Zhang Feng's CRISPR gene-editing drug has been awarded the title of rare pediatric disease again, and is expected to cure β-thalassemia at one time

April 28, 2022 / eMedClub News/--On April 26, 2022, Editas Medicine, a leading genome editing company, announced that the U.
S.
Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its gene-editing drug EDIT-301 for the treatment of beta - Thalassemia
.

The FDA has previously granted Rare Pediatric Disease Designation to EDIT-301 for the treatment of sickle cell disease (SCD)
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The Rare Pediatric Disease designation is for serious, life-threatening diseases that primarily affect people 18 and younger and affect fewer than 200,000 people
.

The program aims to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases
.

If the Biologics License Application (BLA) for EDIT-301 is approved, Editas will be eligible to receive a Priority Review Voucher (PRV), which can be redeemed for priority review for any subsequent New Drug Application and can be sold or transferred
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 EDIT-301 for the treatment of β-thalassemia β-thalassemia is a common autosomal recessive disorder with an annual incidence of approximately 1 in 100,000 symptomatic individuals worldwide
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Beta-thalassemia reduces or disrupts beta-globin expression
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Insufficient β-globin production affects erythropoiesis, and the destruction of red blood cells leads to chronic hemolytic anemia and compensated extramedullary hematopoiesis
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 According to clinical severity and transfusion requirements, β-thalassemia can be divided into non-transfusion-dependent (NTDT) and transfusion-dependent β-thalassemia (TDT)
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TDT is the most severe form of beta-thalassemia, and patients require lifelong regular blood transfusions to prevent organ failure and death
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Long-term blood transfusion is prone to be complicated by iron overload leading to organ dysfunction and failure
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If left untreated, TDT patients have a high mortality rate, with a survival rate of only 15% at age 5 due to severe anemia
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 EDIT-301 is a cell therapy drug based on CRISPR/Cas12a (Cpf1) gene editing technology for the treatment of severe SCD and TDT
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EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells that edit the HBG1/2 promoter region in the β-globin locus via CRISPR/Cas12a (Cpf1) ribonucleoprotein (RNP)
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  ▲ Editing in the HBG1/2 promoter region (Image source: Editas official website) Studies have shown that red blood cells derived from edited CD34+ cells can continuously increase the production of fetal hemoglobin (HbF), which may be a source of severe SCD and TDT.
Patients are provided with a one-time, long-lasting treatment
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 EDIT-301 is underway in a clinical study (RUBY trial, NCT04853576) in patients with severe SCD
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Editas expects to initiate a Phase 1/2 study of EDIT-301 in TDT patients in 2022
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 Other β-thalassemia gene-editing drugs CRISPR Therapeutics and Vertex Pharmaceuticals jointly developed CTX001, an autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy, based on CRISPR/Cas9 cleavage of a BCL11A gene that inhibits HbF expression.
role
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It is worth mentioning that the Phase 1/2 clinical trial of CTX001 was approved in the EU for β-thalassemia and in the US FDA for SCD
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Recommended reading: The clinical trial results of CRISPR in the treatment of hereditary hemoglobinopathies are positive, and many companies are committed to the development of similar strategiesYimai Meng broke the news that Sangamo Therapeutics' ST-400 uses zinc finger protease (ZFN) gene editing technology to treat patients' own (autologous) ) produced by ex vivo gene editing of hematopoietic stem cells to generate functional progeny red blood cells by increasing fetal hemoglobin for the treatment of TDT
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Recommended reading: ASH2019: Sangamo Announces Phase 1/2 Clinical Data for β-Thalassemia, New Gene Editing Cell Therapy Worth Further Exploration Zi's ​​autologous CD34+ hematopoietic stem and progenitor cell injection is an autologous, in vitro gene editing cell therapy research product in clinical development for the treatment of transfusion-dependent β-thalassemia
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ET-01 is the first gene editing therapy and hematopoietic stem cell therapy research product approved by the State Food and Drug Administration for clinical trials in China
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On September 8, 2021, ET-01 has completed the initiation of the Phase 1 clinical trial and the first patient enrollment
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 Recommended reading: Boya Jiyin Gene Editing Therapy for β-Thalassemia Completes Phase I Clinical Trial and Enrollment of the First Patient -The safety and efficacy of thalassemia (thalassemia) clinical research has been carried out in Xiangya Hospital of Central South University and the 923rd Hospital of the PLA Joint Logistics Support Force
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Through multi-center clinical trials, the gene therapy provided by Bangyao Bio has cured 5 children with severe β-thalassemia, which further confirmed the feasibility and good efficacy of the technology
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At present, Bangyao Bio is making every effort to promote the process of new drug application, and strive to achieve the launch of new drugs as soon as possible, benefiting a wider population of thalassemia major patients
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Recommended reading: Leading the world! Bangyao Biologics Announces Preliminary Data of a Multi-Center Clinical Study on Gene Therapy for ThalassemiaYimai Meng broke the news that Refine Bio has developed a new type of precision editing drug targeting HBG1/2.
The two patients who completed treatment have been discharged successfully
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Clinical study data reflect the product's leading curative and safety advantages
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The two patients with transfusion-dependent thalassaemia were 9.
8 and 13.
7 years old, respectively, and both had the most severe β0/β0 genotype
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After receiving the treatment, the patients achieved the need to get rid of blood transfusion (hemoglobin concentration above 90 g/L) at 28 and 40 days after transplantation, respectively, and their platelets exceeded 100 G/L at 28 and 34 days after transplantation, respectively
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Recommended reading: The world's first clinical study of new target gene editing therapy! Refine Biotech opens a new chapter in gene therapy for thalassaemia.
CRISPR enables scientists to make very precise changes to DNA, thereby increasing new hope for the prevention and treatment of various diseases.
It is expected that this treatment strategy will benefit more patients as soon as possible! Reference: 1.
https:// -thalassemia/2.
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