Yahoo Finance

Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting

WOODBRIDGE, Conn., June 24, 2025--(BUSINESS WIRE)--Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful ...
Business Wire

Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting Spinocerebellar Ataxia Type 3 (SCA3)

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the ...
Yahoo Finance

Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9)

Designation supports development of CRD-003, an investigational therapy targeting the root cause of Limb-Girdle Muscular Dystrophy Type R9 WOODBRIDGE, Conn., September 24, 2025--(BUSINESS WIRE)--Cure ...
BioWorld

Cure Rare Disease’s CRD-003 designated orphan drug for LGMD2i/R9

The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic ...
BioWorld

Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3

The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia ...