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1250 articles, sorted by publication time.
Tudriqev combined with nivolumab adds an option for patients whose disease continues to progress after anti-PD-1 therapy; however, the approval is based on an uncontrolled trial, leaving attribution of efficacy and subsequent confirmation as key tests.
Royalty Pharma takes over milestone payments and most sales royalties, while Zealand converts a non-core asset into cash; the deal price also reflects the market’s willingness to assume risk before the regulatory outcome is known.
This cell-based detection workflow achieved 97.6% sensitivity in benchmark testing with two guide RNAs and was extended to high-fidelity Cas9 and base editors; however, candidate sites still require separate sequencing confirmation, and standardized safety testing remains some way off.
By tracking the fate of editors across more than 200 million cells, researchers identified six restriction factors, including GJB2 and BET1L. Temporarily reducing two of them in retinal cells derived from patients with inherited blindness significantly increased base editing and restored some ion channel function.
ENDURANCE will focus on patients with earlier-stage amyotrophic lateral sclerosis and evaluate the autologous cell therapy based on functional changes over 24 weeks. However, public registries still show that enrollment has not begun, while a funding shortfall leaves the launch timeline highly uncertain.
Insilico Medicine says ISM0900 reduced lipoprotein(a) by up to 83.9% in preclinical studies and demonstrated oral pharmacokinetics. The candidate offers another possibility in a field dominated by injectable therapies, but all data still await independent review and validation in human studies.
This synthetic implant aims both to support the damaged joint surface and guide cartilage repair; however, with only five participants, the trial’s primary task is to confirm device safety over one year, not to demonstrate efficacy.
U.S. regulators believe the existing evidence is sufficient to manage the risks of blue-light cystoscopy through special controls. If the proposal is finalized, new devices could reach the market more quickly, but this does not mean relaxing diagnostic performance and safety requirements.
Poolbeg Pharma has added another layer to its intellectual property portfolio for its p38 MAPK inhibition strategy; however, the patent nearing grant protects use claims and does not yet demonstrate that the candidate drug can safely prevent CRS in patients.
CSB-001 will move from a once-targeted indication into a pivotal trial involving about 400 people; new funding and a commercial team are in place, but key efficacy data have yet to undergo public scrutiny.
This oral drug candidate does not primarily seek to suppress immune responses, instead targeting PAD2, which is associated with myelin damage; however, its efficacy in patients remains unknown.
Made from a patient’s blood and tumor material, DOC1021 seeks to reawaken T cells to attack cancer cells; this Phase 1/2 trial has expanded to HonorHealth, but its safety and efficacy still await answers from human data.