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rare diseases
Biotech
Kyverna pubs 1-year miv-cel data as CAR-T peers hit safety snags
Kyverna offered one-year updates for miv-cel in SPS and myasthenia gravis, showcasing sustained efficacy alongside an unblemished safety profile.
Angus Liu
Sep 24, 2026 6:02am
Beacon lights path to approval for vision loss gene therapy
Sep 21, 2026 2:00am
Fierce Pharma
Ionis scores ‘breakthrough’ rare disease approval
Sep 4, 2026 11:18am
Ultragenyx fails ph. 3 Angelman test as shares tumble
Sep 3, 2026 7:10am
Electra aims to light up Nasdaq with IPO for phase 2/3 I&I push
Aug 31, 2026 11:27am
Spruce’s successful FDA talks plant seeds for Q4 filing
Aug 24, 2026 9:57am
