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

Pills, capsule and thermometer on currency symbolising investment in rare disease biotech

INVESTMENT

9 Dec 2025

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

Mirum’s agreement to buy Bluejay highlights renewed investor interest and growing competition for late-stage rare disease assets

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

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.