The Phase 1 clinical trial of ET-01, a gene-edited hematopoietic stem cell candidate for transfusion-dependent β thalassaemia, completed the last patient transfusion

On November 18, 2022, Boyaa Ginet announced that its multicenter Phase 1 clinical trial of ET-01, a gene-edited hematopoietic stem cell candidate for transfusion-dependent β thalassemia, has completed the last patient infusion
.
Boyaa Gene is a clinical-stage global biopharmaceutical company focused on gene editing technology translation, committed to developing transformative therapies
for unmet clinical needs.

"The completion of the eighth and final patient infusion in the ET-01 multicenter Phase 1 clinical trial is another important milestone in our development of a potential one-off cure for transfusion-dependent β thalassemia patients," said Dr.
Wei Dong, CEO of Boyaa Gein.
"We are very grateful to all the patients, families and investigators who participated in this clinical trial.
Their contributions have been instrumental
in advancing this Phase I clinical trial.
The results of this multicenter Phase I clinical trial will demonstrate the safety and efficacy of ET-01 in specific patient populations, and we plan to discuss the strategy and protocol of the Phase II clinical trial with regulatory agencies in 2023 so that ET-01 can benefit transfusion-dependent β thalassemia patients
in China as soon as possible.
" ”

β thalassemia is an inherited haemolytic anaemia disorder, and the current standard of care for transfusion-dependent β thalassemia is lifelong blood transfusion and iron removal
.
ET-01 is designed to achieve a one-time functional cure
with a single infusion of CRISPR/Cas9 genetically modified autologous hematopoietic stem progenitor cells.

This Phase I clinical trial is a multicenter, open-label clinical study to evaluate the safety and efficacy of a single dose of ET-01 in patients with transfusion-dependent β thalassaemia, with a total of 8 participants enrolled
.
Previously, in September 2021, the company announced that the principal investigator's institution completed the initiation of clinical trials and the enrollment of the first patient
.

About ET-01

ET-01, the CRISPR/Cas9 gene modified BCL11A erythroid enhancer's autologous CD34+ hematopoietic stem progenitor cell injection, is used for the treatment of transfusion-dependent β thalassemia
.
ET-01 is the first gene editing therapy and hematopoietic stem cell therapy product in China to be approved by the State Food and Drug Administration for clinical trials
.

About Boya Gein

Boya Gein is a global, clinical-stage biopharmaceutical company focused on gene editing technology translation, committed to developing transformative therapies
for unmet clinical needs.

Based on its scientific research and technical strength in gene editing technology, high-throughput genome editing screening and bioinformatics, the company promotes the development
of international leading in vitro and in vivo gene editing therapies.
The company has established a number of therapeutic platforms
, including in vitro therapy hematopoietic stem cell platform, in vivo therapy RNA base editing platform, in vitro therapy universal CAR-T platform, etc.

Founded in 2015, Boyaa Gein is headquartered in Beijing and has offices in Guangzhou, Shanghai and the greater Boston area
.