Yesterday Ultragenyx Pharmaceutical provided a Phase I/II update for its investigational antisense oligonucleotide GTX-102, demonstrating substantial symptomatic improvement in patients with the rare genetic disorder Angelman syndrome.
Tag Archive for: Phase I/II study
Preliminary Phase I/II data demonstrated that Merck and Kelun-Biotech’s TROP2-directed antibody-drug conjugate SKB264 can elicit promising disease control and even potentially extend survival among heavily pretreated patients with gastric or gastroesophageal junction cancer, according to an abstract presentation Tuesday at the 2024 American Association for Cancer Research annual meeting.
Roivant Sciences announced Tuesday during its third-quarter financial report that it will discontinue the development of its investigational SF3B1 modulator RVT-2001, following an interim analysis of a Phase I/II study in myelodysplastic syndromes.
The FDA put 4D Molecular Therapeutics’ gene therapy program, 4D-310, on clinical hold, the company announced February 2.