“Life science” is the science that studies the life phenomena and laws of life activities of organisms, and is the foundation of biomedicine and artificial intelligence. Research and development in life sciences has been a major research priority worldwide for many years.
In 2022, the new crown epidemic will seriously affect the operation and development of the global economy, and it will once again verify the strategic importance of life science research. According to incomplete statistics, in 2022, the global investment in research in the field of life sciences will reach 170 billion US dollars.
At present, the field of biomedicine is developing rapidly, and new technologies emerge in an endless stream. Zhang Xu, an academician of the Chinese Academy of Sciences, predicts that in 2023, life science research and development will remain a key area of scientific and technological development, and related research and development achievements in the fields of biomedicine, biomedical engineering, synthetic biology, and intelligent technology will also continue to emerge and develop. Transformed to the ground.2023and alsoIt will be an important year for the development of China’s biomedicine and biomedical engineering enterprises.
Gene technology leads life science innovation
In 2022, the immune escape mechanism of the new coronavirus mutant strain, the human early embryo translation genome map and the zygotic genome activator research, the high-precision life panorama spatiotemporal gene expression mapping, the subversive gene decoding technology, the molecule of autophagy initiation in multicellular organisms Mechanism and other research were selected into China’s top ten advances in life sciences of the year, allowing the world to see the fruitful results of China’s life science research.
Recently, MIT Technology Review announced that among the “Ten Global Breakthrough Technologies” for 2023, the field of life sciences has three exclusive seats, namely gene editing technology for high cholesterol, on-demand organ production and ancient DNA analysis.
CRISPR gene editing technologyExcitement awaits in 2022turning pointthe therapeutic range of this technology is expected to expand from rare genetic diseases to other common diseases such as hypertension.On the basis of CRISPR, single base editing can complete the precise editing of genes without cutting the double strand of DNA; the lead editor can not only convert any base into other bases, but also accurately insert DNA fragments into the genome, providing a basis for replacement Disease genes offer the possibility. After evaluating enough clinical data, the above innovative gene editing technologies will greatly expand the scope of application of gene editing to treat diseases.
In 2022, the world’s first patient who received a pig heart transplant died 2 months later. A cell therapy company conducts clinical trials to grow a mini-liver in the human body that can perform the same function…Scientists are drawing lessons from such operations and experiments to improve the success rate of xenotransplantation and use human cells to build complex tissues and organs.In the future, organoids that can mimic the functions of specific human organs will largely solve the problem of organ transplant shortage.
Today, ancient DNA analysis technology has matured.With the development of more innovative and affordable sequencing technologies, deciphering damaged DNA is no longer a difficult task.In 2022, the Nobel Prize in Physiology or Medicine will go to the field of paleogenetics. Dr. Svant Pabo extracted DNA from bone samples of extinct humans, and the evolutionary tree shows the relationship between Homo sapiens and these extinct hominids. The enlightenment brought by ancient DNA has rewritten our understanding of history. Through this technology to mine the genetic information of ancient humans and combine it with modern molecular biology techniques, we can more clearly understand the impact of human evolution history on our human health today.
In addition, using AI to predict protein structures to aid drug development will also achieve substantial results in 2023.In July last year, the artificial intelligence company DeepMind announced that it has deciphered almost all known protein structures. The database built by its AlphaFold algorithm now contains more than 200 million known protein structures, which has become a powerful tool for the development of new drugs. At present, hundreds of start-up companies are exploring ways to use artificial intelligence to accelerate drug discovery. DeepMind will launch some blockbuster biotechnology in 2023. The initial results of clinical trials of AI pharmaceuticals are also expected to be shown to the world in 2023. Perhaps in a few years, we are expected to see AI-assisted drug development on the market in batches.
Four major clinical therapies are worth looking forward to
While revolutionary discoveries cannot be predicted, it is possible to predict which ongoing projects will reach new heights in the coming months. In biomedical research, a number of clinical trials due to be completed by 2023 promise advances in treatments for cancer, Alzheimer’s, multiple sclerosis and obesity.
In 2013, a new generation of cancer immunotherapy drugs emerged and became the top ten scientific breakthroughs of the year.Unlike chemotherapy and radiation, which attack cancer cells directly, immunotherapy aims to boost the body’s normal immune system’s ability to fight tumors.In recent years, the field of immunotherapy was bornPD-1/PD-L1,CAR-Tnonspecific immune stimulation, oncolytic virusWait for the explosion. A decade after the advent of immunotherapy drugs, the field of immunotherapy for cancer is in full bloom.Results of clinical trials expected in 2023 will increase the number of patients benefiting from immunotherapy. Advances this year are expected to extend beyond immunotherapy drugs to include vaccines and cell therapies.
On January 6, the FDA approved Lecanemab, a monoclonal antibody to amyloid-beta, jointly developed by Eisai and Biogen as a drug for the treatment of Alzheimer’s disease (AD). At the end of last year, Eli Lilly announced the positive data of a phase III clinical trial of donanemab, a monoclonal antibody targeting β-amyloid protein: after 6 months of treatment, compared with the baseline, donanemab reduced early Alzheimer’s disease. Amyloid plaque levels were reduced by 65.2 percent in the brains of the patients with Alzheimer’s disease, compared with 17.0 percent in the active control group.This suggests that amyloid-beta mAbs can rapidly and effectively alter the biology of Alzheimer’s disease in the early stages of treatment.In 2023, more data on the number of clinical trials of AD drugs will clarify the expected effect of β-amyloid inhibitors.
Multiple sclerosis (MS) is a severe, lifelong, progressive, and disabling neuroimmune disease. In 2022, the total number of MS patients worldwide will be 2.9887 million.In the second half of 2023, the results of the first two phase III clinical trials of the new drug BTK enzyme inhibitor for the treatment of multiple sclerosis will be announced.Meanwhile, an international panel of experts is revising the diagnostic criteria for multiple sclerosis. They will make recommendations in 2023 that consider objectifiable biological indicators, rather than making a diagnosis based solely on patient-reported symptoms. In 2024, the world’s first BTK inhibitor for multiple sclerosis is expected to be approved for marketing.
The treatment of obesity is about to enter a new era with the advent of two drugs that can achieve bariatric surgery-like results.Semaglutide is a glucagon-like peptide (GLP-1) receptor agonist. In 2022, the drug has been approved in Europe and the United States for the treatment of obesity. Although it has not been officially approved in China, it has become a popular drug. Domestic Internet celebrity diet pills. In the second quarter of 2023, Tirzepatide, a weight-loss drug developed by Lilly that combines GLP-1 and GIP two hormone synthetic analogs, will complete phase III clinical trials. Preliminary results show that the drug has good efficacy and few side effects. Approved for the treatment of obesity.
Research on the six popular targets continues to deepen
According to statistics, from 2017 to 2022, the top five popular targets in China are: PD-L1, EGFR, PD-1, VEGFR and HER2.Only in 2022, the top 6 popular targets in China are PD-L1, HER2, PD-1, EGFR, CD3, TIGIT, and the popularity of targets such as VEGFR, FGFR and Claudin18.2 has decreased.
PD-1, an immune checkpoint receptor expressed by activated T cells, is an important immunosuppressive molecule. PD-L1 is the ligand of PD-1. When PD-L1 is combined with PD-1, the activity of T cells will be inhibited and the original killing effect will be lost, resulting in the inability of T cells to recognize cancer cells. immune escape”.Thus, therapies that block the PD-1/PD-L1 pathway have become the focus of cancer immunotherapy.As of November 2022, China has approved 4 PD-L1 inhibitors, with a total of 10 approved indications. According to Frost & Sullivan data, by 2023, the global PD-(L)1 inhibitor market will reach 63.9 billion US dollars, and the domestic market will reach 66.4 billion yuan in the same period.
Antibody-drug conjugates are a hot track in the field of innovative drugs, and HER2 is the number one target.HER2, human epidermal growth factor receptor 2, is a member of the epidermal growth factor receptor family with tyrosine kinase activity. Polymerization of receptors leads to phosphorylation of receptor tyrosine residues and initiates multiple signaling pathways leading to cell proliferation and tumorigenesis. As a prognostic and predictive biomarker, gene amplification or overexpression of HER2 contributes to breast cancer and gastric/esophageal cancer. According to statistics, there are currently 73 antibody-conjugated drugs targeting HER2 in the world, of which 4 have been approved, 1 has been applied for marketing, and 30 are in the clinical research stage.
EGFR epidermal growth factor receptor is a transmembrane glycoprotein, and EGFR family members participate in the occurrence and development of various cancers such as non-small cell lung cancer (NSCLC) through specific driver mutations or gene amplification.As a broad-spectrum anti-tumor target protein, EGFR is the earliest and most studied target. In recent years, with the rise of ADCs and double antibodies, this target has shown timeless characteristics.At present, there have been three generations of EGFR-TKI targeted drugs for non-small cell lung cancer, and the fourth generation of products will also be launched on the market. Continuous efforts have been made to improve survival, overcome drug resistance, and reduce side effects.
CD3 is a transmembrane protein on the surface of T cells, which can combine with TCR on T cells to form a receptor complex to activate T cells, and is the number one target for the development of double antibody drugs.CD3 bispecific antibodies can redirect CD3+ T cells to tumor sites, which is a potential immunotherapeutic strategy for the treatment of hematological malignancies and solid tumors.As of December last year, a total of 333 CD3-related projects are in progress around the world. Experimental data show that the global CD3 target track is progressing rapidly, and there have been obvious breakthroughs in the treatment of leukemia, diabetes, and tumors.
The full name of TIGIT is T cell immunoglobulin and ITIM domain protein. It is a co-inhibitory receptor and is regarded as one of the most potential immune checkpoints after PD-(L)1. Studies have found that TIGIT is highly expressed in a variety of malignant tumors and is closely related to the prognosis of patients. Targeting TIGIT monoclonal antibody can effectively restore T cell function, and then play an anti-tumor effect.In 2023, leading pharmaceutical companies including Roche will continue to release the latest research data on TIGIT therapy.
Dozens of blockbuster new drugs are about to be approved
In 2022, the FDA’s Center for Drug Evaluation and Research (CDER) has approved 37 innovative drugs. Among them, 21 are first-in-class innovative drugs, accounting for 57%, reaching the highest level in previous years. Tumor is still a hot area for new drug development, followed by nervous system and skin drugs, and many rare diseases have also ushered in new treatment options.
In 2022, the National Medical Products Administration (NMPA) has approved a total of 49 new drugs, including 30 imported new drugs and 19 domestically produced new drugs. Anti-tumor drugs took the lead, accounting for 49%; drugs for the treatment of new crowns ranked second, accounting for 10.2%; followed by new drugs for blood diseases, new drugs for non-new coronavirus infections, and new drugs for immune system diseases.
In the new year, exa-cel, the gene-editing therapy for sickle cell disease (SCD),The first drug mavorixafor for patients with rare disease WHIM syndromeinnovative oral antibiotic gepotidacin for acute cystitis, long-acting insulin LY3209590 for type 2 diabetes, anti-APRIL monoclonal antibody for immunoglobulin nephropathy, KarXT, the first new drug for schizophrenia in 50 years, Tirzepatide for obstructive sleep apnea, Duchenne muscular nutrition for treatment More than 10 innovative drugs, including gene delivery therapy for adverse diseases, are expected to be approved by the FDA for marketing.
At the same time, Jaktinib, a new JAK inhibitor drug for the treatment of medium- and high-risk myelofibrosis, Hekirenza, a cell therapy product for the treatment of B-cell acute lymphoblastic leukemia, detrastuzumab for injection for the treatment of breast cancer, and Alopecia areata JAK3 selective inhibitor ritlecitinib, small molecule CGRP receptor antagonist Nurtec for adult migraine, GIP and GLP-1 dual-target receptor agonist Mounjaro for type 2 diabetes, Vyvgart for myasthenia gravis, etc. 20 A number of new drugs are also expected to be approved by CDE, NMPA and other relevant agencies for domestic marketing.
The above-mentioned new drugs mainly involve the fields of tumors, autoimmune diseases, anesthesia and analgesia, cardiovascular and cerebrovascular and metabolic diseases, ophthalmology and rare diseases, including small molecule targeted drugs, bispecific antibodies, antibody-drug conjugates (ADCs), and siRNA therapy , CAR-T products and other types.
Under the influence of macro factors such as the impact of the epidemic and the downturn of the capital market, the medical investment and financing market will show a relatively cold trend in 2022. 2023 has come, and the WHO will have a new definition on whether the new crown pandemic will constitute a global health emergency, and life science investment is expected to enter a new historical cycle. At the same time, the continuous advancement and breakthrough of innovative technologies, therapies, targets and even drugs have also injected confidence in the investment and financing of the life science field this year. The investment of top funds in biology will continue to increase, and the investment in biomedicine in the future will also follow the new principles. law.
