Top News

RNA strand illustration representing personalised antisense therapy development

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

BridgeBio Attruby medicine box for transthyretin amyloid cardiomyopathy

INSIGHTS

30 Oct 2025

BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

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

RESEARCH

20 Sep 2025

From Niche to Notable: Rare Diseases Reshape Biotech

Latest News

Why Small Molecules Are Back in the Spotlight

Blocks labeled Rare Diseases beside stethoscope on medical desk surface

MARKET TRENDS

19 Mar 2026

Drugmakers pivot to scalable small molecule therapies in rare disease pipelines

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

RNA strand illustration representing personalised antisense therapy development

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

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.