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Recent popular reports from Yimicke ★Jianxun Wang, Chief Scientist of Junhou Bio: The first CAR-T retroviral vector CDMO platform in China to develop vector development for next-generation gene and cell therapyFengke Creative ★The organoid market is expected to reach billions in the future Level, how does domestic organoid research keep up with the international? MedClub broke the news March 25, 2021/eMedClub News/--Recently, at the MDA virtual clinical and scientific conference, Solid announced the latest clinical research results for Duchenne muscular dystrophy (DMD).
In terms of efficacy, IGNITE DMD data shows that the new drug SGT-001 has improved in some indicators, and no serious side effects occurred in the seventh patient's administration.
IGNITE-DMD is an open-label Phase I/II escalating dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne's muscular dystrophy (DMD) .
SGT-001 is designed to deliver a functional substitute for the DMD gene, which contains a substitute for the function of the nNOS gene that is believed to protect against ischemic injury.
The therapy uses AAV9 as a gene carrier to deliver the truncated mini-dystrophin gene to the patient’s muscle cells, replacing the genetically defective dystrophin gene, and allowing the patient to produce a certain amount of dystrophin.
Cells are effectively protected.
In the IGNITE DMD Phase 1/2 clinical trial, 6 subjects clearly benefited from the results, but some patients had serious adverse reactions, and the clinical trial was suspended for more than a year.
After Solid revised the clinical protocol, the experiment will be restarted in 2020.
The Biopharmaceutical Innovation Technology Conference (BPIT) will kick off in Shanghai from April 9th to 10th, 2021.
The four major conference venues are exciting (click to view the focus topics).Participants who successfully registered for the Shanghai 2021 BPIT conference will receive a limited edition of "CAR-T Cell Therapy Industry Research Report" 2021.
V3.
1 produced by Xingyao Research Institute under Medical Media.
In terms of efficacy, IGNITE DMD data shows that the new drug SGT-001 has improved in some indicators, and no serious side effects occurred in the seventh patient's administration.
IGNITE-DMD is an open-label Phase I/II escalating dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne's muscular dystrophy (DMD) .
SGT-001 is designed to deliver a functional substitute for the DMD gene, which contains a substitute for the function of the nNOS gene that is believed to protect against ischemic injury.
The therapy uses AAV9 as a gene carrier to deliver the truncated mini-dystrophin gene to the patient’s muscle cells, replacing the genetically defective dystrophin gene, and allowing the patient to produce a certain amount of dystrophin.
Cells are effectively protected.
In the IGNITE DMD Phase 1/2 clinical trial, 6 subjects clearly benefited from the results, but some patients had serious adverse reactions, and the clinical trial was suspended for more than a year.
After Solid revised the clinical protocol, the experiment will be restarted in 2020.
The Biopharmaceutical Innovation Technology Conference (BPIT) will kick off in Shanghai from April 9th to 10th, 2021.
The four major conference venues are exciting (click to view the focus topics).Participants who successfully registered for the Shanghai 2021 BPIT conference will receive a limited edition of "CAR-T Cell Therapy Industry Research Report" 2021.
V3.
1 produced by Xingyao Research Institute under Medical Media.
