Be Biopharma, a clinical-stage company based in Cambridge, Massachusetts, has announced its participation in the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, scheduled for May 13-17, 2025. The company will showcase its innovative research through two key presentations: one oral and one poster session. These presentations focus on the potential of Engineered B Cell Medicines (BCMs) for treating genetic diseases and improving patient outcomes. The oral presentation, titled "Ex Vivo Gene Editing of Autologous B Cells Produces Sustained Levels of Tissue Nonspecific Alkaline Phosphatase In Vivo for the Potential Treatment of Hypophosphatasia," will be delivered by Hanlan Liu, Ph.D., MBA, Senior Vice President, Head of Late Research and Non-Clinical Development at Be Biopharma, on May 17, 2025, from 10:45 to 11:00 AM CT. The presentation, part of the B-Cell and Solid Organ Therapies session, will unveil nonclinical data for the company's BE-102 program. Hypophosphatasia (HPP) is a rare genetic disorder characterized by low levels of tissue-nonspecific alkaline phosphatase (TNSALP) due to mutations in the ALPL gene. This deficiency results in multi-systemic issues, primarily affecting bone mineralization. Currently, the only approved treatment is enzyme replacement therapy (ERT), which requires frequent injections throughout a patient's lifetime and is only effective for certain age groups, often leading to significant side effects and reduced quality of life. To address these limitations, Be Biopharma's BE-102 program involves isolating and activating primary human B cells, then using CRISPR/Cas9 to precisely insert the human ALPL gene into the CCR5 locus, a safe harbor location in the genome. The resulting engineered B cells, named BE-102, secrete active TNSALP, which helps break down inorganic pyrophosphate (PPi), a substance that accumulates and causes problems in individuals with HPP. In vitro tests have shown that BE-102 effectively restores TNSALP activity and corrects metabolic imbalances. Furthermore, in vivo studies conducted on immune-deficient NOG-hIL6 mice demonstrated long-term engraftment and continuous production of TNSALP after a single intravenous injection. This nonclinical proof-of-concept suggests that BE-102 could offer a durable, potentially disease-modifying therapy for HPP, with the added advantage of being titratable and redosable as needed. Additionally, Xuqing Zhang, Ph.D., Director of Immunology at Be Biopharma, will present a poster titled "Exploration of Allogeneic Shielding Strategies by Precise CRISPR/Cas9 Genome Engineering of Primary Human B Cells to Enable Off-the-shelf B Cell Medicines for Sustained Delivery of in vivo Biologics" on May 13, 2025, during the Tuesday Poster Reception from 6:00 to 7:30 PM CT. This poster will highlight the company's efforts to develop allogeneic, or universal donor, BCMs. Allogeneic BCMs can be administered to any patient without the need for individual genetic matching, making them a promising solution for broadening access to cell therapies. The research explores shielding strategies to reduce the risk of immune rejection, which is a critical challenge in allogeneic cell therapies. Using precise CRISPR/Cas9 genome editing, Be Biopharma has successfully engineered B cells to be more immunologically stealthy, showing that they can persist in the body and deliver therapeutic proteins without triggering a strong immune response. Engineered B Cell Medicines represent a breakthrough in cellular therapy, leveraging the unique capabilities of B cells to produce therapeutic proteins over extended periods. Precision genome editing allows for the insertion of specific genes into B cells, enabling them to become potent manufacturers of biologics. This innovative approach promises to be durable, allogeneic, and easy to administer, potentially transforming the treatment landscape for various genetic diseases, cancers, and other serious conditions. Be Biopharma, founded in October 2020, is at the forefront of this new field. Backed by prominent investors such as ARCH Venture Partners, Atlas Venture, and RA Capital Management, the company aims to dramatically improve the lives of patients with conditions like Hemophilia B and HPP. The team consists of dedicated scientists, technologists, manufacturing experts, and business leaders who collaborate to create advanced cell therapies that can be finely tuned and repeated as necessary. Industry insiders have praised Be Biopharma's work, noting the significant potential of BCMs to revolutionize current treatment paradigms. Dr. David M. Livingston, a senior researcher in the field, stated, "The development of BE-102 and the exploration of allogeneic shielding strategies by Be Biopharma are groundbreaking steps in gene and cell therapy. These innovations could not only provide more effective treatments but also expand access to cellular therapies to a wider population." Be Biopharma continues to push the boundaries of what is possible in cellular medicine, aiming to bring much-needed solutions to patients facing challenging medical conditions. For more information, visit Be.Bio and their LinkedIn page.