Design Therapeutics, Inc. announced the initiation of a Phase 1 clinical trial for its gene-targeted treatment DT-216P2 in healthy volunteers, with patient dosing for Friedreich Ataxia (FA) set to ...

The collaboration marks a turning point in AI-driven drug design and trial forecasting, de-risking the drug development ...

Nine-month findings show mean improvement in Composite Unified Huntington's Disease Rating Scale from baseline of +0.64 ...

Recent Trends and Patient Outcomes of Phase I Trials: A Single-Institution Experience in the Era of New Therapeutic Agents This study reviewed all industry-sponsored phase I clinical trial protocols ...

Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with ...

First patient in Cohort four has been treated with TTX-MC138 Ten patients have been treated with TTX-MC138 at escalating dose levels Additional patients being evaluated for eligibility for expanded ...

Cohort 4 data demonstrated positive safety and efficacy results, including additional clinical benefit observed following ...

John Mascarenhas, MD, discusses the rationale, mechanism, and design of the phase 1 INCA33989-101 and -102 trials investigating a CAL-R–targeted antibody for patients with essential thrombocythemia.

Clinical trials are studies that test new medical interventions, such as a new type of drug, surgery, or medical device, on human volunteers. They aim to determine whether the new treatment option is ...

The approval of this accelerated pathway not only demonstrates the FDA’s full recognition of the quality, safety, and efficacy of the Phase 1 clinical data of hNPC01 in China, but also will ...