Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day. Read More
Ascletis Pharma Inc.’s once-daily oral fatty acid synthase inhibitor, denifanstat, demonstrated statistically significant and clinically meaningful improvements compared to placebo, meeting all primary and secondary endpoints in a phase III trial for moderate to severe acne vulgaris. Read More
The EMA has issued a new guideline on how to include and/or retain pregnant and breastfeeding women in clinical trials, in a move that it says “marks a change in the paradigm.” The aim is to ensure that trial sponsors generate robust clinical data in these populations. Read More
Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added. Read More
A publication based on longitudinal and cross sectional data and led by researchers at the U.S. NIH’s National Institute on Aging published on June 5, 2025 in Science has stated that the impact of taurine supplementation at delaying aging or treating aging-related conditions is context-dependent, and that the circulating levels of taurine are impacted by factors unique to each individual rather than declining with age. To qualify taurine as a true marker of aging, it should change with age across diverse populations over time and ideally supported by longitudinal data. Read More
Turning the human body into a biofactory of precision protein therapeutics is the focus of newly launched Signify Bio, which emerged with an oversubscribed $15 million initial financing to advance three platforms with broad potential across therapeutic areas. Read More
Ligachem Biosciences Inc. signed two license agreements with Novarock Biotherapeutics Inc., a subsidiary of CSPC Pharmaceutical Group Ltd., to bring in two of Novarock’s antibodies and create antibody-drug conjugate (ADC) candidates with novel cancer targets. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Anaptysbio, Ascletis, Atyr, Corvus, Halo, Immix, Innovent, Insilico, J&J, Moleculin, Sionna, Tolremo, Vasa. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alvotech, Bayer, Camurus, Eli Lilly, Vividion, Xbrane. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Bayer, Cellectar, Hyloris, Oncovita, Roche, Sarepta, SFL Pharmaceuticals Deutschland Takeda. Read More
