How biotechnology is gearing up to eradicate disease

12-01-2023 | News

Over the next decade, research has the potential to create effective new diagnostics, drugs and vaccines. But advances in genetic engineering technologies open discussions.

by Andrea De Tommasi

Aging as the last great enemy to defeat. It is the most ambitious terrain, perhaps even the most reckless, on which a number of biotech companies will compete in the coming years. 2022 has been an important year for longevity research in biotechnology.

Billionaires like Jeff Bezos is Vitalik Buterin are investing in anti-aging startups, but also countries like the Japan they aim for the big target: drugs and gene therapies that reverse aging in humans. The "medical rejuvenation" is also the goal of Richard Klausner by Altos Labs, a research company financed by large Silicon Valley investors, which a few months ago showed the data of the experiments conducted in its laboratory, in which sick mice returned to health thanks to "cellular reprogramming" technologies. The experiments proved promising and spurred other groups of scientists to pursue a similar approach in different animal models. Yet the biology of rejuvenation applied to human cells remains enigmatic, at best.

In recent days, however, signs have emerged that mRNA technology (the one used for anti-Covid vaccines) would make it possible to create personalized cancer vaccinesModerna has released the results of the second phase of experimentation of the mRNA-4157/V940 vaccine, used in combination with Keytruda immunotherapy. The CEO of Moderna Stephan Bancel, explained: “mRNA has marked a breakthrough for Covid-19 and now, for the first time ever, we have demonstrated the potential of the same technology with the results of the new clinical study on melanoma”. The result is that with the vaccine the risk of recurrence or death, compared to immunological treatment alone, was reduced by 44%. “We will initiate further studies in melanoma and other forms of cancer with the aim of offering patients truly personalized cancer treatments,” Bancel announced.

mRNA vaccines, according to proponents, have the potential to alter current therapies for a wide range of diseases, and the field is expanding. Last January Modern started a human trial of a HIV vaccine. Although the vaccine reduced the risk of infection in monkeys by about 80%, it is not an easy task: HIV is a difficult target due to its complexity and the main challenge is to know the antigen, the substance that induces body's immune response to fight the virus. mRNA vaccination provides renewed hope for treatment as well autoimmune or neurodegenerative diseasesBiontech, the German startup that helped develop the Pfizer vaccine, is using a technique to fight multiple sclerosis: the immune system progressively removes the insulation from the nerve fibers in multiple sclerosis producing slow and irreparable damage. The strategy being worked on could prove to be effective and adaptable to each patient.

This convergence of biotechnologies with information technology and artificial intelligence could also favor another process: thegetting medicines to market fasterAlex Peterson, McKinsey biotech expert, in a deepening published by the consultancy, illustrates the concept: “We may be able to have drugs in a tenth of the time, from the discovery of the disease to the ability to treat patients. Today, many diseases simply have no treatment. Ultimately, we will have life-changing drugs, on a scale and at a pace we've never seen before, getting to the right patient at the right time."

IS there promise of personalized medicine: treating that patient, with that disease, at that specific stage. Another McKinsey expert, Christopher Sandler, tells what conditions will facilitate this process: “In the not so distant future, we could collect health data through different inputs: from wearable devices, from our electronic health records or from clinical research. And we will have the opportunity, on a voluntary basis, to upload this data to a central, secure and reliable data storage system”.

L'genome editing is of great interest in prevention and treatment of human diseases. Think of the gene-editing variants of Crispr, which are significantly more efficient and safer than their predecessors. CrisproOFF, for example, uses the mechanisms of epigenetics to turn genes on and off reversibly without cutting or altering the gene itself; the prime edits, so versatile that it has been updated to accurately edit up to 10,000 letters of DNA in a variety of cells; the twin prime editing, with the potential to rewrite entire genes. These advanced types of Crispr now allow researchers to address previously untreatable genetic abnormalities.

They arise ethical concerns when genome editing, using technologies such as Crispr-Cas9, is used to alter human genomes. Changes made to genes in eggs or sperm or to genes in an embryo could be passed on to future generations. The modification of the genome of the cells and the embryo raises a number of challenges, such as whether it is permissible to use this technology to enhance normal human traits (such as height or intelligence). Based on ethics and safety concerns, germ cell and embryo genome editing is currently illegal in many countries. Other lines of research have instead completely abandoned Crispr relying instead on a sophisticated bacterial mechanism to modify millions of DNA sequences without breaking a single strand.

Meanwhile, artificial intelligence is helping in the discovery of new proteins, known as anti-Crispr, which could prevent harmful side effects. DeepMind and theUniversity of Washington they developed an AI capable of developing a protein based solely on its genetic code. AI-powered brain implants have revealed themselves able to fight depression, treating chronic pain and converting the electrical impulses of the brain. also onAlzheimers therapies are being studied to address and contain the disease.

Whether or not technology can address some of our toughest ailments remains to be seen, but the industry is booming. An experiment of University College London took it a step further by changing a faulty gene in the liver that damages the heart and nerves. Unlike previous attempts, the Crispr apparatus was delivered to the bloodstream in a single infusion to deactivate the gene by significantly reducing production of the mutant protein. Another experiment targeted a faulty gene that causes blindness: Volunteers were able to improve their perception of light by directly injecting the therapy into the retina. alphabet, the parent company of Google, founded its own Isomorphic laboratories to embark on the discovery of new drugs.

If we look at the current landscape of biotechnology in the world, we come to a conclusion: in Europethe Biotech companies are growing at a rapid pace. The key factors making the European biotechnology market more attractive to investors are the abundance of top talent, powerful research centers, industry expertise to support basic science and innovations. Most of Europe's biotech companies are based in FranceGermany is UK. Interestingly, most biotech companies in the US have focused on advanced therapy-based medicines. On the contrary, the European market has focused as a key product on the production of vaccines. Another great challenge of biotechnology: how to prevent and deal with the next pandemic.

Published December 20 on Futura Network.

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