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    Home > Medical News > Medical World News > Introduction of R & D pipeline and related products in gene therapy

    Introduction of R & D pipeline and related products in gene therapy

    • Last Update: 2020-02-18
    • Source: Internet
    • Author: User
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    This paper introduces the R & D pipeline and related products of gene editing technology company We have combed and analyzed the income of gene editing companies in the world in 2018 For the listed companies, the income is ranked from high to low in 2018; for the non listed companies, the income is ranked from high to low in 2018 The ranking of top 5 companies is shown in the table below Data source: collated by Yaodu consulting team Next, we will introduce the R & D pipeline of the above companies in turn Sangamo therapeutics was founded in 1995, formerly known as Sangamo Biosciences, which was changed to the current name in January 2017 Headquartered in Richmond, California, USA, Sangamo therapeutics is a gene therapy company focusing on Zen editing technology Its core technology platform involves gene therapy, cell therapy, in vivo genome editing and in vivo genome regulation The R & D pipeline is shown in the figure below Figure 1 latest development pipeline of Sangamo therapeutics Data source: Official Website of Sangamo therapeutics, sorted out by the pharmaceutical consulting team As shown in the figure, Sangamo therapeutics is currently developing 16 products, of which 6 products have entered the clinical stage In addition to rare diseases in the blood system, the research and development pipeline also involves genetic and metabolic diseases, such as MPS II and Fabry Disease (Fabry's disease) and phenylketonuria (PKU), central nervous system diseases, such as multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), frontotemporal degeneration (FTLD), Huntington's chorea, etc., also include anti-tumor products and other gene therapy products During 2017-2018, Sangamo therapeutics Co., Ltd and Pfizer and kit Co., Ltd worked on the development of hemophilia A and anti-tumor car-t cell therapy products, which had a positive impact on their revenue growth [1] In 2017, the company's annual revenue was $36.567 million, with a growth rate of 133% in 2018 In addition, Sangamo therapeutics has worked with Sanofi and Takeda to develop gene therapy products Founded in 2007, horizon discovery is a biotechnology company headquartered in Cambridge, UK It was listed on the London Stock Exchange in 2014 The company has many technical patents, such as rAAV, CRISPR, zinc finger, etc., which can efficiently, quickly and accurately realize the fixed-point editing of genes or chromosomes At present, the company's main business income comes from gene editing related cro technical services In 2017, horizon acquired dharmacon Inc As a leader in custom RNA synthesis, dharmacon is an early player in RNA interference (RNAi), contributing several key scientific discoveries and the first commercial reagents The acquisition will significantly expand horizon's business scope and enable it to provide customers with solutions in various aspects of RNAi interference, including siRNA, lentivirus shRNA, microRNA research tools, genome-scale library for RNAi function screening, microRNAs and long-chain non coding RNA, etc Founded in November 2013, editas medicine is co founded by Zhang Feng and Dr doudna Although the two are now separated, editas is affiliated with MIT haver's broad research institute It was listed on NASDAQ in February 2016 and is the first CRISPR / cas9 technology company to be listed In January 2020, its market value is US $1.5 billion The latest R & D pipeline is shown in the figure below Figure 2 latest development pipeline of editas medicine Data source: Official Website of the company It can be seen from the above that the key development indications of editas medicine in vivo gene therapy are rare eye diseases developed in cooperation with Allergan, including Burt's congenital amaurosis-10 (edit-101 entering clinical stage), Usher's syndrome and retinitis pigmentosa Bergheit's congenital amaurosis is a rare blindness or severe visual impairment at birth or shortly after birth The birth prevalence rate is 2 to 3 per 100000 newborns, which is caused by mutations of several different genes, among which 10 types are caused by mutations of CEP290 gene, accounting for about 15% [2] Usher 's syndrome, also known as hereditary deafness retinitis pigmentosa syndrome, incidence rate is about 2.2/10 million -4.4/10 [3] Retinitis Pigmentosa is a hereditary chronic degenerative disease of primary retinal dystrophy Its eyes are mostly poor and its incidence rate is about 1/20000-5/1000[4] In addition, there are cell therapy products for β - thalassemia, sickle cell disease and anti-tumor in the company's R & D pipeline Intellia therapeutics was founded in 2014 with Jennifer doudna as one of the founders The company was listed on NASDAQ in May 2016 and has a market value of $700 million in January 2020 The latest R & D pipeline is as follows Figure 3 latest development pipeline of intellia therapeutics Data source: Official Website of the company As shown in the figure, in the R & D pipeline of intellia therapeutics, in vivo gene therapy focuses on the development of indications for thyroxine delivery protein amyloidosis (attr) and hereditary angioedema (HAE) In the development pipeline of intellia therapeutics, in vitro gene therapy products mainly focus on sickle cell disease, acute myeloid leukemia and solid tumor In vitro treatment products are all cell treatment products based on CRISPR / cas9 technology, including hematopoietic stem cell treatment products, TCR treatment products, car-t treatment products and other products Precision Biosciences was founded in January 2006 and headquartered in Durham, North Carolina It is committed to developing a new generation of genome editing technology platform arcus in the field of cancer immunotherapy, genetic diseases and food The R & D pipeline is as follows Figure 4 latest R & D pipeline of precision Biosciences Data source: Official Website of the company As shown in the figure, in the company's R & D pipeline, car-t products for in vitro gene therapy focus on the indications for myeloma and hematological system tumors, such as non Hodgkin's lymphoma, leukemia, etc In vivo gene therapy products focus on chronic hepatitis B and genetic diseases, such as familial hypercholesterolemia, lipoprotein lipase deficiency and primary hyperoxaluria In 2018, precision Biosciences and Gilead reached a strategic cooperation agreement, which will use precision's unique arcus gene editing technology platform to jointly develop innovative gene therapy to eliminate hepatitis B virus (HBV) in vivo Founded in 2016, casebia therapeutics is a joint venture between Bayer, a German pharmaceutical giant, and CRISPR therapeutics Casebia therapeutics has obtained gene editing technology from CRISPR therapeutics The products involve specific disease fields such as blood and Ophthalmology, and obtained protein engineering technology through Bayer, so as to significantly improve the ability of CRISPR / cas9 gene editing platform According to the latest news, in October 2019, Bayer transferred all operation rights of casebia biotechnology to CRISPR therapeutics At present, because the official website of casebia has been merged with CRISPR therapeutics, it is unable to obtain the R & D pipeline exclusively belonging to casebia At the 2019 International Society for thrombosis and hemostasis conference, Dr Alan Brooks, chief scientist of Casebia, reported A study on gene editing method for hemophilia A in this study, the CRISPR/Cas9 technology was used to genetically modify mice to stably express the drivable coagulation factor VIII in order to realize the disease control of hemophilia A Poseida therapeutics was founded in 2015 with investors including airling capital, longitude capital, Malin Corporation, Novartis and perceptive advisors The company is headquartered in San Diego, USA The latest R & D pipeline of the company is shown in the figure below Figure 5 latest development pipeline of Poseida therapeutics Data source: Official Website of the company As shown in the figure above, the company's R & D pipeline is divided into three parts, including anti-tumor car-t cell candidates, in vivo gene therapy candidates for ornithine carbamyltransferase deficiency (OTC) and methylmalonic acidemia (MMA), and in vitro hematopoietic stem cell (HSC) transplantation related products Founded in 2017, beam therapeutics, located in Delaware, USA, is the first biotechnology company in the world to develop precise gene drugs by using single base editing technology It is co founded by Professor Zhang Feng, Professor David Liu and Professor J Keith joung, three "great gods" in CRISPR technology field The company's investors include arch venture partners, eight roads ventures, f-prime capital partners, GV and redmile group The company focuses on the development of four fields, namely, gene correction, gene modification, gene activation, gene silencing and multi gene coding The latest R & D pipeline is shown in the figure below Figure 6 latest development pipeline of beam therapeutics Data source: Official Website of the company It can be seen from the above that the company initially established the R & D pipeline of auto and allogeneic car-t candidates, focusing on the treatment of blood malignant tumors After that, the company expanded its R & D pipeline and began to develop new gene therapy products, involving in the fields of rare blood diseases, liver metabolic diseases and central nervous system diseases At present, all products under research of beam company are in pre clinical research stage Syntego was founded in 2012, with its headquarters in Silicon Valley, California, USA It is a company engaged in gene editing technology services Its main business is to provide gene engineering cells and use crisp revolution kit technology to synthesize RNA series products, such as gene knockout kit v2 These products can be used in CRISPR gene editing and research design experiments to improve the efficiency of gene editing experiments and reduce the cost of experiments In October 2018, synthego company completed C rounds of financing of up to US $110 million, with investors including founders fund, 8vc and Menlo ventures, etc The funds will be used for further research and development of new projects in the company's existing businesses, and to bring new service projects to the market But in the short term, the funds will be mainly used to expand the company's two main business areas: one is to create CRISPR kits, which can create different gene families according to the requirements of researchers and scientists, and the other is to create "clinical grade" products, which means that they can be used for clinical trials of animal (and potential human) subjects Founded in 2015, instripta is a gene editing technology service company, headquartered in Colorado, USA It is committed to creating revolutionary gene editing technology and tools, bringing changes to human food, fuel and medical care In 2018, the company completed the c-round financing of 85.5 million US dollars and the C + round financing of 20 million US dollars [5], with the total financing amount of 105.5 million US dollars The investors include: venlock, foresite capital, m é Rieux D é velopement, Paladin capital group, MLS capital and nanodimension The new generation of CRISPR endonuclease of instripta is called madzymes Madzymes can recognize different PAM sequences and cut DNA efficiently At present, instripta has published the DNA sequence of mad7 on its nuclease discovery and engineering platform Mad7 is also the first enzyme found in the madzymes family of instripta In July 2018, the first nuclease mad7 gene editing system of instripta was patented by the U.S patent and Trademark Office and proved to be effective in both microbial and mammalian systems In December 2018, instripta announced that it would widely use nuclease mad7 in its R & D products and services This nuclease is a proprietary CRISPR enzyme provided by the company for business partners and academic researchers
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