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Apertura Gene Therapy

PharmaDeviceNews Apertura Gene Therapy

Apertura Gene Therapy’s TfR1 CapX delivery platform will support an ARPA-H-backed consortium developing gene-editing therapies for Dravet syndrome, alternating hemiplegia of childhood and other rare pediatric neurological diseases. Representative image.

Pharma & Biotech

Apertura joins $34.5m ARPA-H PERC program for rare pediatric CNS gene editing

by Pallavi Madhiraju
July 25, 2026
0 Comments

Apertura joins Broad’s $34.5 million ARPA-H PERC program, pairing TfR1 CapX with gene editing for rare pediatric brain diseases.

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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