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October 14, 2022 /eMedClub News / -- Recently, Sangamo Therapeutics announced its AAV gene therapy ST-920 The latest data from a Phase 1/2 clinical trial (STAAR) for the treatment of patients with Fabry's disease.
The results showed that as of July 21 this year, 9 patients treated with ST-920 had α- Galactosidase A (α-Gal A) activity continues to rise for up to 23 months
。
Fabry disease is a rare X-linked inherited lysosomal storage disorder caused by mutations in the α-galactosidase A gene (GLA), resulting in a lack of α-Gal A activity and inability to metabolize sphingosine trimerexin (Gb3)
normally.
The current standard of care for most patients is intravenous enzyme replacement therapy (ERT), but this therapy is slow to respond and does not address symptoms in the brain and nervous system, and is more likely to trigger an immune response that leads to a decline
in efficacy.
At the same time, lifelong infusions every other week place a huge burden
on patients and families.
ST-920, on the other hand, requires a single infusion without pretreatment and is designed to deliver a functional copy of the GLA gene to the patient's liver via a rAAV 2/6 vector, allowing cells to resume production of α-Gal A, thereby providing stable, long-term levels of α-GalA activity
in patients with Fabry's disease 。 Importantly, the continued production of α-Gal A reduces and potentially clears the Fabry disease substrate Gb3 and plasma sphingosine trimerexoside (lyso-Gb3).
The therapy has now received orphan drug designation
from the FDA and EMA.
▲ AAV gene therapy for Fabry disease Source: Sangamo official website
The STAAR study included nine patients
who were receiving ERT, who had stopped ERT for 6 months or more, and who had not received ERT.
The data showed that in all patients with Fabry disease treated with four dose groups (0.
5e13 vg/kg, 1e13 vg/kg, 3e13 vg/kg, and 5e13 vg/kg), the activity of α-Gal A was 2-30 times
higher than the normal average at the time of the last measurement.
Of these, 5 patients were free of ERT and two patients with the highest lyso-Gb3 were 40-55%
lower than baseline after treatment.
In terms of safety, the four dose cohorts were generally well tolerated, with no treatment-related serious adverse events or elevated
liver enzymes requiring steroid therapy.
Based on the positive data obtained above, ST-920 is expected to provide an alternative to the current standard of care, and Sangamo is currently planning a phase 3 clinical trial in anticipation of further progress
with this therapy.
Progress of AAV gene therapy for rare diseases at home and abroad
At present, there are more than 7,000 known rare diseases in the world, accounting for about 10% ofhuman diseases.
Among the wide variety of rare diseases, about 80% are caused by genetic defects and are hereditary
.
In recent years, gene therapy has brought new hope to common and rare genetic diseases, and in the race of gene therapy, AAV vectors have quickly become one of the main platforms for gene introduction in vivo, showing obvious therapeutic effects in multiple disease treatment clinical trials without causing significant adverse immune responses
.
At present, the global research and development of AAV gene therapy rare diseases is hot, and the rapid progress at home and abroad has entered phase III clinical trials
。 On October 12, 2022, BioMarin Pharmaceutical's biologics license application for the AAV gene therapy valoctocogene roxaparvovec (BMN 270) for the treatment of adults with severe hemophilia A was accepted by the FDA, which means that gene therapy has taken another big step
forward in the field of rare diseases.
There are also many domestic companies developing AAV gene therapy for rare diseases, among which Newforth Biologics has made rapid clinical progress in ophthalmic gene therapy and has entered clinical phase
III.
▲ Progress of AAV gene therapy in the treatment of rare diseases
epilogue
It is undeniable that gene therapy has brought more and better treatment options to patients with rare diseases, and in recent years, gratifying progress has been made in the field of rare disease treatment, and the enthusiasm of pharmaceutical companies for research and development has also increased, and various companies and cutting-edge companies are competing for layout.
But we also need to realize that the number of rare diseases is huge, and there are still a large number of gaps to innovate and break through
.
It is expected that with the continuous expansion of the field of AAV research and development, researchers will be able to overcome the challenges currently facing the field, unleash the full potential of AAV gene therapy, and bring new hope
to patients with rare diseases.
Resources:
1.
com/article/releases/sangamo-therapeutics-announces-updated-preliminary-phase-1-2-data-in-fabry-disease-showing-continued-tolerability-and-sustained-elevated-%ce% b1-gal-a-enzyme-activity-in-nine-patients/
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