Ultragenyx's AAV9 gene therapy is the first treatment for Sanfilippo syndrome Type A. Full approval on 17 treated children against a natural history control now collides with a diagnostic window most families still miss.
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KRAS sat on the undruggable list for decades. Silexion's SIL204 is built to silence the mutation earlier in the cell, and the Phase 2/3 program is now moving into patients.
Solstice Oncology launched with a $225 million Series A led by RA Capital to advance Harbour BioMed-licensed porustobart into neoadjuvant MSS colon cancer.
Pharvaris reported Phase 3 CHAPTER-3 data for deucrictibant extended-release 40 mg, delivering an 83% attack reduction in hereditary angioedema and matching the efficacy of injectable biologics.