Acquisition of shire mRNA drug development platform by Rana therapeutics

Source: U.S - China drug source January 5, 2017 [news event]: today, Rana therapeutics in Cambridge, Massachusetts announced the acquisition of the mRNA drug development platform of shire, a rare disease drug development giant Through this technology platform, Rana plans to introduce two mRNA candidates to clinical practice in the first half of 2018, respectively, for the treatment of cystic fibrosis (CF) and urea circulatory disorder (UCD) 12 employees of shire engaged in mRNA drug development will join Rana therapeutics along with the acquisition Although Rana did not disclose the detailed cost of the acquisition this time, it is estimated that the first payment is very low Shire mainly obtains some equity, mileage and sales dividends of Rana [drug source analysis]: the development strategy of shire mRNA drug is similar to the mRNA technology platform of Moderna and curevac The concept of these technology platforms is very direct, that is, if the output of a certain protein in patients is not enough or damaged, the corresponding mRNA will be injected to patients to encode a protein with alternative function In theory, this method of direct injection of mRNA can provide any protein needed, and avoid generating health risks similar to those of some gene therapy, rather than permanently modifying the genome as gene therapy does However, there are some differences between the mRNA drug development platform of Rana and the technology of Moderna and curevac In addition to the development of chemically modified mRNA drugs, Rana also has a transcription platform, which uses oligonucleotides or highly selective small molecule drugs to block the competition of non coding RNA (lncrna) for the formation of chromatin complexes with coding RNA The post transcribed platform of Rana aims to improve the stability or half-life of mRNA in cells and increase protein expression Moderna's mRNA technology platform originated from the chemically modified mRNA discovered by scientists of Penn University It can not only encode proteins with substitution effect, but also serve as antigens to stimulate the immune system to produce therapeutic antibodies Although Moderna currently has only three clinical product lines, it is the most concerned mRNA drug R & D enterprise, with a total financing of about $2 billion and a valuation of $5 billion Rana's technology is close to Moderna, but its strength is obviously much smaller than the latter, and there is no 200000 square foot cGMP production workshop for Moderna Although RNA, Moderna, curevac, Arcturus, and biontech, these mRNA drug R & D companies have made continuous breakthroughs in technology, which makes capital full of confidence in this field, they still haven't completely solved the key problems of delivery, Miss target effect, immunogenicity, etc Of course, 2016 is the year when RNA drugs broke out Ionis and Bainian's SMN2 anti translation RNA drug nusinersen (trade name spinraza) was approved by the US FDA as a Christmas gift Considering the drug price of 750000 US dollars per year, it will become the first heavyweight RNA product in history But also last year, arrowhead, one of the major developers of RNA drugs, terminated the clinical development of EX1 delivery RNA drugs arc520, arc521, and arc-aar due to side effects; revusiran of Alnylam announced a three-phase clinical suspension due to clinical trial death event; mrx34, the main asset of miRNA, terminated the first phase clinical development due to cytokine storm Although RNA drug research and development has entered the main road of new drug development after 30 years of ups and downs, the revolution has not been successful, and comrades still need to work hard.