Latest News

  • INNOVATION

    11 Feb 2026

    Can Nanomedicine Rescue Stalled Rare Drugs?

  • MARKET TRENDS

    5 Feb 2026

    Rare Disease Drug Boom Sparks Dealmaking and Rivalry

  • PARTNERSHIPS

    3 Feb 2026

    Why Scale Is Suddenly Everything in Rare Disease Drugs

  • MARKET TRENDS

    14 Dec 2025

    Rare Disease Boom Redefines Pharma Strategy

An Experimental Gene Therapy Signals New Hope for Rare Disorders

DNA strand being edited to represent experimental rare disease gene therapy

INNOVATION

11 Dec 2025

A custom SLC6A1 gene therapy and Mirum’s Bluejay deal point to early, cautious momentum in rare disease innovation

AI generated medical model illustrating rare disease drug discovery

TECHNOLOGY

8 Dec 2025

AI Models Take on Medicine’s Rarest Challenges

AI is speeding up rare disease research and reshaping drug discovery worldwide

BioCryst Pharmaceuticals headquarters linked to rare disease drug development

PARTNERSHIPS

4 Dec 2025

BioCryst Bets on Convenience in High-Stakes HAE Deal

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

US Food and Drug Administration headquarters sign in Washington DC

REGULATORY

17 Nov 2025

FDA Floats Faster Route for Ultra-Rare Therapies

A new FDA proposal could speed rare disease drug approvals using small trials, with tighter science standards and long-term patient follow...

Eli Lilly corporate headquarters reflecting investment in rare disease research

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 Attruby medicine box for transthyretin amyloid cardiomyopathy

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...

Illustration of mitochondria linked to rare disease drug research and biotech innovation

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

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