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Sanofi began to seek new technologies to improve its gene therapy
Sanofi began to seek new technologies to improve its gene therapy. Sanofi has reached a cooperation with the German Sirion Biotechnology Company to use its proprietary technology platform to develop viral vector-based gene delivery technology, and at the same time provide better and safer adeno-associated virus (AAV) vectors with improved tissue selectivity Gene therapy.
Since the first success of gene therapy, it has attracted large companies such as Roche, Novartis, Bayer, South Bird Bio, Voyager Therapeutics, Pfizer, Eli Lilly and other large companies. Up to now, 22 new gene therapy drugs have been launched to address some unmet clinical needs.
According to a report previously released by Evaluate Pharma, it is expected that as many as 60 gene therapies will be approved in the next six years, and its sales in 2024 will reach $14.6 billion, accounting for approximately 1.2% of total global pharmaceutical revenue. Therefore, all pharmaceutical companies are constantly improving their technology to seize the market.
Sanofi has always believed that it has decades of expertise in the production of inactivated viruses, which can be well transformed into gene therapy. Therefore, Sanofi has decided to greatly reduce R&D investment in the field of cardiovascular disease and diabetes, and prioritize gene therapy. the study. Since the acquisition of Bioverativ, a US biotechnology company that was separated from Biogen and focused on the treatment of hemophilia, for USD 11.6 billion in 2018, Sanofi has begun to focus on gene therapy.
In recent years, Sanofi has continuously announced its research results in the field of gene therapy. Based on comprehensive data, Sanofi’s announced failed projects have exceeded all projects that have achieved phased success. Therefore, Sanofi began to seek new technologies to improve its gene therapy.
01 Active progress in many disease fields harvested in recent years
In May 2018, Sanofi’s subsidiary Bioverativ and Sangamo jointly announced that the FDA had accepted the IND application for its candidate gene therapy BIVV003 for the treatment of sickle cell disease, and subsequently began a phase 1/2 clinical trial to test the effect of BIVV003 in sickle cell disease Safety, tolerability and efficacy in patients.
In October 2019, Sanofi’s first AAV-based gene therapy entered the clinic for the treatment of rare ophthalmology in Leber congenital amaurosis (LCA) caused by autosomal recessive guanylate cyclase 2D (GUCY2D) mutations disease.
In May 2020, Sanofi announced that their new PAH gene fragment optimized PKU gene therapy has made new progress, in which the modified PAH gene fragment (hPAH-V1) is 7 times more efficient than the unmodified version. Then they packaged hPAH-V1 into an AAV vector for testing in a mouse model. The study found that the phenylalanine content in the blood and brain dropped significantly. In the liver, after hPAH-V1 treatment, the content of phenylalanine hydroxylase is 10 times that of the unmodified version.
Sanofi and Oxford BioMedica (OXB), a developer of lentiviral vectors, have formed a cooperative relationship more than 10 years ago, and used OXB to develop and produce lentiviral vectors for the treatment of hemophilia. Gene therapy for hemophilia.
Unlike the adeno-associated virus (AAV) vectors currently used in clinical practice, lentiviral gene therapy does not have existing neutralizing antibodies in humans, and can integrate transgenes into the genome of patient cells. These transgenes can be accompanied by The patient’s cells divide and grow, thereby providing the possibility of “cure”. Based on the previous effects, the gene therapy is expected to enter the clinical stage in 2022.
In addition, on February 24, Sanofi reached a cooperation with Sirion Biotechnology in Germany to develop gene delivery technology based on viral vectors. Among them, Sirion has developed a new type of therapeutic viral vector, which can use a proprietary technology platform based on lentivirus, adenovirus and adeno-associated virus to help partners improve the efficiency of new drug development. In addition, Sanofi will also cooperate with Professor Dirk Grimm, a partner of Heidelberg University Hospital in Germany, to provide better and safer gene therapy through new and improved tissue-selective adeno-associated virus (AAV) vectors.
“Through this cooperation, we will increase our technical level in the important emerging field of gene therapy.” Christian Mueller, head of Sanofi’s genomic medicine, said: “Using our expertise in viral vaccines and viral vector manufacturing, plus Dirk With Professor Grimm’s cutting-edge AAV capsid evolution technology and Sirion’s expertise and capabilities in AAV vector manufacturing, we will be able to better meet unmet medical needs and open up new directions for drug discovery in genomic medicine.”
02 Although it has been in the game for many years, it is still waiting for a “success”
In order to catch up in the field of gene therapy, Sanofi will transform a vaccine facility near Lyon, France in 2019 to produce GMP-grade AAV virus vectors. However, Sanofi still has problems that cannot be solved. For example, in June 2020, Sanofi gave up two gene therapies that could not be completed and returned them to OXB. It is reported that these two therapies can treat Stargardt disease (hereditary macular degeneration) and Usher syndrome (hereditary deafness) respectively.
In October 2017, Sanofi announced its withdrawal from the Parkinson’s gene therapy project in cooperation with Voyager. Two years later, Sanofi also gave up the cooperation project signed with Voyager for the treatment of Huntington’s disease and Friedrich’s ataxia two gene therapies. According to Voyager, its gene therapy program for the treatment of Parkinson’s disease and Huntington’s disease gene therapy program itself has great commercial value, and immediately after Sanofi withdrew, it sought a new partner to continue research on gene therapy for Huntington’s disease.
Taking a comprehensive look at Sanofi’s current layout, its gene therapy mainly involves rare diseases and blood diseases such as hemophilia, and these fields themselves have certain challenges. Only in the field of hemophilia, there is strong competition. Among them, Roche, Pfizer, BioMarin, Bayer and other pharmaceutical companies have also announced their latest developments in hemophilia gene therapy, but there is still no product on the market, and the once most promising BioMarin hemophilia gene therapy product Roctavian is also unable to Proved whether it has a long-term effect on the bleeding rate and was rejected by the FDA.
For Sanofi’s progress in the field of gene therapy, Shell Club will continue to keep an eye on it.
(source:internet, reference only)