-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
▎WuXi AppTec content team editor
Fabry disease is a lysosomal storage disease in which there is α insufficient activity of the enzyme enzyme α-galactosidase A, which is necessary for metabolizing acylsphingosine trihexose (Gb3
) due to mutations in the gene encoding α-galactosidase.
The buildup of Gb3 in cells can cause serious damage to vital organs such as the kidneys, heart, nerves, eyes, intestines, and skin
.
Symptoms of Fabry disease are varied and include decreased or absent sweat production, heat intolerance, angiokeratomas (skin spots), vision problems, kidney disease, heart failure, gastrointestinal disorders, mood abnormalities, neuropathic pain, and tingling at the ends of the
limbs.
The current standard of care for Fabry's disease is enzyme replacement therapy (ERT), but this therapy is not only slow to act, does not solve the symptoms in the brain and nervous system, and is more likely to trigger an immune response in patients, resulting in a decline
in efficacy.
of gene and cell therapies and other high-end therapies.
WuXi Biologics can help customers around the world bring more innovative therapies to market sooner rather than later, benefiting patients
.
If you have related business needs, please click the picture below to fill in the specific information
.
▲If you have any business needs, please long press to scan the QR code above, or
Sangamo Therapeutics announced the latest results
of a Phase 1/2 clinical trial evaluating its gene therapy ST-920 for Fabry disease.
According to newly released data, the overall safety and tolerability of the therapy was good, with significant and lasting improvements in levels of the key biomarker α-galactosidase A (α-Gal A) in all patients, up to 23 months
by the end of follow-up.
Fabry disease is a lysosomal storage disease in which there is α insufficient activity of the enzyme enzyme α-galactosidase A, which is necessary for metabolizing acylsphingosine trihexose (Gb3
) due to mutations in the gene encoding α-galactosidase.
The buildup of Gb3 in cells can cause serious damage to vital organs such as the kidneys, heart, nerves, eyes, intestines, and skin
.
Symptoms of Fabry disease are varied and include decreased or absent sweat production, heat intolerance, angiokeratomas (skin spots), vision problems, kidney disease, heart failure, gastrointestinal disorders, mood abnormalities, neuropathic pain, and tingling at the ends of the
limbs.
The current standard of care for Fabry's disease is enzyme replacement therapy (ERT), but this therapy is not only slow to act, does not solve the symptoms in the brain and nervous system, and is more likely to trigger an immune response in patients, resulting in a decline
in efficacy.
This study is a global, open-label, single-dose, dose-ranged, multicenter Phase 1/2 trial to evaluate the safety and tolerability of ST-920, a one-time, liver-targeted Fabry disease gene therapy candidate that has been granted orphan drug designation
by the US FDA and the European Medicines Agency 。 A total of 9 patients were included in the study divided into 4 dose groups (0.
5e13 vg/kg, 1e13 vg/kg, 3e13 vg/kg, and 5e13 vg/kg) for dose escalation testing, and up to the end of the data, the therapy remained well tolerated and no treatment-related serious adverse events
occurred in the trial.
All 9 patients showed a sustained increase in α-Gal A enzyme activity (approximately 2-30 times normal), with the longest follow-up lasting 23 months
.
Based on follow-up results, these patients maintained high α-Gal A enzyme levels
even after stopping standard therapy.
Emboldened by these data, Sangamo Therapeutics has initiated an expanded trial of the study, dosing
four patients.
At the same time, the company is also planning to launch a Phase 3 study
of ST-920 gene therapy.
"We have been excited about the bright future of the data on the Company's Fabry disease program," said Dr.
Nathalie Dubois-Stringfellow, senior vice president and chief development officer at Sangamo Therapeutics α
, in a press release 。 We are preparing for future Phase 3 trials and we look forward to sharing more data
from the nine dose-escalation phase patients as well as our newly initiated extended phase patients.
" ”
of gene and cell therapies and other high-end therapies.
WuXi Biologics can help customers around the world bring more innovative therapies to market sooner rather than later, benefiting patients
.
If you have related business needs, please click the picture below to fill in the specific information
.
▲If you have any business needs, please long press to scan the QR code above, or
