Alto met its enrollment goal with 83 patients across 13 US clinical sites
Achondroplasia is a genetic condition driven by FGFR3 that affects more than stature alone
PiNACLE is a head-to-head study comparing its next-generation CAR T-cell therapy against established therapies liso-cel and axi-cel in patients with relapsed or refractory large B-cell lymphoma
PBGENE-DMD is designed to permanently correct mutations in the dystrophin gene between exons 45 and 55, the “hot-spot” region affecting roughly 60% of DMD patients
The milestone means more than 1,000 men are now enrolled across both Phase 3 programs
N17350 is the company’s first-in-class tumor-directed therapeutic, designed to harness the ELANE pathway
The study tested whether PF’3944 could maintain efficacy when switching from weekly to monthly injections and remain safe and well-tolerated
The trials, which enrolled over 1,000 patients across 398 sites in 17 countries, showed that the drug was safe and well-tolerated—but ultimately failed to deliver the hoped-for clinical results
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