regulatory

News

New FDA knockback hits uniQure and Huntington patients hard

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.

R&D

Rare disease at an inflection point: Why the next wave will ...

As rare disease becomes more competitive, information parity is disappearing.

News

Moderna closes on EU approval for combined flu/COVID jab

Moderna's combined vaccine for both flu and COVID-19 has been recommended for approval in the EU, after being knocked back in the US.

News

US lawmakers take FDA to task over rare diseases record

Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.

News

FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.

R&D

Navigating development in a complex molecule world

For decades, the pharmaceutical industry has relied on accelerated development and manufacturing timelines to bring new therapies to patients quickly.

New FDA knockback hits uniQure and Huntington patients hard

Rare disease at an inflection point: Why the next wave will ...

Moderna closes on EU approval for combined flu/COVID jab

US lawmakers take FDA to task over rare diseases record

FDA maps new regulatory route for ultra-rare diseases

Navigating development in a complex molecule world

LSX2026: On promise and patience, with Hans Schambye

Using AI-driven synthetic personas to take your insights fur...

regulatory

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