Latest News

  • INNOVATION

    20 Feb 2026

    Regulators Clear Path for BAG3 Heart Trial

  • 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

Rare Disease Boom Redefines Pharma Strategy

Pharma executives shaking hands to mark rare disease drug partnership

MARKET TRENDS

14 Dec 2025

Big drugmakers step up investment in niche therapies as science and partnerships turn small patient groups into core growth areas

U.S. Food and Drug Administration approval document with red approved stamp

INNOVATION

20 Feb 2026

Regulators Clear Path for BAG3 Heart Trial

EMA orphan nod and FDA IND clear BAG3 gene therapy for a 2026 trial in rare heart disease

Biotechnology site developing nanoparticle drug delivery systems

INNOVATION

11 Feb 2026

Can Nanomedicine Rescue Stalled Rare Drugs?

A new nanoparticle platform seeks to solve stubborn formulation hurdles in rare disease drug development

Roche logo displayed on company building exterior

MARKET TRENDS

5 Feb 2026

Rare Disease Drug Boom Sparks Dealmaking and Rivalry

More diagnoses and bold acquisitions are reshaping the orphan drug market, even as pricing and access pressures intensify

Amicus Pharmaceuticals office interior shown during BioMarin deal coverage

PARTNERSHIPS

3 Feb 2026

Why Scale Is Suddenly Everything in Rare Disease Drugs

BioMarin’s Amicus deal highlights a pivot toward scale, steady revenue, and broader portfolios as rare disease companies brace for more me...

Pharma executives shaking hands to mark rare disease drug partnership

MARKET TRENDS

14 Dec 2025

Rare Disease Boom Redefines Pharma Strategy

Big drugmakers step up investment in niche therapies as science and partnerships turn small patient groups into core growth areas

DNA strand being edited to represent experimental rare disease gene therapy

INNOVATION

11 Dec 2025

An Experimental Gene Therapy Signals New Hope for Rare Disorders

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

SUBSCRIBE FOR UPDATES

By submitting, you agree to receive email communications from the event organizers, including upcoming promotions and discounted tickets, news, and access to related events.