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1250 articles, sorted by publication time.
DOC1021 uses a patient’s own dendritic cells to carry both tumor lysate and tumor RNA, seeking to rekindle an immune attack after anti-PD-1 treatment has failed; at this stage, the first questions this Phase 1/2 trial must still answer are safety and feasibility.
Oral levosimendan failed to meet the primary efficacy endpoint in the LEVEL trial, setting back a PH-HFpEF therapy that had been approaching the threshold for registration and once again highlighting the difficulty of treating this type of disease at the cardiopulmonary interface.
SION-719 failed to further reduce sweat chloride, dealing a clinical setback to the concept of directly stabilizing the NBD1 structure of the CFTR protein; the company will discontinue this add-on treatment path and reassess its allocation of capital.
The FDA did not question ITM-11’s clinical or safety data, but manufacturing and inspection issues at a third-party commercial facility were still enough to block approval, highlighting that competition in radiopharmaceuticals depends not only on efficacy but also on whether supply chains can consistently meet regulatory requirements.
Skylark Bio is expanding inner-ear gene therapy from rare OTOF deficiency to the more common GJB2-related hearing loss; the first participant has been dosed, but efficacy, durability, and surgical risks remain to be answered by a small early-stage trial.
BL-M14D1 will be combined with immunotherapy and directly compared with the current platinum-based chemotherapy regimen; the 550-patient trial will use time to disease progression and survival as primary endpoints, beginning the test of whether early efficacy signals can reshape first-line treatment.
The clinical research services provider, which focuses on small and midsize biotech companies, said oncology trials drove second-quarter bookings, while proposal demand continued to increase. Nearly $2 billion in backlog provides near-term visibility but does not eliminate risks from industry financing conditions and project changes.
The research team trained a multimodal model using the long-term health records and chest X-rays of 3.67 million people, demonstrating its ability to identify cancer risk in retrospective analyses and external data; however, population differences, screening bias, and clinical benefit still need to be addressed by large prospective trials.
The HOPE-3 trial showed that quarterly deramiocel infusions reduced the extent of upper-limb function decline by 54% in patients with advanced disease and produced signals of improvement in cardiac measures; however, one year of data is not yet sufficient to answer questions about long-term benefits and risks.
HM17321 was optimized with the assistance of a protein-sequence prediction platform and is undergoing a Phase 1 dose-escalation trial in the United States; whether the body-composition improvements seen in animal studies can be replicated in humans remains the central unanswered question.
The FDA has cleared the investigational new drug application for UGN-501. UroGen expects to initiate a Phase 1 study in the fourth quarter of 2026 to test the safety and feasibility of locally instilling an oncolytic virus; evidence currently remains at the preclinical stage.
This cash-and-stock transaction targets not a new drug itself, but the manufacturing infrastructure that maintains the quality of living cells; if completed successfully, Repligen will add BioLife’s preservation media and cell-processing tools to its existing bioprocessing portfolio.