Clinical Trials Arena January 14, 2026
Full pipelines and rising revenues lead rare disease biotechs to restyle themselves as more mature, global biopharma companies.
Developers in rare disease emphasise cashflow and R&D expansion as evidence of their transition from emerging biotechs to established companies after strong performances in 2025.
Several rare disease biotechs hailed commercial successes in 2025 as marking a turning point in the sector’s maturation, despite lingering reliance on single lead assets and pressure to cut staff to remain financially stable. Even so, developers promised deepening pipelines and regulatory advancement in 2026 to attendees at the 2026 J.P. Morgan Healthcare conference in San Francisco, taking place from 12 to 15 January.
2025 was a “breakout year” for siRNA biotech Alnylam Pharmaceuticals according to CEO...
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