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Recent popular reports from Yimaike ★ Sanofi has invested 1 billion US dollars on new targets, which is the best multiple myeloma track Yimai Meng broke the news ★ 2021 UCTD How does general cell medicine break through? Click on the picture to register now July 10, 2021/eMedClub News/--Recently, ASC Therapeutics announced that the US FDA has approved its research second-generation gene therapy ASC618 IND application for the treatment of severe or moderate A hemophilia
.
ASC618 is a gene therapy AAV, carrying the codon-optimized B-domain deleted Factor VIII gene and the minimum length of the liver-specific promoter can be endogenous production of factor VIII, thereby treating hemophilia A
.
Preclinical studies show, at significantly lower than other hemophilia A gene therapy dosages, ASC618 still able to produce a sufficient level of factor VIII
.
According to reports, ASC618 uses ASC's "high secretion" protein optimization strategy
.
When constructing the gene, special genetic modification was made and a series of optimizations were made to the unfold protein response (UPR), which greatly improved the secretion efficiency of intracellular therapeutic protein in the process of gene therapy.
Only 1/10 of the traditional AAV vector can be used.
The dose can reach the same therapeutic protein secretion
.
This strategy greatly reduces the dose level of AAV gene therapy, directly reduces hepatotoxic side effects caused by gene therapy, and improves the safety of gene therapy
.
Dr.
Jiang Ruhong, founder and CEO of ASC Therapeutics, said: "The FDA's IND approval of ASC618 is an important recognition of our discovery, preclinical, analytical, clinical, regulatory, quality, manufacturing and overall capabilities
.
This milestone marks us.
Transition to a clinical stage gene and cell therapy biopharmaceutical company
.
"ASC Therapeutics ASC Therapeutics was established in 2008 and is headquartered in the San Francisco Bay Area, California, with strategic deployments in the United States and China
.
Since its establishment in 2008, ASC Therapeutics has received tens of millions of US dollars in Series A and Series B financing from several internationally renowned bio venture capital institutions during its development.
"Biotechnology (Nature Biotechnology)" was named "Global Top 10 Companies Leading the Application Technology of Precision Gene Editing"
.
ASC owns TARGATT™, TARGATT™-Alt and CRISPR technology platforms, and has developed a variety of cell and gene therapies for blood diseases based on these technology platforms
.
▲ ASC Therapeutics’ R&D pipeline (picture source: company’s official website) ASC’s fastest-growing product pipeline is ASC-930, an allogeneic ecdysis for the treatment of steroid-refractory graft-versus-host disease (SR-aGvHD) Membrane stromal cells (DSC) are currently in phase 2 clinical studies
.
ASC's core products also include ASC-518, a gene editing therapy for children and all ages of hemophilia
.
ASC-518 can overcome the limitations of traditional AAV gene therapy (persistence problems caused by cell division/loss).
ASC-518 can directly edit the DNA in the nucleus and permanently retain it in the patient's body
.
ASC Therapeutics expects to advance ASC-518 into clinical trials in the second quarter of 2022
.
Reference materials: 1.
https:// -gene-therapy-for-hemophilia-a/2.
Arterial Network "Optimizing protein secretion, reducing 90% AAV dose for gene therapy, ASC Therapeutics new drug for hemophilia approved in the United States IND" Yimike has always been committed to the forefront of innovative biological drugs Original news reports on technology, industry trends, industry insights, etc.
, all-media high-end matrix users reached 160,000+, of which industrial users accounted for more than 50%, scientific research and clinical users accounted for about 30%, and investment institutions accounted for more than 5%
.
In order to promote interactive exchanges in industry segments, we have established a number of professional WeChat groups, welcome to scan the QR code to add groups
.
.
ASC618 is a gene therapy AAV, carrying the codon-optimized B-domain deleted Factor VIII gene and the minimum length of the liver-specific promoter can be endogenous production of factor VIII, thereby treating hemophilia A
.
Preclinical studies show, at significantly lower than other hemophilia A gene therapy dosages, ASC618 still able to produce a sufficient level of factor VIII
.
According to reports, ASC618 uses ASC's "high secretion" protein optimization strategy
.
When constructing the gene, special genetic modification was made and a series of optimizations were made to the unfold protein response (UPR), which greatly improved the secretion efficiency of intracellular therapeutic protein in the process of gene therapy.
Only 1/10 of the traditional AAV vector can be used.
The dose can reach the same therapeutic protein secretion
.
This strategy greatly reduces the dose level of AAV gene therapy, directly reduces hepatotoxic side effects caused by gene therapy, and improves the safety of gene therapy
.
Dr.
Jiang Ruhong, founder and CEO of ASC Therapeutics, said: "The FDA's IND approval of ASC618 is an important recognition of our discovery, preclinical, analytical, clinical, regulatory, quality, manufacturing and overall capabilities
.
This milestone marks us.
Transition to a clinical stage gene and cell therapy biopharmaceutical company
.
"ASC Therapeutics ASC Therapeutics was established in 2008 and is headquartered in the San Francisco Bay Area, California, with strategic deployments in the United States and China
.
Since its establishment in 2008, ASC Therapeutics has received tens of millions of US dollars in Series A and Series B financing from several internationally renowned bio venture capital institutions during its development.
"Biotechnology (Nature Biotechnology)" was named "Global Top 10 Companies Leading the Application Technology of Precision Gene Editing"
.
ASC owns TARGATT™, TARGATT™-Alt and CRISPR technology platforms, and has developed a variety of cell and gene therapies for blood diseases based on these technology platforms
.
▲ ASC Therapeutics’ R&D pipeline (picture source: company’s official website) ASC’s fastest-growing product pipeline is ASC-930, an allogeneic ecdysis for the treatment of steroid-refractory graft-versus-host disease (SR-aGvHD) Membrane stromal cells (DSC) are currently in phase 2 clinical studies
.
ASC's core products also include ASC-518, a gene editing therapy for children and all ages of hemophilia
.
ASC-518 can overcome the limitations of traditional AAV gene therapy (persistence problems caused by cell division/loss).
ASC-518 can directly edit the DNA in the nucleus and permanently retain it in the patient's body
.
ASC Therapeutics expects to advance ASC-518 into clinical trials in the second quarter of 2022
.
Reference materials: 1.
https:// -gene-therapy-for-hemophilia-a/2.
Arterial Network "Optimizing protein secretion, reducing 90% AAV dose for gene therapy, ASC Therapeutics new drug for hemophilia approved in the United States IND" Yimike has always been committed to the forefront of innovative biological drugs Original news reports on technology, industry trends, industry insights, etc.
, all-media high-end matrix users reached 160,000+, of which industrial users accounted for more than 50%, scientific research and clinical users accounted for about 30%, and investment institutions accounted for more than 5%
.
In order to promote interactive exchanges in industry segments, we have established a number of professional WeChat groups, welcome to scan the QR code to add groups
.
