Application of BRL-101 gene therapy of Bangyao Biotechnology in children with thalassemia who are free from blood transfusion dependence

On the 2nd anniversary of the follow-up, the attending doctor Fu Bin, deputy chief physician, took a group photo with "Xixi"

Gene therapy, one-time treatment can achieve a life-long curative effect,

Changes of red blood cells and total hemoglobin after transplantation

PBMCs，，

， CRISPR/Cas9 HSPC，β-，β- β0/β0

，，，

At present, Bangyao Bio has achieved good results in the clinical trial of the treatment of β0/β0 thalassaemia patients with its self-developed hematopoietic stem cell platform (ModiHSC ^® ) based on gene editing technology, and has successfully helped many cases nationwide Patients with β-thalassemia are free from transfusion dependence
.
It is worth mentioning that the outstanding achievements of Bangyao Biotechnology in the field of thalassemia have been selected into the treatment methods section of the second edition of the Blue Book on the Prevention and Control of Thalassaemia in China (2020); and on May 31, 2022, The clinical application of "BRL-101 autologous hematopoietic stem and progenitor cell injection" of Bangyao Bio has been accepted by the Center for Drug Evaluation (CDE) of the State Food and Drug Administration (acceptance number: CXSL2200236)
.
In the future, the age of the research subjects for this project will be further expanded to 35 years old, which is expected to benefit the "old and difficult group" in the field of thalassemia transplantation - the elderly and patients with no hope of matching
.

As a cell gene pharmaceutical company with the mission of "leading innovation with gene editing, developing breakthrough therapies, and benefiting all mankind", Bangyao Biotechnology has continuously overcome industry barriers and carried out a multi-target strategic layout.
Excellent clinical results have been achieved in projects such as thalassemia, non-viral PD1 site-specific integration of CAR-T, and UCART
.
In the future, we will continue to accelerate the transformation and implementation of innovative drugs to benefit patients with rare genetic diseases and malignant tumors around the world, including thalassemia!

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About Bangyao

 

 

Shanghai Bangyao Biotechnology Co.
, Ltd.
is committed to becoming the world's leading cell gene pharmaceutical company in the new commercial civilization era.
The "Shanghai Gene Editing and Cell Therapy Research Center" jointly established with universities has produced more than 100 patents in the past 5 years, 5 projects have carried out clinical trials initiated by researchers in 8 well-known hospitals, and many projects have entered the IND application stage
.
Among them, projects such as gene editing treatment for β-thalassemia, non-viral PD1 site-specific integration of CAR-T, and UCART have achieved excellent clinical results, leading the world, and have been published in well-known academic journals such as Nature, Nature Medicine, and Nature biotechnology.
Several academic papers
.
Bangyao Bio has built five technology platforms with independent intellectual property rights, including gene editing technology innovation platform, hematopoietic stem cell platform, non-virus fixed-point integrated CAR-T platform, universal cell platform, and enhanced T cell platform, and has a 7,000-square-meter GMP pilot test base.
And an operation team of nearly 200 people, which effectively guarantees the rapid transformation and application of innovative research results
.
Bangyao Biotech continuously promotes rapid update and iteration of R&D products through patient needs and clinical feedback
.
And uphold the attitude of openness, sharing, and win-win, together with the global innovative biopharmaceutical ecological chain enterprises, accelerate the transformation and implementation of innovative drugs, and benefit the global patients with genetic diseases and malignant tumors!