Close-up of a sleeping newborn in a hospital hat with a nasal tube, smiling, next to a brown knitted toy

INNOVATION

20 May 2026

From One Baby to Many: Gene Therapy Finds a Repeatable Model

A prime editing platform for seven rare metabolic disorders is heading to U.S. clinical trials under a new FDA fast-track pathway

Wall St street sign with American flags flying on the New York Stock Exchange facade in the background

INVESTMENT

18 May 2026

No Cure, 1.4 Million Patients. Terremoto Has $108M to Try.

Terremoto Biosciences raises $108M Series C to advance a potential first-in-class therapy for HHT, a rare inherited vascular disease

Close-up of the Intellia Therapeutics logo in purple and red-orange lettering on a white surface in a lab

INNOVATION

15 May 2026

CRISPR Slashes Rare Swelling Attacks by 87%

Intellia's lonvo-z slashed hereditary angioedema attacks 87% in a landmark Phase 3 trial, becoming the first in vivo CRISPR therapy to suc...

Newborn baby with hearing test and forehead monitoring equipment in a clinical setting

TECHNOLOGY

13 May 2026

Born Deaf No More: FDA Greenlights Historic Treatment

Regeneron's Otarmeni restores hearing in children born with a rare genetic form of deafness and it won't cost patients a cent

Panel of four speakers at a conference table addressing a seated audience in a conference room

INSIGHTS

11 May 2026

Why FDA Approval Is No Longer Enough for Rare Disease Access

US rare disease developers must redesign evidence strategy to serve payers, not just regulators, NORD's 2026 Symposium finds

FDA Office of the Commissioner seal with US Food and Drug Administration text

REGULATORY

7 May 2026

One and Done: FDA Rips Up the Two-Trial Playbook

The FDA now accepts a single pivotal trial as its default for drug approval, a shift that could transform rare disease development

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