Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD), though the case of respiratory failure was determined by the investigator to be caused by busulfan myeloablative conditioning and not related to BEAM-101. The latter is an investigational therapy that produces base edits meant to alleviate the effects of SCD by mimicking genetic variants seen in people with hereditary persistence of fetal hemoglobin. Leerink analyst Mani Foroohar said in a report that news of the death “demonstrate[s] the challenges of autologous ex vivo transplant medicines.” Shares of the firm were trading at $23.48, down 88 cents.

Sana cuts programs, staff, as it refocuses on autoimmune assets

Extending its cash runway into 2026, Sana Biotechnology Inc. is prioritizing certain autoimmune assets and punting an oncology program and a central nervous system diseases program to a potential licensing partner or spinout company. The changes equate to a reduction in workforce for the company. Sana did not say how many jobs would be lost, but it last reported 328 employees, 251 of them in R&D, as of the end of 2023. While its cash, cash equivalents and marketable securities were $251.6 million as of the end of the second quarter, the company noted that the reduction in force could increase its 2024 operating cash burn above the prior guidance of less than $200 million. At the forefront of Sana’s upcoming efforts will be SC-291 for B-cell mediated autoimmune diseases, UP-421 for type 1 diabetes, and SC-262 for refractory blood cancers.

Ring Therapeutics expands Singapore footprint with new deals

Ring Therapeutics Inc. has joined forces with Singapore’s Agency for Science, Technology, and Research and with the Singapore Eye Research Institute to advance the first new class of viral vectors in more than 50 years, Ring CEO Tuyen Ong told BioWorld. He started Ring at the height of the COVID-19 pandemic four years ago, and the idea that “we all have viruses inside of us” intrigued him. Ring is harnessing commensal anelloviruses that have co-habited and co-evolved with humans over millions of years, yet have remained largely understudied and untapped.

Report: Drug regulators enabling forced labor in supply chain

Drug regulators around the world have a unique opportunity – and, in some cases, a legal mandate – to remove the taint of forced labor from the biopharma supply chain. But some of them, including the U.S. FDA and Japan’s PMDA, may be turning a blind eye to those responsibilities, according to a recent report from the nonprofit Centers for Advanced Defense Studies. In response to that report, Sen. Marco Rubio, R-Fla., is demanding that two China-based biopharma companies allegedly linked to slave labor, Xinjiang Nuziline Bio-Pharmaceutical Co. and SEL Biochem Xinjiang Co., be banned from importing active pharmaceutical ingredients and finished drugs into the U.S.

OSE’s IL-7 therapy hits mark in ulcerative colitis trial

OSE Therapeutics SA has reported positive data for lusvertikimab in a phase II trial in ulcerative colitis, boosting the monoclonal antibody’s prospects of becoming the first anti-interleukin-7 therapy to reach the market. The results “represent a major milestone in the clinical development of lusvertikimab and a strong catalyst for the subsequent steps,” said Nicolas Poirier, CEO of OSE.

Japan PMDA opens second overseas office in Washington

Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) set up its second overseas regulatory office in Washington, four months after the drug and med-tech regulator opened its first Asia base in Bangkok, Thailand, in July 2024. The latest U.S. expansion, according to the agency, will work to support the increasing number of innovative drugs being developed by overseas start-ups, especially in the U.S., and to accelerate the development of such drugs in Japan.

Also in the news

Adial, Alvotech, Ascendis, Atossa, Belite, Bristol Myers Squibb, Cadrenal, Cinfina, Conduit, Crossbridge, Disc Medicine, Electra, Huidagene, Journey Medical, Kadimastem, Kalvista, Lomond, Neurocrine, Neurogene, NLS, Nobelpharma, Novo Nordisk, Nurexone, OSE, Relief, Renexxion, Seaport, Surrozen, Tarsus, TCGFB, Teva, Tevogen, Viking, Y-Mabs