Shanghai-based BRL Medicine Inc. has announced a significant breakthrough in the treatment of thalassemia major. The company reported the first cure in a multi-center Phase clinical study assessing the safety and efficacy of its BRL-101, an autologous hematopoietic stem progenitor cell therapy. This marks a major milestone in the development of gene therapies for inherited hemolytic disorders.
Previous Trial Successes
BRL’s gene editing therapeutic product, BRL-101, demonstrated excellent clinical effects in a previous investigator-initiated trial (ITT). The therapy has successfully cured six patients with beta-thalassemia worldwide, who have been free from dependence on blood transfusions for over one year. This success highlights the potential of BRL-101 to provide long-term solutions for patients suffering from this debilitating condition.
BRL-101 and the ModiHSC Platform
BRL-101 is a gene therapy developed through BRL’s in-house developed hematopoietic stem cell platform, ModiHSC. The therapy is primarily designed to treat beta-thalassemia, an inherited hemolytic disorder and one of the most common monogenic disorders. BRL-101 obtained clinical trial approval in China in August 2022, and a multi-center Phase I/II trial was initiated in October. The ModiHSC platform enables the development of therapies that provide lifelong cures after a single administration, according to the company.
Innovation and Pipeline
BRL Medicine offers a range of gene editing technology innovations, including the ModiHSC platform, a non-virus fixed-point integration CAR-T platform, a universal cell platform, and an enhanced T-cell platform, all with intellectual property rights. The company currently has five programs undergoing investigator-initiated trials, two with formal clinical trial approvals, and multiple others ready for investigational new drug (IND) filings. This robust pipeline positions BRL Medicine at the forefront of gene therapy innovation.-Fineline Info & Tech
