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Recent popular reports from Yimaike ★ Cinda Bio-PD-1/CD137 double antibody released positive preclinical data Yimai Meng broke the news ★ The next step for EU listing! New Coronary Antibody Therapeutics Receives Positive Opinion from CHMPMedMeng broke the news November 20, 2021/MedClub News/--Next-generation gene editing company Graphite Bio recently announced that its gene-edited autologous hematopoietic stem cell therapy GPH 101 The first patient has been included in a phase 1/2 clinical trial.
The drug is designed to correct the genetic mutation that causes sickle cell disease (Scd)
.
SCD is a serious, life-threatening genetic blood disease that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent single-gene disease in the world
.
SCD is caused by a mutation in the β-globin gene, which causes red blood cells to deform, resulting in an increased risk of anemia, blood flow obstruction, severe pain, stroke and organ damage, and a shortened life span of about 20-30 years
.
Despite progress in treatment and care, progressive organ damage continues to cause early death and severe morbidity, highlighting the need for therapeutic treatment
.
Graphite Bio is a new generation gene editing company focused on developing potential therapies for the treatment of serious diseases
.
Graphite's targeted gene integration platform utilizes the homology-directed repair (HDR) approach to effectively repair gene defect sites and accurately deliver gene fragments to achieve new specific cell functions
.
New DNA corrects defective genes or inserts new genes .
▲ Image source: Graphite Bio’s ongoing CEDAR is a phase 1/2, multi-center, open-label clinical trial that will test the safety, implantation success, gene correction rate, total hemoglobin, and drugs of GPH101 in patients with severe SCD Efficacy and other indicators are evaluated
.
The trial will enroll about 15 adult and adolescent participants at five clinical trial sites in the United States.
It is expected to treat the first GPH 101 patients in the first half of 2022 and provide preliminary proof-of-concept data by the end of 2022
.
"GPH 101 is the first research therapy to enter the clinical development stage.
It uses our next-generation gene editing platform technology to directly correct mutations in the β-globin gene that cause sickle cell disease
.
" Graphite Bio CEO Josh Lehrer Said, "Using our genetic correction method, we have demonstrated in preclinical studies the ability to reduce the production of harmful sickle hemoglobin and restore normal adult hemoglobin expression
.
This method has the potential to restore normal physiology and is considered a treatment The gold standard for sickle cell disease
.
We are very pleased that our first patient has now participated in our CEDAR clinical trial.
We look forward to evaluating the potential of GPH 101 because we will continue to urgently advance the development of GPH 101 with a view to Shape cell patients provide a new treatment option
.
"Gene editing is widely used in SCDCTX001, an investigational therapy for CRISPR editing in vitro, which uses CRISPR/Cas9 to introduce patients’ autologous hematopoietic stem cells through electroporation in vitro
.
The therapy is currently undergoing two phase 1/2 clinical studies, respectively for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD).
So far, 40 patients in the two studies have received CTX001 transfusion.
Note
.
Recommended reading: EHA 2021: The latest results of clinical trials of CRISPR and Vertex for the treatment of hereditary hemoglobinopathy are positive Yimai Meng broke the news that the candidate product of gene editing cell therapy EDIT-301 was awarded the title of Rare Pediatric Disease (RPD) by the US FDA, EDIT-301 It is being developed to treat sickle cell disease (SCD)
.
Recommended reading: The candidate therapy of CRISPR giant Editas has been awarded the title of rare pediatric disease by the FDA, and the gene therapy track for sickle cell disease has been opened.
CRISPR/Cas9 genome-edited hematopoietic stem cell (HSCs) therapy for the treatment of SCD
.
The therapy introduces the gene expressing fetal hemoglobin into the patient's hematopoietic stem cells, and is currently in phase 1/2 clinical trial research
.
CRISPR_SCD001 is a therapy developed by the University of California (San Francisco, Berkeley, and Los Angeles) to treat SCD.
The therapy uses CRISPR/Cas9-edited hematopoietic stem cells (called CRISPR_SCD001 drugs) for transplantation (HSCT)
.
In addition to hematopoietic stem cells, future gene editing technologies will also realize the use of other cell types, and possibly even gene editing treatments in the body
.
In addition, this new method may also go far beyond the application of gene editing in genetic diseases in the future, that is, it has broad therapeutic potential for non-hereditary diseases such as cancer, neurodegenerative diseases, and enzyme or metabolic defect diseases.
.
Reference materials: 1.
https://ir.
graphitebio.
com/press-releases/detail/60/graphite-bio-enrolls-first-patient-in-phase-12-clinical
The drug is designed to correct the genetic mutation that causes sickle cell disease (Scd)
.
SCD is a serious, life-threatening genetic blood disease that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent single-gene disease in the world
.
SCD is caused by a mutation in the β-globin gene, which causes red blood cells to deform, resulting in an increased risk of anemia, blood flow obstruction, severe pain, stroke and organ damage, and a shortened life span of about 20-30 years
.
Despite progress in treatment and care, progressive organ damage continues to cause early death and severe morbidity, highlighting the need for therapeutic treatment
.
Graphite Bio is a new generation gene editing company focused on developing potential therapies for the treatment of serious diseases
.
Graphite's targeted gene integration platform utilizes the homology-directed repair (HDR) approach to effectively repair gene defect sites and accurately deliver gene fragments to achieve new specific cell functions
.
New DNA corrects defective genes or inserts new genes .
▲ Image source: Graphite Bio’s ongoing CEDAR is a phase 1/2, multi-center, open-label clinical trial that will test the safety, implantation success, gene correction rate, total hemoglobin, and drugs of GPH101 in patients with severe SCD Efficacy and other indicators are evaluated
.
The trial will enroll about 15 adult and adolescent participants at five clinical trial sites in the United States.
It is expected to treat the first GPH 101 patients in the first half of 2022 and provide preliminary proof-of-concept data by the end of 2022
.
"GPH 101 is the first research therapy to enter the clinical development stage.
It uses our next-generation gene editing platform technology to directly correct mutations in the β-globin gene that cause sickle cell disease
.
" Graphite Bio CEO Josh Lehrer Said, "Using our genetic correction method, we have demonstrated in preclinical studies the ability to reduce the production of harmful sickle hemoglobin and restore normal adult hemoglobin expression
.
This method has the potential to restore normal physiology and is considered a treatment The gold standard for sickle cell disease
.
We are very pleased that our first patient has now participated in our CEDAR clinical trial.
We look forward to evaluating the potential of GPH 101 because we will continue to urgently advance the development of GPH 101 with a view to Shape cell patients provide a new treatment option
.
"Gene editing is widely used in SCDCTX001, an investigational therapy for CRISPR editing in vitro, which uses CRISPR/Cas9 to introduce patients’ autologous hematopoietic stem cells through electroporation in vitro
.
The therapy is currently undergoing two phase 1/2 clinical studies, respectively for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD).
So far, 40 patients in the two studies have received CTX001 transfusion.
Note
.
Recommended reading: EHA 2021: The latest results of clinical trials of CRISPR and Vertex for the treatment of hereditary hemoglobinopathy are positive Yimai Meng broke the news that the candidate product of gene editing cell therapy EDIT-301 was awarded the title of Rare Pediatric Disease (RPD) by the US FDA, EDIT-301 It is being developed to treat sickle cell disease (SCD)
.
Recommended reading: The candidate therapy of CRISPR giant Editas has been awarded the title of rare pediatric disease by the FDA, and the gene therapy track for sickle cell disease has been opened.
CRISPR/Cas9 genome-edited hematopoietic stem cell (HSCs) therapy for the treatment of SCD
.
The therapy introduces the gene expressing fetal hemoglobin into the patient's hematopoietic stem cells, and is currently in phase 1/2 clinical trial research
.
CRISPR_SCD001 is a therapy developed by the University of California (San Francisco, Berkeley, and Los Angeles) to treat SCD.
The therapy uses CRISPR/Cas9-edited hematopoietic stem cells (called CRISPR_SCD001 drugs) for transplantation (HSCT)
.
In addition to hematopoietic stem cells, future gene editing technologies will also realize the use of other cell types, and possibly even gene editing treatments in the body
.
In addition, this new method may also go far beyond the application of gene editing in genetic diseases in the future, that is, it has broad therapeutic potential for non-hereditary diseases such as cancer, neurodegenerative diseases, and enzyme or metabolic defect diseases.
.
Reference materials: 1.
https://ir.
graphitebio.
com/press-releases/detail/60/graphite-bio-enrolls-first-patient-in-phase-12-clinical
