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Recent popular reports from Yimike ★ Genxi Bio announced the completion of the first patient enrollment in the Phase I/II registered clinical study of the donor-derived allogeneic CAR-T cell therapy GC007g for relapsed/refractory B-ALL.
Automated cell culture: Quantum liberates hands and redefines cell therapyMedClub broke the news April 3, 2021/eMedClub News/--April 1, 2021, Passage Bio announced the first case in the Imagine-1 study Infant GM1 ganglion patients have been successfully administered PBGM01.
At present, the patient has been taking the drug in the global phase 1/2 clinical trial plan of PBGM01 and has received PBGM01 treatment at the Children's Hospital of Saint Peter's University Hospital.
Dr.
Bruce Goldsmith, President and CEO of Passage Bio, said: "The company aims to develop treatments for rare central nervous system diseases.
We are excited for the first patient's drug delivery to promote the clinical development of PBGM01 and look forward to sharing clinical safety data and safety data at the end of the year.
Biomarker data.
"Recommended reading: An AAV gene therapy IND application founded by James Wilson, a company founded by AAV Daniel, has been approved by the US FDAYi Mai Meng reveals the mechanism of PBGM01 in the treatment of GM1.
Imagine-1 is a Phase 1/2 multinational study , Designed to test the safety, tolerability and effectiveness of PBGM01 in early-onset (type 1) and late-onset (type 2a) GM1 patients.
The study was confirmed by low β-gal enzyme activity and genetic testing.
GM1 gangliosidosis (GM1 gangliosidosis) is an autosomal recessive genetic disease caused by a lack of the lysosomal enzyme β-galactosidase (β-gal) encoded by the GLB1 gene mutation.
β-gal is an enzyme necessary for the degradation of GM1 ganglioside and keratan sulfate.
The decrease of β-gal level will cause the accumulation of the toxic level of GM1 ganglioside in the whole brain neurons, leading to the rapid development of the disease.
Infant GM1 is the most common and severe form of the disease, usually with abnormal gait at 4 months and degeneration at 6 months.
Babies with the disease experience rapid and fatal degeneration at approximately 2 years of age, with a life expectancy of 2-4 years.
PBGM01 is an AAV gene therapy under development for the treatment of infantile GM1.
PBGM01 is administered by ICM injection (injected into the cerebellar medulla oblongata at the craniocervical junction).
The AAVhu68 capsid is used to deliver the GLB1 gene encoding β-gal to the brain and surrounding tissues.
PBGM01 reduces the accumulation of GM1 gangliosides by increasing the level of β-gal.
PBGM01 has the potential to reverse neuronal toxicity, so that GM1 infants can resume their development.
In preclinical models, the wide distribution of PBGM01 in the brain and the wide absorption of β-gal in the central nervous system (CNS) and key peripheral organs show the therapeutic potential of PBGM01 for GM1.
Passage Bio has a strong background Passage Bio, inc is a clinical-stage genetic drug company dedicated to the development of conversion therapies for the treatment of rare single-gene central nervous system (CNS) diseases.
AAV Daniel, Dr.
James M.
Wilson is one of the co-founders of Passage Bio.
Dr.
Wilson is a professor at the University of Pennsylvania (hereinafter referred to as Penn) School of Medicine.
His team has made important contributions in the field of gene therapy for more than 20 years.
Has been at the forefront of AAV research.
▲James Wilson (picture source: xconomy) Tachi Yamada, who was the chief scientific officer of SmithKline-Beecham (now GlaxoSmithKline), is also one of the co-founders of Passage Bio.
Another Dr.
Stephen Squinto, who has more than 25 years of experience in the biotechnology industry and is also a venture partner of Aobo Capital, is also the co-founder of the company.
Passage Bio signed a research, cooperation and licensing agreement with the Gene Therapy Program (GTP) of the University of Pennsylvania (UPenn).
Penn was responsible for preclinical work, and then handed over the gene therapy to Passage for clinical testing and final commercialization.
Today, the biotechnology company is further deepening its cooperation with its academic partners.
On May 7, Passage Bio and GTP announced an expansion of the cooperation agreement to include five additional projects and the extension of Passage Bio's term to three years (to 2025) to implement new projects.
In addition, Passage Bio will provide funding for the discovery research of GTP, and under certain limited circumstances, will obtain exclusive rights to the technologies generated by the Passage Bio product discovery program developed with GTP, such as new capsids and technologies to reduce toxicity.
And delivery and formulation improvements.
In addition, in February 2019, Passage Bio received US$115 million in Series A financing led by Aobo Capital when it debuted as a cutting-edge gene therapy company; in September of the same year, it received US$110 million in Series B financing led by Access Biotechnology.
; In February 2020, Passage Bio went public on NASDAQ, set the issue price at a maximum of 18 US dollars, and received 216 million US dollars from the IPO.
In order to smoothly advance the clinical development of various candidate products, Passage Bio also signed a cooperation agreement with Paragon Gene Therapy on July 10, 2019.
A set of dedicated cGMP manufacturing facilities developed by Paragon is expected to be put into use in the second half of 2020.
Support Passage Bio's AAV gene therapy products from clinical to commercial supply. Recommended reading: AAV gene therapy has great potential, and the company founded by gene therapy pioneer James Wilson expands cooperation with Penn UniversityYi Mai Meng broke the news and set off again! AAV big cow James Wilson received US$115 million in Series A financing for the founding of a gene therapy company.
Neurological diseases may become a new trend for investment.
Yimai Meng broke the news that gene therapy pioneer James Wilson’s founding company received another US$110 million in financing.
Rare CNS diseases may be next A place of contention | Yimai Meng broke the news.
In recent years, gene therapy has shown amazing effects on genetic diseases, fulfilling people's expectations for it half a century ago.
It also allowed this emerging field that had suffered setbacks to enter the heyday of development worldwide.
In in vivo gene therapy, vector technology is a core part of the field.
The correct gene fragments are delivered to target cells through vectors to prevent these gene fragments from being degraded by the human body before they take effect.
Despite the rapid development of non-viral vector technology, viral vectors currently dominate the industry, among which rAAV is the most important viral vector.
Therefore, rAAV technology has also been favored by more researchers, and more and more rAAV production processes have emerged.
It is worth noting that rAAV will be directly injected into the human body in in vivo gene therapy, so the quality control of rAAV is very important, and reliable tools and methods must be used to characterize and analyze rAAV.
Medical Microphone Class and Waters Technology (Shanghai) Co.
, Ltd.
, a global professional measuring instrument company, will launch a live event on April 7 with the theme "LC and LC-MS technology in recombinant adeno-associated virus (rAAV) vectors and proteins The online live course of "Applications in Innovative Drug Research and Development", this course will have three experts bring a feast of knowledge to the audience.
Automated cell culture: Quantum liberates hands and redefines cell therapyMedClub broke the news April 3, 2021/eMedClub News/--April 1, 2021, Passage Bio announced the first case in the Imagine-1 study Infant GM1 ganglion patients have been successfully administered PBGM01.
At present, the patient has been taking the drug in the global phase 1/2 clinical trial plan of PBGM01 and has received PBGM01 treatment at the Children's Hospital of Saint Peter's University Hospital.
Dr.
Bruce Goldsmith, President and CEO of Passage Bio, said: "The company aims to develop treatments for rare central nervous system diseases.
We are excited for the first patient's drug delivery to promote the clinical development of PBGM01 and look forward to sharing clinical safety data and safety data at the end of the year.
Biomarker data.
"Recommended reading: An AAV gene therapy IND application founded by James Wilson, a company founded by AAV Daniel, has been approved by the US FDAYi Mai Meng reveals the mechanism of PBGM01 in the treatment of GM1.
Imagine-1 is a Phase 1/2 multinational study , Designed to test the safety, tolerability and effectiveness of PBGM01 in early-onset (type 1) and late-onset (type 2a) GM1 patients.
The study was confirmed by low β-gal enzyme activity and genetic testing.
GM1 gangliosidosis (GM1 gangliosidosis) is an autosomal recessive genetic disease caused by a lack of the lysosomal enzyme β-galactosidase (β-gal) encoded by the GLB1 gene mutation.
β-gal is an enzyme necessary for the degradation of GM1 ganglioside and keratan sulfate.
The decrease of β-gal level will cause the accumulation of the toxic level of GM1 ganglioside in the whole brain neurons, leading to the rapid development of the disease.
Infant GM1 is the most common and severe form of the disease, usually with abnormal gait at 4 months and degeneration at 6 months.
Babies with the disease experience rapid and fatal degeneration at approximately 2 years of age, with a life expectancy of 2-4 years.
PBGM01 is an AAV gene therapy under development for the treatment of infantile GM1.
PBGM01 is administered by ICM injection (injected into the cerebellar medulla oblongata at the craniocervical junction).
The AAVhu68 capsid is used to deliver the GLB1 gene encoding β-gal to the brain and surrounding tissues.
PBGM01 reduces the accumulation of GM1 gangliosides by increasing the level of β-gal.
PBGM01 has the potential to reverse neuronal toxicity, so that GM1 infants can resume their development.
In preclinical models, the wide distribution of PBGM01 in the brain and the wide absorption of β-gal in the central nervous system (CNS) and key peripheral organs show the therapeutic potential of PBGM01 for GM1.
Passage Bio has a strong background Passage Bio, inc is a clinical-stage genetic drug company dedicated to the development of conversion therapies for the treatment of rare single-gene central nervous system (CNS) diseases.
AAV Daniel, Dr.
James M.
Wilson is one of the co-founders of Passage Bio.
Dr.
Wilson is a professor at the University of Pennsylvania (hereinafter referred to as Penn) School of Medicine.
His team has made important contributions in the field of gene therapy for more than 20 years.
Has been at the forefront of AAV research.
▲James Wilson (picture source: xconomy) Tachi Yamada, who was the chief scientific officer of SmithKline-Beecham (now GlaxoSmithKline), is also one of the co-founders of Passage Bio.
Another Dr.
Stephen Squinto, who has more than 25 years of experience in the biotechnology industry and is also a venture partner of Aobo Capital, is also the co-founder of the company.
Passage Bio signed a research, cooperation and licensing agreement with the Gene Therapy Program (GTP) of the University of Pennsylvania (UPenn).
Penn was responsible for preclinical work, and then handed over the gene therapy to Passage for clinical testing and final commercialization.
Today, the biotechnology company is further deepening its cooperation with its academic partners.
On May 7, Passage Bio and GTP announced an expansion of the cooperation agreement to include five additional projects and the extension of Passage Bio's term to three years (to 2025) to implement new projects.
In addition, Passage Bio will provide funding for the discovery research of GTP, and under certain limited circumstances, will obtain exclusive rights to the technologies generated by the Passage Bio product discovery program developed with GTP, such as new capsids and technologies to reduce toxicity.
And delivery and formulation improvements.
In addition, in February 2019, Passage Bio received US$115 million in Series A financing led by Aobo Capital when it debuted as a cutting-edge gene therapy company; in September of the same year, it received US$110 million in Series B financing led by Access Biotechnology.
; In February 2020, Passage Bio went public on NASDAQ, set the issue price at a maximum of 18 US dollars, and received 216 million US dollars from the IPO.
In order to smoothly advance the clinical development of various candidate products, Passage Bio also signed a cooperation agreement with Paragon Gene Therapy on July 10, 2019.
A set of dedicated cGMP manufacturing facilities developed by Paragon is expected to be put into use in the second half of 2020.
Support Passage Bio's AAV gene therapy products from clinical to commercial supply. Recommended reading: AAV gene therapy has great potential, and the company founded by gene therapy pioneer James Wilson expands cooperation with Penn UniversityYi Mai Meng broke the news and set off again! AAV big cow James Wilson received US$115 million in Series A financing for the founding of a gene therapy company.
Neurological diseases may become a new trend for investment.
Yimai Meng broke the news that gene therapy pioneer James Wilson’s founding company received another US$110 million in financing.
Rare CNS diseases may be next A place of contention | Yimai Meng broke the news.
In recent years, gene therapy has shown amazing effects on genetic diseases, fulfilling people's expectations for it half a century ago.
It also allowed this emerging field that had suffered setbacks to enter the heyday of development worldwide.
In in vivo gene therapy, vector technology is a core part of the field.
The correct gene fragments are delivered to target cells through vectors to prevent these gene fragments from being degraded by the human body before they take effect.
Despite the rapid development of non-viral vector technology, viral vectors currently dominate the industry, among which rAAV is the most important viral vector.
Therefore, rAAV technology has also been favored by more researchers, and more and more rAAV production processes have emerged.
It is worth noting that rAAV will be directly injected into the human body in in vivo gene therapy, so the quality control of rAAV is very important, and reliable tools and methods must be used to characterize and analyze rAAV.
Medical Microphone Class and Waters Technology (Shanghai) Co.
, Ltd.
, a global professional measuring instrument company, will launch a live event on April 7 with the theme "LC and LC-MS technology in recombinant adeno-associated virus (rAAV) vectors and proteins The online live course of "Applications in Innovative Drug Research and Development", this course will have three experts bring a feast of knowledge to the audience.
