Latest News

  • MARKET TRENDS

    14 Dec 2025

    Rare Disease Boom Redefines Pharma Strategy

  • INNOVATION

    11 Dec 2025

    An Experimental Gene Therapy Signals New Hope for Rare Disorders

  • INVESTMENT

    9 Dec 2025

    High Risk, High Impact: Rare Disease Biotech Reclaims Investor Attention

  • TECHNOLOGY

    8 Dec 2025

    AI Models Take on Medicine’s Rarest Challenges

BioCryst Bets on Convenience in High-Stakes HAE Deal

BioCryst Bets on Convenience in High-Stakes HAE Deal

PARTNERSHIPS

4 Dec 2025

Pending Astria acquisition centres on long-acting therapy as drugmakers seek more convenient treatments for rare diseases

Why Lilly’s Latest Alliance Matters for Rare Disease Patients

PARTNERSHIPS

11 Nov 2025

Why Lilly’s Latest Alliance Matters for Rare Disease Patients

Eli Lilly’s pact with MeiraGTx around an eye gene therapy points to renewed confidence in rare disease research

BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

INSIGHTS

30 Oct 2025

BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

BridgeBio leans on its ATTR-CM launch to push late-stage assets while facing competition, pricing pressure, and questions about demand dur...

From Niche to Notable: Rare Diseases Reshape Biotech

RESEARCH

20 Sep 2025

From Niche to Notable: Rare Diseases Reshape Biotech

Approval of elamipretide and Mirum Pharma deal point to growing investor interest in high-need therapies with small patient populations

Inside the Push to Personalize RNA Medicine

INNOVATION

1 Feb 2025

Inside the Push to Personalize RNA Medicine

Tailored antisense drugs move from concept to clinic as researchers and companies test faster models for ultra-rare conditions

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