Hemophilia A gene therapy SB-525 shows clinical efficacy
August 13, 2018 Source: Sina Pharmaceutical News
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];On August 8, Sangamo Therapeutics announced the preliminary positive results of its clinical 1/2-phase study (Alta) for the treatment of hemophilia A with its gene therapy candidate SB-525. Sangamo and Pfizer jointly responsible for the clinical development and commercialization of SB-525 through global cooperation.
The Alta study is an open-label, wide-ranging clinical trial designed to assess the safety and tolerability of SB-525 in 20 patients with severe hemophilia. So far, five patients have received three doses of treatment. The sixth patient is scheduled to receive treatment later this month. In the initial dose escalation phase, the study arranged two patients into a group for study. Preliminary results show:
· SB-525 is generally well tolerated, has no serious adverse events associated with treatment, and has no incidents of oral steroids.
· The fifth patient in the study was the first patient to receive a third dose of the drug, which was started in June and has reached therapeutic hemagglutinin factor VIII activity levels.
· A dose-dependent effect was observed in this study, and patients in the second dose cohort reported reduced use of clotting factors.
Sangamo and Pfizer hope to publish detailed data on the Alta study at the Hematology Conference in the fourth quarter.
Dr. Edward Conner, Chief Medical Officer of Sangamo, said: "In this study, we have made good progress in dose escalation studies, and the safety tolerance of the drug has performed well. The first patient in the third dose group also reached Therapeutic levels of factor VIII. We expect more data to assess the consistency and sustainability of observed factor VIII expression."
SB-525 consists of a recombinant adeno-associated virus (rAAV) vector carrying a proprietary synthetic liver-specific promoter VIII factor gene structure. The US FDA has granted the SB-525 orphan drug and fast-track identification, and the European Medicines Agency has also designated the drug as an orphan drug.
At about the same time, Spark also released the latest data on SPK-8011, a type A hemophilia gene therapy that has received much attention from the industry. Three dose levels were also designed for Phase I/II clinical studies. From the 4th week after SPK-8011 infusion, the annualized bleeding rate (0ABR) was reduced by 97% in the three dose groups, and the annualized infusion was reduced. The rate (AIR) is reduced by 97%. However, the latest data also revealed elevated transaminases, steroid treatment, and serious adverse events. (See: Spark gene therapy new data: reduce type A hemophilia hemorrhage but increase transaminase)
It is reported that SPK-8011 is also an adeno-associated virus (AAV) vector that uses the Spark200 capsid and contains a codon-optimized human factor VIII gene. It can transduce human hepatocytes more efficiently and express a codon-optimized B-domain deleted Factor VIII gene, endogenously producing Factor VIII, for the treatment of hemophilia A.
Hemophilia A is a rare blood disease caused by genetic mutations that cause insufficient factor VIII activity. Coagulation protein is a protein used by the body to stop bleeding. There are approximately 16,000 patients in the United States and more than 150,000 patients with hemophilia A worldwide. (Sina Pharmaceutical Compilation / David)
Article Reference Source: Sangamo Announces Positive Preliminary Data from the Phase 1/2 Alta Study Evaluating SB-525 Gene Therapy for Hemophilia A
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