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Gramma Biosciences

Philadelphia, Pennsylvania, United States

Ownership: Private
Development stage: Other

Overview

GEMMABio is a private, pre-clinical genetic medicines company developing AAV-based gene therapies for rare diseases; clinical-stage gene therapy programs for ultra‑orphan lysosomal storage diseases (including GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy) are being advanced in an affiliated spin‑out, Rare Therapeutics, Inc. (RareTx).[3][7][9]

Therapeutic areas: CardiovascularNeurologyRare Diseases
Modalities: Gene therapyAdeno-associated virus based gene therapyBiologic
Industry: Biotech

Partnerships

Fiocruz (Brazilian public health foundation) for delivery of gene therapies through Brazil’s public health system[2]Public‑private partnerships and regional centers of excellence in Brazil and Abu Dhabi for regional gene therapy hubs via affiliate Rare Therapeutics[3][9]

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