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Recent popular reports from Yimaike ★The first case of Vertex's stem cell-derived therapy for the treatment of type 1 diabetes on the 90th day has positive resultsYikemeng broke the news ★Eisai/Biogen wants to get accelerated approval by the FDA, and the Alzheimer’s drug development rules are rewrittenMedical Mai Meng broke the news November 3, 2021/MedClub News/--October 27, 2021, GABAeron’s scientists shared their latest research and development results at the ISSCR/JSRM (Tokyo, Japan) conference.
The company’s development is more inducing Human interneuron precursor cells derived from competent stem cells (iPSC) can be successfully transplanted and integrated into the mouse brain.
The transplanted cells can mature in the body and reverse the dysfunction of the hippocampal network associated with Alzheimer's disease
.
GABAeron recently released an iPSC-based first-in-class cell therapy product at the international conference "Stem Cells: From Basic Science to Clinical Transformation" organized by the International Society for Stem Cell Research (ISSCR) and the Japanese Society for Regenerative Medicine (JSRM).
The product has shown gratifying therapeutic potential in preclinical studies for the treatment of Alzheimer's disease
.
This is the company’s first public sharing of research data since its establishment in 2017, focusing on the use of transplantation of human iPSC-derived interneuron precursor cells in the treatment of Alzheimer’s disease and other neurological diseases accompanied by the loss or loss of interneurons.
Potential
.
GABAeron scientists successfully differentiated GABAergic interneuron precursor cells from human iPSCs, and showed that when these cells were transplanted into a mouse model of Alzheimer’s disease carrying APOE4 (apolipoprotein E4, the main genetic risk factor) When in the brain, these cells can mature and integrate into the hippocampus, reversing the hippocampal network dysfunction associated with Alzheimer’s disease
.
"We are extremely excited about this.
These data demonstrate the safety and effectiveness of our new iPSC-based therapy in a mouse model of Alzheimer's disease
.
" said Dr.
Robert W.
Mahley, CEO and Chief Scientific Officer of GABAeron "Based on these results, we will continue to advance research and development, and then develop a cell replacement therapy to treat APOE4-positive Alzheimer's patients
.
" Dr.
Mahley is the founding first director of the Glaston Institute, University of California Professor of Pathology and Medicine at the University of San Francisco, dedicated to the research of APOE protein for many years
.
APOE gene has several different genotypes.
Compared with the common APOE3 genotype population, the APOE4 genotype population has an increased risk of Alzheimer's disease, and the age of onset is earlier
.
It is worth noting that APOE4 is associated with 60-75% of Alzheimer's cases
.
Dr.
Huang Yadong, the scientific co-founder of GABAeron, is the director of the Glaston Institute's Transformation Promotion Center.
He discovered why APOE4 is related to the onset of Alzheimer's disease
.
Dr.
Huang’s laboratory proved that APOE4 can cause damage and loss of GABAergic interneurons in the hippocampus
.
GABAergic interneurons are key cells to maintain normal hippocampal activity and are necessary for normal learning and memory, but they are damaged or lost in the brains of Alzheimer's patients
.
"The establishment of GABAeron is based on the premise that if we can replace these interneurons through cell therapy, we can restore normal hippocampal activity, thereby slowing or reversing many memory and cognitive impairments related to Alzheimer's disease
.
"Dr.
Huang said, "If this method works, a single treatment may have a lasting impact on the patient
.
"In this new study, the research team led by Dr.
Wen-Chin (Danny) Huang and Iris Avellano has developed a new and effective method that can induce human iPSCs carrying the APOE3 genotype to differentiate into GABAergic interneurons.
Precursor cells, and the obtained cells show high viability, high purity and powerful functionality, and more than 90% of the cells have the correct developmental lineage
.
The research team led by Dr.
Wan-Ying Hsieh transplanted these interneuron precursor cells In the hippocampus of 10-month-old mice carrying human APOE4 genotype; interneuron precursor cells showed strong survival ability and matured into functional GABAergic interneurons
.
At 7 months after transplantation, more than half of the The surviving cells have migrated out of the local area, distributed to the hippocampus, and established connections with other existing neurons throughout the hippocampus
.
"These exciting data show that GABAeron’s human iPSC-derived interneuron precursor cells have very High quality, long-term survival and integration into the mouse brain," Dr.
Hsieh said, "The important thing is that the transplanted cells of more than 100 mice did not form tumors
.
"Then the mice’s hippocampal network activity was studied through electrophysiological recording
.
As expected, APOE4 mice’s hippocampal activity was defective, suggesting memory impairments related to Alzheimer’s disease, namely, sharp ripples in the hippocampus of mice.
The related slow gamma energy is less, and these two energies are essential for the formation and recovery of memory
.
When each mouse is transplanted with about 120,000 human interneuron precursor cells derived from iPSC carrying APOE3, 7 Months later, the measured values of these hippocampal activity were improved to the level of healthy mice
.
"Overall, these results reveal a key step in the treatment of APOE4-related Alzheimer's disease by potential interneurons
.
"GABAeron's senior director, Dr.
Qin Xu, said, "This has laid a solid foundation for us to further develop clinical-grade human iPSC
.
"GABAeron scientists are currently adjusting the relevant cell culture technology to achieve large-scale production of clinical-grade human iPSC-derived GABAergic interneuron precursor cells
.
They will further determine the molecular characteristics of mature GABAergic interneurons.
These interneurons Successfully integrated into the hippocampus in a mouse model of Alzheimer's disease
.
"With the realization of this key milestone, GABAeron will confidently advance future human clinical trials," said Dr.
Sheng Ding, co-founder of GABAeron Science.
"We also plan to explore the effectiveness of this cell-based therapy for other neurological diseases that accompany the loss or loss of interneurons
.
"As a serial entrepreneur, Dr.
Ding Sheng participated in the founding of two leading listed companies, Fate Therapeutics (FATE) and Tenaya Therapeutics (TNYA)
.
About GABAeron GABAeron is a biopharmaceutical company focused on the treatment of neurodegenerative diseases, 2017 Founded in the United States, based on the pioneering work of the Glaston Institute, it is committed to combining precision medicine, regenerative medicine and drug intervention
.
GABAeron is exploring a new first-in-class IND drug candidate to replace or Repair damaged or lost neurons in the brains of patients with neurodegenerative and neurodevelopmental disorders
.
Reference materials: 1.
https:// 2.
.
com
The company’s development is more inducing Human interneuron precursor cells derived from competent stem cells (iPSC) can be successfully transplanted and integrated into the mouse brain.
The transplanted cells can mature in the body and reverse the dysfunction of the hippocampal network associated with Alzheimer's disease
.
GABAeron recently released an iPSC-based first-in-class cell therapy product at the international conference "Stem Cells: From Basic Science to Clinical Transformation" organized by the International Society for Stem Cell Research (ISSCR) and the Japanese Society for Regenerative Medicine (JSRM).
The product has shown gratifying therapeutic potential in preclinical studies for the treatment of Alzheimer's disease
.
This is the company’s first public sharing of research data since its establishment in 2017, focusing on the use of transplantation of human iPSC-derived interneuron precursor cells in the treatment of Alzheimer’s disease and other neurological diseases accompanied by the loss or loss of interneurons.
Potential
.
GABAeron scientists successfully differentiated GABAergic interneuron precursor cells from human iPSCs, and showed that when these cells were transplanted into a mouse model of Alzheimer’s disease carrying APOE4 (apolipoprotein E4, the main genetic risk factor) When in the brain, these cells can mature and integrate into the hippocampus, reversing the hippocampal network dysfunction associated with Alzheimer’s disease
.
"We are extremely excited about this.
These data demonstrate the safety and effectiveness of our new iPSC-based therapy in a mouse model of Alzheimer's disease
.
" said Dr.
Robert W.
Mahley, CEO and Chief Scientific Officer of GABAeron "Based on these results, we will continue to advance research and development, and then develop a cell replacement therapy to treat APOE4-positive Alzheimer's patients
.
" Dr.
Mahley is the founding first director of the Glaston Institute, University of California Professor of Pathology and Medicine at the University of San Francisco, dedicated to the research of APOE protein for many years
.
APOE gene has several different genotypes.
Compared with the common APOE3 genotype population, the APOE4 genotype population has an increased risk of Alzheimer's disease, and the age of onset is earlier
.
It is worth noting that APOE4 is associated with 60-75% of Alzheimer's cases
.
Dr.
Huang Yadong, the scientific co-founder of GABAeron, is the director of the Glaston Institute's Transformation Promotion Center.
He discovered why APOE4 is related to the onset of Alzheimer's disease
.
Dr.
Huang’s laboratory proved that APOE4 can cause damage and loss of GABAergic interneurons in the hippocampus
.
GABAergic interneurons are key cells to maintain normal hippocampal activity and are necessary for normal learning and memory, but they are damaged or lost in the brains of Alzheimer's patients
.
"The establishment of GABAeron is based on the premise that if we can replace these interneurons through cell therapy, we can restore normal hippocampal activity, thereby slowing or reversing many memory and cognitive impairments related to Alzheimer's disease
.
"Dr.
Huang said, "If this method works, a single treatment may have a lasting impact on the patient
.
"In this new study, the research team led by Dr.
Wen-Chin (Danny) Huang and Iris Avellano has developed a new and effective method that can induce human iPSCs carrying the APOE3 genotype to differentiate into GABAergic interneurons.
Precursor cells, and the obtained cells show high viability, high purity and powerful functionality, and more than 90% of the cells have the correct developmental lineage
.
The research team led by Dr.
Wan-Ying Hsieh transplanted these interneuron precursor cells In the hippocampus of 10-month-old mice carrying human APOE4 genotype; interneuron precursor cells showed strong survival ability and matured into functional GABAergic interneurons
.
At 7 months after transplantation, more than half of the The surviving cells have migrated out of the local area, distributed to the hippocampus, and established connections with other existing neurons throughout the hippocampus
.
"These exciting data show that GABAeron’s human iPSC-derived interneuron precursor cells have very High quality, long-term survival and integration into the mouse brain," Dr.
Hsieh said, "The important thing is that the transplanted cells of more than 100 mice did not form tumors
.
"Then the mice’s hippocampal network activity was studied through electrophysiological recording
.
As expected, APOE4 mice’s hippocampal activity was defective, suggesting memory impairments related to Alzheimer’s disease, namely, sharp ripples in the hippocampus of mice.
The related slow gamma energy is less, and these two energies are essential for the formation and recovery of memory
.
When each mouse is transplanted with about 120,000 human interneuron precursor cells derived from iPSC carrying APOE3, 7 Months later, the measured values of these hippocampal activity were improved to the level of healthy mice
.
"Overall, these results reveal a key step in the treatment of APOE4-related Alzheimer's disease by potential interneurons
.
"GABAeron's senior director, Dr.
Qin Xu, said, "This has laid a solid foundation for us to further develop clinical-grade human iPSC
.
"GABAeron scientists are currently adjusting the relevant cell culture technology to achieve large-scale production of clinical-grade human iPSC-derived GABAergic interneuron precursor cells
.
They will further determine the molecular characteristics of mature GABAergic interneurons.
These interneurons Successfully integrated into the hippocampus in a mouse model of Alzheimer's disease
.
"With the realization of this key milestone, GABAeron will confidently advance future human clinical trials," said Dr.
Sheng Ding, co-founder of GABAeron Science.
"We also plan to explore the effectiveness of this cell-based therapy for other neurological diseases that accompany the loss or loss of interneurons
.
"As a serial entrepreneur, Dr.
Ding Sheng participated in the founding of two leading listed companies, Fate Therapeutics (FATE) and Tenaya Therapeutics (TNYA)
.
About GABAeron GABAeron is a biopharmaceutical company focused on the treatment of neurodegenerative diseases, 2017 Founded in the United States, based on the pioneering work of the Glaston Institute, it is committed to combining precision medicine, regenerative medicine and drug intervention
.
GABAeron is exploring a new first-in-class IND drug candidate to replace or Repair damaged or lost neurons in the brains of patients with neurodegenerative and neurodevelopmental disorders
.
Reference materials: 1.
https:// 2.
.
com
