EHA 2021: The latest results of clinical trials of CRISPR and Vertex for the treatment of hereditary hemoglobinopathy are positive

Recent popular reports from Yimaike ★ Invitation to the event2021 Oncolytic Virus Drug Development Forum is about to open in Shanghai ★ The financing has been hundreds of millions in half a year, and the oncolytic virus has entered the fast laneYimai New Observation Click on the picture and register now June 16, 2021 /MedClub News/--A few days ago, CRISPR Therapeutics and Vertex Pharmaceuticals announced the Phase 1/2 clinical trial data of the autologous hematopoietic stem cell therapy CTX001 based on CRISPR gene editing at the 2021 European Hematology Association (EHA) annual meeting
.

CTX001 is an investigational therapy for in vitro editing of CRISPR.
It uses CRISPR/Cas9 to introduce patients’ autologous hematopoietic stem cells through electroporation in vitro, and cuts a BCL11A gene that can inhibit the expression of fetal hemoglobin (HbF), so that hematopoietic stem cells can produce high levels of content.
Red blood cells with HbF levels replace adult hemoglobin to provide oxygen to the body
.

HbF is a hemoglobin that can carry a lot of oxygen.
It is usually expressed at birth and is replaced with adult hemoglobin (HbA) in adulthood
.

Recommended reading: EHA 2020: The results of clinical trials of CRISPR in the treatment of hereditary hemoglobinopathy are positive, and there will be a number of CRISPR therapies to be promoted in clinical practice this yearMedical Maimen broke the news.
At present, CTX001 has been granted RMAT and fast track for TDT and SCD by the US FDA , Orphan drug and rare pediatric disease designation; and obtained PRIME certification granted by the European Medicines Agency (EMA) for the treatment of TDT and SCD, as well as orphan drug designation by the European Commission
.

CTX001 is currently undergoing two phase 1/2 clinical studies for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD).
So far, 40 patients in the two studies have received CTX001 infusion
.

The data released this time involved 22 patients, including 15 TDT patients and 7 SCD patients.
The follow-up time was at least 3 months (range: 4-26 months).
The study showed consistent effectiveness and sustained response
.

All 15 TDT patients, including 6 patients with β0/β0 or other severe genotypes, did not require blood transfusion at the last follow-up; 7 patients with severe SCD received CTX001 infusion, but did not have blood vessels at the last follow-up Obstructive crisis (VOC)
.

Dr.
Reshma Kewalramani, Chief Executive Officer and President of Vertex, said: “The 22 patient data provided this time are impressive in terms of the consistency and durability of the efficacy.
It also provides more evidence that CTX001 is likely to provide TDT and SCD patients.
One-time functional cure
.

" The press release pointed out that CTX001 is the leading clinical pipeline among gene editing methods for research/evaluation of TDT and SCD
.

 For more specific data, scan the QR code to read the complete reference materials: 1.
http:// -data-in-22-patients-with-greater-than-3-months-follow-up-post-treatment-with-investigational-crispr-cas9-gene-editing-therapy-ctx001-at-european-hematology-association -an Yimike has always been committed to original news reports such as cutting-edge technology, industry trends, and industry insights in bio-innovative drugs.
The high-end matrix users of all media reached 160,000+, of which industrial users accounted for more than 50%, and scientific research and clinical users accounted for about 30 %, more than 5% of users of investment institutions
.

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
.