Beam Therapeutics Reports Patient Death in BEAM-101 Sickle Cell Disease Trial
Key Insights
Beam Therapeutics reported a patient death in their Phase I/II trial of BEAM-101 for sickle cell disease (search), attributed to busulfan conditioning, not the therapy.
Sana Biotechnology is refocusing on autoimmune assets, leading to workforce reductions and program cuts to extend its cash runway into 2026.
OSE Therapeutics (search) announced positive Phase II data for lusvertikimab in ulcerative colitis (search), marking a significant step for the anti-IL-7 therapy.
Beam Therapeutics Inc. has disclosed a patient death during the Phase I/II clinical trial of BEAM-101, a novel base editing therapy for sickle cell disease (search) (SCD). The patient's death, resulting from respiratory failure, was determined by the trial investigator to be caused by busulfan myeloablative conditioning, a common pre-transplant treatment, and not directly related to BEAM-101. BEAM-101 aims to alleviate the effects of SCD by mimicking genetic variants that lead to hereditary persistence of fetal hemoglobin.
Sana Biotechnology Restructures to Focus on Autoimmune Programs
Sana Biotechnology Inc. is prioritizing its autoimmune assets, including SC-291 for B-cell mediated autoimmune diseases (search) and UP-421 for type 1 diabetes. This strategic shift involves discontinuing an oncology program and a central nervous system diseases program, potentially through licensing or a spinout. The restructuring includes a workforce reduction, though the exact number of affected employees was not disclosed. Sana's cash, cash equivalents, and marketable securities totaled $251.6 million as of the end of the second quarter. The company anticipates that the reduction in force may increase its 2024 operating cash burn above the prior guidance of less than $200 million.
Ring Therapeutics Expands Viral Vector Research in Singapore
Ring Therapeutics (search) Inc. is collaborating with Singapore’s Agency for Science, Technology, and Research and the Singapore Eye Research Institute to develop a new class of viral vectors. Ring is focusing on commensal anelloviruses, which have co-evolved with humans. The company aims to harness these viruses for therapeutic applications.
OSE Therapeutics' Lusvertikimab Shows Promise in Ulcerative Colitis
OSE Therapeutics (search) SA announced positive results from a Phase II trial of lusvertikimab in ulcerative colitis (search). Lusvertikimab, a monoclonal antibody, targets interleukin-7 (IL-7) and could potentially be the first anti-IL-7 therapy to reach the market. According to Nicolas Poirier, CEO of OSE, these results represent a major milestone in the clinical development of lusvertikimab.
Japan PMDA Opens Washington Office
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has established a second overseas regulatory office in Washington, D.C., following the opening of its first Asian base in Bangkok, Thailand. The U.S. expansion aims to support the increasing number of innovative drugs being developed by overseas start-ups, particularly in the U.S., and to accelerate their development in Japan.