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    Home > Medical News > Medical World News > 2020 R & D pipeline report: which areas deserve attention?

    2020 R & D pipeline report: which areas deserve attention?

    • Last Update: 2020-01-02
    • Source: Internet
    • Author: User
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    2020 has come! At the beginning of the new decade, what are our expectations for the era of Biomedical Innovation? Recently, pharmaceutical executive released its 2020 Pipeline Report This is the 16th annual R & D pipeline report released by the company This report reviews the R & D pipeline in many R & D fields, including cell and gene therapy, CRISPR gene editing, Alzheimer's disease (AD), etc Today, the content team of Wuxi apptec will introduce you to the highlights of this report According to the data provided by signals analytics, a data analysis platform company, more than 3300 cells and gene therapy (CGT) are currently in the phase 2 to phase 4 clinical development stage, which shows that research and development activities in this field are extremely active CGT can be used to treat a variety of diseases including cancer, hemophilia, Huntington's chorea, sickle cell anemia, amyotrophic lateral sclerosis (ALS) Last year, zolgensma of Novartis for spinal muscular atrophy (SMA) was approved by the U.S FDA, and zynteglo, developed by Bluebird bio for β - thalassemia, was also approved by the European Union Moreover, the National Institute for health and care excellence (NICE) recommended gene therapy luxturna as a treatment for hereditary retinal dystrophy caused by RPE65 gene mutation This represents recognition of this innovative treatment model by regulators and payers Due to the rapid development of car-t therapy, the most popular target in the field of cell and gene therapy is CD19 There are more than 300 clinical trials to test cell and gene therapy targeting CD19 BCMA, a new hot target of car-t therapy, also ranks in the top ten In addition to the well-known car-t therapy and CRISPR Technology (described further below), research in this field also includes the development of innovative therapies based on induced pluripotent stem cells (IPSC) For example, at last year's ash annual meeting, fate therapeutics first published the results of clinical trials using IPSC to generate "ready to use" T cells and natural killer cells Last August, Bayer announced its acquisition of bluerock therapeutics The company's technical expertise is based on the invention of Professor Shinya Yamanaka of Kyoto University to build the IPSC technology platform ' Bayer's acquisition of bluerock is just one example of how big pharmaceutical companies expanded their expertise in gene therapy and cell therapy research and development by acquiring biotechnology companies last year Last year, Roche completed the acquisition of spark therapeutics, the star company of gene therapy, and at the end of the year, it paid up to $1.15 billion for Sarepta's gene therapy in development for DMD The report says the emergence of potential "curative" therapies shows research results in regenerative medicine CRISPR technology enables researchers to edit genome more easily and effectively, which brings revolutionary changes to genome editing research Although CRISPR technology is still in the early stage of development, there is growing interest in this technology in the industry CRISPR research has received $2 billion in research funding over the past five years, according to signals analytics More pharmaceutical companies began to publish research papers and patents related to CRISPR technology Last year, ctx001, jointly developed by CRISPR therapeutics and vertex, released the results of clinical trials for the first time Ctx001 is a hematopoietic stem cell therapy using crispr-cas9 system for gene editing in vitro One patient with transfusion dependent β - thalassemia (TDT) and one patient with severe sickle cell anaemia (SCD) received ctx001, and both achieved the effect of stopping transfusion dependence The first T-cell therapy edited by CRISPR technology in the United States also showed good safety, and the clinical trial results of this therapy were published at the ash annual meeting Editas medicine, founded by famous scholar Dr Zhang Feng, also announced in July last year that the world's first in vivo CRISPR gene editing clinical trial was started to treat 10 patients with Leber's congenital amaurosis These clinical trials are still in progress, and their results will help researchers further understand the safety and efficacy of gene editing therapy based on CRISPR technology In addition to being used to develop innovative gene editing therapies, CRISPR technology has also been used to develop sensitive and simple molecular diagnostic techniques Dr Zhang Feng founded Sherlock Biosciences in March last year Its core technology platform is Sherlock diagnostic technology platform based on CRISPR technology and insectr molecular diagnostic platform based on synthetic biology In 2018, another CRISPR technology pioneer, Dr Jennifer doudna, co founded the molecular diagnostics company mammoth Biosciences based on CRISPR technology In March last year, it obtained an exclusive R & D license from the University of California, Berkeley, for cas14, allowing the company's diagnostic technology to search for double stranded DNA, single stranded DNA and RNA In 2019, locus Biosciences, one of "field biotechnology's 2019 field 15", opened up a new application field for CRISPR technology The company expressed the gene encoding Cas3 nuclease in phages The difference between Cas3 and the normal cas9 nuclease is that cas9 only cut the DNA double strand at the finger location point, and Cas3 can completely cut the designated DNA sequence Locus aims to use this characteristic of Cas3 to produce phages with specific killing effect on drug-resistant bacteria The company and Janssen reached a $818 million R & D deal early last year Its leading in research therapy has entered the stage of clinical development, targeting Escherichia coli in patients with urinary tract infection Preliminary clinical progress in CRISPR indicates that this breakthrough technology is gaining more attention in the industry However, whether it can bring benefits to patients still needs to be verified in clinical trials According to the Centers for Disease Control and Prevention (CDC), cardiovascular disease (CVD) is still the leading cause of death in men and women Hypertension, high cholesterol and smoking are the key risk factors for CVD Although there are many approved therapies for high blood pressure, high cholesterol and the factors that lead to CVD disease, researchers are still opening up a "new battlefield" for CVD treatment According to signals analytics, hypertension, congestive heart failure, and atherosclerosis are the most frequently studied indications Among the CVD targets, PCSK9 and slc5a2 (also known as SGLT2) are the fastest developing targets Several PCSK9 inhibitors have been approved for listing, and Novartis Inc.'s RNAi therapy inclisiran, which targets PCSK9, is expected to apply for listing this year SGLT2 inhibitors, including dapagliflozin, canagliflozin and empagliflozin, have been approved to reduce the risk of different types of cardiovascular events in patients with type 2 diabetes In addition, AstraZeneca's dagliejing has obtained the FDA's fast track qualification, which is expected to reduce the risk of heart failure in patients with non-2 diabetes It reduced the risk of increased heart failure in patients with non-2 diabetes by 27% in a phase 3 clinical trial called dapa-hf In addition to SGLT2 inhibitors, the combination of Novo Nordisk's GLP-1 receptor agonist semaglutide with standard therapy can reduce the risk of major cardiovascular adverse events by 21% (oral dosage form) or 26% (injection type) Novo Nordisk has submitted regulatory applications to the FDA and is expected to be approved for extended indications early this year High levels of vasopressin in the blood are associated with the progression of congestive heart failure Bay1753011 developed by Bayer company is an antagonist of vasopressin receptor 1 and 2 (VR1 and VR2), which aims to alleviate the progress of heart failure by blocking the vasopressin signal pathway Currently, it is tested in phase 2 clinical trials In August last year, idorsia released the results of its phase 2 clinical trial of aprocitentan, an oral dual endothelin receptor antagonist, in the treatment of hypertensive patients Aprocitentan significantly reduced blood pressure compared with placebo At present, idorsia company has launched three phase clinical trials to treat patients with uncontrolled blood pressure The first study on chimeric antigen receptors (cars) was published in scientific journals nearly 30 years ago However, it is not until recent years that researchers have been able to translate this scientific breakthrough into innovative therapies that benefit patients According to the statistics of signals analytics, the number of scientific papers, patents and clinical trials related to car-t has been growing rapidly in the past few years, which is faster than the whole ecosystem After being approved by FDA for the first time in 2017, three new approved therapies are expected to come into the car-t field this year, namely, CD19 targeted, LISO cel targeted at Bristol Myers Squibb (BMS), kte-x19 targeted at Gilead science, and IDE cel targeted at BCMA jointly developed by BMS and Bluebird bio Generally, car-t therapy is designed to allow T cells to target tumor cells With the development of car-t technology, emerging biotechnology companies began to design a new generation of car-t therapy to treat autoimmune diseases For example, caballetta based on the University of Pennsylvania study Bio's technology platform, called CAAR, enables car-t therapy to specifically target B cells expressing autoantigen receptors by expressing autoantigens that cause autoimmune reactions in the extracellular domain of car, so as to achieve complete and lasting elimination of B cells leading to autoimmune diseases, while protecting other B cell populations At present, the company has a number of car-t treatments for autoimmune diseases in the preclinical stage, and is expected to start its first clinical trial this year The strategy of quell therapeutics for autoimmune diseases is to install car on regulatory T cells (Tregs) with immunosuppressive function Let them play the role of immunosuppression in a specific environment Founded in March last year and financed by $40 million, the company is one of the "biotech giants" of 2019 Car Treg therapy developed by the company can be used to treat organ transplant rejection, autoimmune diseases and inflammatory diseases Sangamo also has a car Treg therapy which is about to enter the clinical development stage This innovative therapy named TX200 will be used to treat the immune rejection caused by solid organ transplantation The report points out that in the field of car-t therapy, the traditional car-t design technology has gradually matured, and the focus of research work has turned to improve the effectiveness of this therapy and reduce the side effects, so as to expand the scope of car-t therapy for cancer, and beyond cancer, to treat other diseases Alzheimer's disease is the most common type of dementia Although the development of new drugs for the treatment of this disease has been very active in the past decade, the clinical results with promising input and output are very few According to the analysis of signals analytics, Alzheimer's disease is the most advanced disease in preclinical research and development compared with other central nervous system diseases
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