Vor Bio, a clinical-stage cell and genome engineering company, has reported progress in its clinical trials for the treatment of acute myeloid leukemia (AML). The company announced that it has made significant advancements in its vision of creating a Treatment System for AML that could potentially deliver more potent and durable responses in the post-transplant setting without on-target toxicity.
Dr. Robert Ang, President and CEO of Vor Bio, expressed his satisfaction with the progress made during the quarter. He highlighted the recent clearance of the Investigational New Drug (IND) application for VCAR33 as a significant step forward. This milestone opens up new possibilities for the development of a Treatment System for AML, aiming to improve patient outcomes and quality of life.
Vor Bio’s ongoing clinical trial, known as VBP101 or Trem-cel, continues to actively enroll patients. The company expects to provide further updates on engraftment and Mylotarg hematologic protection data from this study by the end of the year. Trem-cel is a genome-edited allogeneic hematopoietic cell transplant (HCT) designed to replace standard of care transplants for AML patients and potentially other blood cancers. Its unique composition, lacking the CD33 protein, enables targeted therapies in the post-transplant setting, including the use of Mylotarg and CD33-targeted CAR-Ts.
In addition to its clinical progress, Vor Bio has achieved key milestones in its manufacturing capabilities. The company is ready to initiate in-house clinical manufacturing of VCAR33, with the completion of the first engineering runs in preparation for Trem-cel tech transfer. Furthermore, Vor Bio has secured a worldwide non-exclusive license from Editas Medicine for ex-vivo Cas9 gene-edited hematopoietic stem cells (HSCs) therapies for the treatment and/or prevention of hematological malignancies. This license provides access to crucial intellectual property and supports the continued development and commercialization of edited HSCs, including Trem-cel (VOR33) and potential future product candidates.
Vor Bio’s dedication to advancing the standard of care for patients with blood cancers stems from its focus on engineering hematopoietic stem cells to enable targeted therapies post-transplant. The company aims to deliver innovative solutions that may positively impact patient outcomes and disease progression.
While Vor Bio continues to make strides in its clinical trials and manufacturing capabilities, it remains committed to transparency and sharing updates with the medical community. By adhering to rigorous clinical development processes and actively engaging in partnerships and licensing agreements, Vor Bio is positioning itself as a leader in the field of cell and genome engineering.
It is important to note that forward-looking statements have been made in this press release. Vor Bio may not achieve the plans, intentions, or expectations disclosed in these statements due to various factors. These factors include uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, availability and timing of results, expectations for regulatory approvals, manufacturing capabilities, and funding requirements.
About Vor Bio:
Vor Bio is a clinical-stage cell and genome engineering company dedicated to changing the standard of care for patients with blood cancers. By focusing on manipulating hematopoietic stem cells, Vor Bio aims to enable targeted therapies post-transplant, potentially altering the course of disease and improving patient quality of life. For more information, please visit www.vorbio.com.
