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Cure Rare Disease

Woodbridge, Connecticut, United Statescureraredisease.org ↗

Ownership: Private
Development stage: Other

Overview

Successfully developed a First-in-Human CRISPR transcriptional activator that received FDA IND approval in July 2022. The first patient (founder's brother) unfortunately passed away during early clinical trial stages due to an immune reaction to the viral vector (AAV).

Therapeutic areas: Rare DiseasesNeurology
Modalities: Gene therapyCRISPR gene editingantisense oligonucleotides (ASOs)
Industry: Biotech

Drug pipeline

14 assets

CRD-TMH-001

13 more assets in the full pipeline.

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