Recently, gene therapy company Sangamo announced the results of the clinical trial of the world's first individual gene editing experiment at the annual seminar of the Society of Congenital Metabolic Abnormalities (SSIEM) in Athens, Greece. The preliminary results show that the technique is rare. The effect of the disease is safe, but its validity remains to be confirmed by more data.
It is understood that the clinical trial is called CHAMPIONS, the whole test period is 36 weeks, the results of the above publication are from the four patients who participated in the study for 16 weeks, CHAMPIONS aims to correct the genome of patients with rare genetic diseases Hunter syndrome Wrong, Hunter's syndrome is a type II mucopolysaccharidosis (MPSII), and it is also a rare, life-threatening hereditary disease, only 1 in 150,000 people, and the majority of the cases are male, patients Symptoms of the symptoms are mental retardation, degeneration of the body, aggressive behavior and ADHD, and the majority of patients die during puberty. The study found that the disease is due to insufficient or lack of enzyme level of the human lysosomal enzyme iduronic acid-2-sulfate (IDS), which causes the cellular metabolic waste (mucopolysaccharide) to be decomposed and accumulated in tissues and organs. Eventually leading to dysfunction of cell congestion, organ enlargement, tissue destruction and organ system, the current treatment is to receive an infusion of IDS enzyme to supplement the original damaged enzyme in the body. But because healthy enzymes are rapidly depleted with the body, patients must refill with new doses every week.
To this end, the Sangamo R&D team is trying to find a way to permanently replace the enzyme. Sangamo developed SB-913 to insert a new copy of the IDS gene into the precise location of the liver cell DNA using the ZFN genome editing technology to enable the patient's liver to The deficiencies of human IDS enzyme are provided to treat MPS II. SB-913 is a single intravenous infusion of AAV6 vector containing a copy of ZFN and IDS gene, which is designed to be unlocked only by hepatocytes. The pattern enters the cell as an inactive DNA instruction. Once "unlocked", ZFN recognizes, binds to, and cleaves DNA at specific locations within the albumin gene. Sangamo believes that using the natural DNA repair process of cells, hepatocytes can insert the IDS gene into a specific site. Under the driving of the strong albumin promoter, the IDS gene will express the IDS enzyme and be absorbed by tissues and cells. Among them, GAG in dermatan sulfate and heparan sulfate is decomposed into monosaccharides.
The CHAMPIONS study is the first to evaluate in vivo genome editing therapies in humans. The clinical trial plans to assess the safety and efficacy of three different doses of SB-913 therapy during the 36-month study period. Patients received weekly ERT therapy while receiving SB-913. The report included early safety and efficacy results in cohort 1 (low dose group) and cohort 2 (medium dose group) with 2 patients in each treatment group. . The results showed that the total GAGs, dermatan sulfate and heparan sulfate levels in the urine of the cohort 2 were reduced by 51%, 32% and 61% on average 16 weeks after administration. However, the plasma activity of IDS enzymes was consistently lower than the current detection means during the 16 weeks prior to treatment and SB-913 treatment.
In terms of safety, SB-913 was generally well tolerated in all subjects, and no serious adverse events (SAE) associated with SB-913 were reported. Most adverse events (AE) were mild. (Level 1), can be resolved without treatment. All AEs associated with SB-913 were mild (grade 1), resolved without treatment, and showed no evidence of dose dependence. Both SAEs that occurred during the trial were primary MPS II disease and were not associated with SB-913 treatment. In addition, no sustained elevation of transaminase was observed in all subjects.
At the same time, the CHAMPIONS trial also recently injected two subjects in cohort 3 (high dose, 5 times the medium dose), and the safety monitoring committee of the study will review cumulative data for all three dose cohorts later this year. In addition, Sangamo plans to provide long-term safety and effectiveness results for CHAMPIONS research at a world symposium in Orlando, Florida, in February next year.
Sangamo Chief Medical Officer Edward Conner said: "We are encouraged by the safety and tolerability observed so far and the reduction in GAG in the 16th week of the organization. We recently infused two patients in cohort 3, The doses used are five times higher than the medium doses in cohort 2. We expect to see these results. If the long-term data for this study continue to be positive, therapeutic genome editing may bring significant medical advances to MPS II and other monogenic diseases. â€
It should be noted that the researchers also pointed out in the press release that the amount of IDS enzyme in the blood has not been increased correspondingly, which also puts a question mark on the validity of the genome editing. For this, Sangamo President Sandy Macrae The company is investigating a more sensitive method for detecting low levels of enzymes, which they believe may increase in the body's enzymes, suggesting that increased enzymes may be rapidly absorbed from the blood by tissues that continue to require IDS enzymes. . He added that the company may have a clearer answer when testing higher doses of treatment.
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