uniQure N.V. has announced that the initial participant has received a dose in the Phase I/II clinical study of AMT-162.

The application of AI in drug discovery can reduce the development timeline and increase the success rate of new drugs from 12% to 14%.

Three novel drugs by Gan & Lee Pharmaceuticals-GZR18, GZR4, and GZR101 injections-meet primary goals in Phase 2 trials.

Lumasiran works by specifically binding to HAO1 mRNA and promoting its degradation, thereby reducing the synthesis of GO enzyme.

Amgen reveals favorable Phase 3 results for Uplizna® (inebilizumab-cdon) in treating generalized myasthenia gravis at AANEM 2024.

Golodirsen is a phosphorodiamidate morpholino oligomer (PMO), a class of synthetic oligonucleotides designed for high stability and resistance to enzymatic degradation. 

In a Phase 2/3 study, nipocalimab effectively controls symptoms in teenagers with generalized myasthenia gravis.

Beacon Therapeutics Reports Favorable 24-Month Results from Phase 2 SKYLINE Study of AGTC-501 in X-Linked Retinitis Pigmentosa Patients.

Topas Therapeutics released encouraging top-line outcomes from a Phase 2a study examining their leading candidate, TPM502.

Circle Pharma Begins Phase 1 Clinical Trial with CID-078, an Oral Cyclin A/B RxL Inhibitor, Administered to Initial Patients for Advanced Solid Tumors.

Transgene updates on Phase II trial for TG4001 cancer vaccine targeting recurrent or metastatic HPV16-positive cervical and anogenital cancers.

GSK shares favorable phase III findings from ANCHOR trials on depemokimab for chronic rhinosinusitis with nasal polyps.