was completed in one year from the discovery of the virus, the acquisition of the viral gene sequence, and the launch of the vaccine.
    This was unimaginable before.
    This is entirely due to the accumulation of mRNA technology development.

    The development of RNA technology has also made "drug customization" possible, that is, promoting the arrival of the single patient era.
    Mila is the first patient to try drug development and customization.
    She suffers from the rare disease "Batten'disease", which is caused by the mutation of gene MFSD8 inserted into SVA.
    Without treatment, the girl will soon die of brain degeneration.
    Dr.
    Tiothy Yu of Boston Children's Hospital designed an antisense nucleic acid drug targeting SVA for her, named "Milasen", and commissioned CRO to complete the custom synthesis.
    Through repeated communication with the FDA, for the first time, the FDA approved a drug developed for a single patient into clinical research.
    After Mila injected "Milasen", her symptoms were controlled.
    However, Mila has now passed away.

    The concept of "single patient" was put forward by the two directors of the FDA, Dr.
    Woodcock and Dr.
    Marks, in comments in the same period after the New England Journal reported on Mila's case in October 2019.
    The main content of the comment at that time was "The development of new technologies such as RNA technology may promote the arrival of the era of drug customization.
    That is, the development of one drug for one patient.
    After entering this era, all pharmaceutical models have been subverted, and patients and doctors are partners.
    Relationship.
    " What is the safety of the drug? How is it effective? Who will pay for the cost? How to ensure the current resource model generated by the past pharmaceutical process? All need to be considered.
    If private customization becomes possible, we cannot predict what business model will be brought about in the future, but I think this day will come after all.

    In fact, apart from these advances in drug technology, the development of biomedicine is actually the development of biology.
    The basic research of biology has also made great progress in the past two years.
    For example, in the past, molecular biology studies are all biological macromolecules, whether it is DNA or protein, they have studied some of their chemical reactions and interactions, but few people pay attention to their physical state.
    Recently, biological phase has become one of the most popular areas of basic biological research.
    Almost every issue of "Science" and "Nature" has articles on biological phase change.
    Sooner or later, this field may win the Nobel Prize, and biological phase change Research will bring a huge opportunity for drug development.
    For example, Dewpoint Therapeutic (the company was established in 2019 and is the world's first biophase change company) recently discovered that the resistance mechanism of an old drug, Tamoxifen, has a lot to do with biophase change.

    Biological phase change has now become a very fast-growing field in the United States.
    In 2020, companies based on biological phase change have been established.
    In November, Nereid Therapeutics, which was incubated by the American venture capital firm Apple Tree Partners (ATP), was established and received US$50 million.
    In December, Faze Medicines was founded by "scientists" in the field of biomolecular condensate, and received $81 million in Series A financing.
    I predict that this field will also become a more important direction in two years.
    It is a pity that we are currently unclear on how this technology is used to make medicine, and what targets are still unclear.
    There are other technologies that can be paid attention to, which is the application of artificial intelligence in the field of biomedicine.

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