Company intelligence / Profile preview

Glafabra Therapeutics

Salt Lake City, Utah, United Statesglafabra.com ↗

Ownership: Private
Development stage: Phase 1

Overview

Glafabra’s lead program is a lentivector-based ex vivo stem cell gene therapy for Fabry disease. The approach has shown safety and efficacy in Canadian clinical trials, with patients reporting normalization of health status post-treatment. US IND filing is planned for mid 2025, with Phase I/II trials to follow. The company aims to provide a one-time treatment that lasts at least five years.

Therapeutic areas: Rare Diseases
Modalities: Ex vivo gene therapy using lentivectors (LV)Cellular gene therapyT cell-based gene therapy (TRAM, iTRAM systems)
Industry: Biotech

Drug pipeline

3 assets

GT-GAA-S04

Preclinical

2 more assets in the full pipeline.

Partnerships

University of Utah Data Coordinating Center (DCC) – clinical trial management partnerCellReGen at University of Utah – technology transfer/manufacturing partnerMedical College of Wisconsin – exclusive rights to novel lentivector backbone technology

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