Group of scientists and a dignitary holding glass trophies at a formal prize ceremony on an outdoor stage

INNOVATION

29 May 2026

Sight Restored, Blood Fixed: Gene Therapy Wins Big

Seven scientists behind the first approved gene therapies for rare inherited blindness and sickle cell disease receive the 2026 Breakthrou...

Two wooden blocks with the words Rare Diseases printed on them beside a stethoscope on a light blue surface

MARKET TRENDS

28 May 2026

New Policies Restore Faith in Orphan Drug Markets

Washington rolls back restrictive price controls and extends crucial FDA voucher programs, sparking a massive investment revival for orpha...

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

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