Who will be the next "star" in the wave of gene therapy?

This month, Astellas Pharma bought Audentes Theapeutics for $3bn, and so far Astellas has invested more than $17bn in the development of "once and for all" gene therapyRoche finally completed its $4.8bn acquisition of Spark TherapeuticsThese two newses not only show us the great interest of well-known pharmaceutical companies in gene therapy, but may also tempt other potential investors in gene therapy to re-enter the marketIt's worth noting that shares in several other gene therapies, including Rocket Therapeutics and Regenebio, also showed gains after news of The Acquisition of AudentesIn the last two years, regulators have also seemed to be showing a strong willingness to develop in the field of gene therapyThis leads us to speculate: Who will be the next gene therapy "star"?in today's article, we'll share with readers several "star" gene therapy companies that will stand out in 2020, in conjunction with a story published in VantageWe divide these companies into two categories, which are described separately belowSpace is limited, if you don't see what you're interested in in the article, you'd also be welcome to click on "Read the original text/Read More" at the end of the article and visit the original report pagecompanies with a complete technology platform?when it comes to potential gene therapy companies, many of them have names that flow into the brain, including Uniqure, Amicus Therapeutics and bluebird bioAmong them, Uniqure developed THE ATM-061, using the AAV5 virus vector carrying the code FIX Padua variant (FIX-Padua) genetically modified, with good safety and tolerance, is currently one of the very promising type B hemophilia gene therapyAlthough the company has been tested in only a handful of patients, it has a market capitalisation of $3 billionMany investors who want to sell want to take action before they announce the results of the key phase 3 trial HOPE-BHowever, the company's treatmentist prices are relatively high at the moment, and from this perspective we may be able to turn to companies that are more "people-friendly"mentions the company's current market value, and the number and stage of clinical development projects (Photo: Supplied)from the Astellas deal, companies with genetic therapy expertise and manufacturing capabilities may have some advantages So we think of companies like Abeona Therapeutics, MeiraGTx, Homology Medicines and Oxford Biomedica, whose common characteristic is their own "skills" Of these powerful companies, Oxford Biomedica is one of the most established At present, it not only has six clinical development stage candidates, but also with Sanofi, Novartis and other well-known pharmaceutical companies on the supply of slow virus vectors reached a cooperation Oxford's current market capitalisation is $553 million, making it more attractive than Uniqure companies working in the field of popular therapy? ophthalmology, neurology and rare diseases are the three major areas of popular treatment, investors will presumably from this point of view to seize the opportunity New York-based MeiraGTx is a leader in ophthalmology MeiraGTx has core capabilities in the design, optimization and production of viral vectors, as well as potential transformational gene regulation techniques MeiraGTx uses AAV as a carrier for transmitting genes, and by customizing vectors, gene therapy can be optimized for different diseases Small differences in carrier shell proteins regulate the efficiency of delivering genes to different types of cells, allowing the selection of different AAV shells to most effectively target specific cell types At present, it has six candidate therapies into clinical development Its development capabilities have gained the attention of major pharmaceutical companies after it signed a global partnership with Janssen, a subsidiary of Johnson and Johnson, to jointly develop treatments for hereditary retinal diseases Abeona is another high-profile gene therapy company that developed the self-contained cell product EB-101, which was awarded by the FDA for advanced regenerative medicine (RMAT) to treat people with large herpes epidermis But in September, the FDA suspended Phase 3 clinical trials of the treatment, skilling down the company's shareprice But the company also has two projects in phase 2 clinical development, which may be a concern for investors Voyager Therapeutics may be a good choice for "risky" investors Voyager's carrier expression antibody platform seeks to express therapeuticantiproteins using its original adeno-related virus (AAV) vector that can cross the blood-brain barrier This method may require only one intravenous fluid to continuously express high levels of therapeutic antibodies in the brain Currently, the company has partnered with a number of pharmaceutical companies, including AbbVie, and has two candidate treatments for Parkinson's disease in clinical development The biological properties of neurological diseases are complex and may involve multiple genes Therefore, the use of gene therapy to treat neurological diseases is a major challenge it is true that gene therapy has broad therapeutic prospects, but there are also some risks The duration of gene therapy treatment is still unknown, and the cost of medical care is a heavy burden for patients We hope that by 2020 more "new stars" will emerge and drive new developments in this therapy References: smh.com.au smh.com.au smh.com.au's despite salys is there sly s a fairly colloidd edgtos out, Retrieved December 23, 2019, from https://