In a shareholder letter released on 1 April 2025, MAIA Biotechnology revealed plans to advance telomere‐targeted therapies in …
In a shareholder letter released on 1 April 2025, MAIA Biotechnology revealed plans to advance telomere‐targeted therapies in …
Alnylam’s Fitusiran gains FDA approval as a first RNAi therapeutic for hemophilia, while Vertex discontinues its innovative insulin-producing …
The FDA approval of Alnylam’s Fitusiran marks a milestone in RNA interference therapeutics, while Vertex discontinues a novel …
Alnylam Pharmaceuticals secures FDA clearance for vutrisiran, marking a significant milestone in RNA interference therapeutics for a rare …
Novartis receives a landmark approval for iptacopan (Fabhalta®), offering new hope for patients with a rare kidney disorder.
Precision BioSciences receives IND clearance to launch US-based trial of PBGENE-HBV, a meganuclease gene-editing candidate for a functional …
European regulatory reforms are accelerating approvals for novel rare disease treatments, attracting global investment and expanding patient access.
The FDA has approved Merilog (insulin-aspart-szjj), marking the first biosimilar to Novolog (insulin aspart), and widening affordable insulin …
The FDA has cleared vimseltinib (Romvimza), a first-in-class CSF1R inhibitor, for symptomatic tenosynovial giant cell tumor—marking a breakthrough …
The FDA has approved SPN-830 (Onapgo)—a novel subcutaneous apomorphine infusion device—offering continuous, effective management of motor fluctuations in …
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