Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
The U.S. FDA granted Abbvie Inc. accelerated approval for antibody-drug conjugate (ADC) Teliso-V (telisotuzumab vedotin), newly branded Emrelis, making it the first treatment for previously treated advanced non-small-cell lung cancer with high c-Met protein overexpression.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Beam, Hyundai, Regenxbio, Sarepta.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Abtherx, Affimed, AFT, Bayer, Endo, Galapagos, Hyloris, Intron, Leap, Mallinckrodt, Qliniq, Single Cell, Vaccinex.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Ascendis, Atea, Biorestorative, Cytodyn, Cytokinetics, Enterobiotix, Iteos, Karyopharm, Mediwound, Novo Nordisk, Roche.
U.S. Health and Human Services Secretary Robert Kennedy made his first appearance May 14 before the Senate Health, Education, Labor and Pensions (HELP) Committee since his January confirmation hearing. Chaos, testy arguments, accusations and surprising agreements ensued.
Apimeds Pharmaceuticals US Inc. (APUS), a subsidiary of Kospi-listed Inscobee Inc., said May 12 that it raised $13.5 million from its stock sale on the NYSE American exchange May 9 through an offering of 3.375 million shares at $4 per share. Hopewell, N.J.-based APUS is the second pharmaceutical company with Asian ties to float shares on the U.S. market this year.
Small interfering RNA (siRNA) gained another vote of confidence, albeit back end-loaded, from big pharma as privately held Adarx Pharmaceuticals Inc. signed a deal with Abbvie Inc. related to disease areas that include neuroscience, immunology, and oncology.
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