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Find Clinical Drug Pipeline Developments & Deals by Avrobio
The combined company will focus on advancing Tectonic’s pipeline, including TX45 (TX000045), a Fc-relaxin fusion protein targeting the RXFP1 receptor and a potential best-in-class agent.
Under the divestment, AVROBIO will sell its investigational hematopoietic stem cell (HSC) gene therapy program, including CTNS-RD-04 (aka AVR-RD-04) for the treatment of cystinosis to Novartis.
AVR-RD-02 is an ex vivo lentiviral gene therapy, being developed by AVROBIO for the treatment of Gaucher's disease, including Gaucher's disease type I.
All four adult GD1 patients in Guard1 clinical trial have been infused with investigational AVR-RD-02 to date saw sustained engraftment with VCN between 0.54 to 0.86 per diploid genome 14 weeks to two years post gene therapy, and reconstitution of GCase enzyme activity.
AVR-RD-04 is designed to genetically modify patients’ own HSCs to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis.
The gene therapy, which AVROBIO refers to as AVR-RD-05, is designed to transduce autologous (a patient’s own) HSCs ex vivo with a lentiviral vector encoding a brain-targeted iduronate-2-sulfatase (IDS) enzyme, which is deficient in these patients.
AVR-RD-05 (lentiviral gene therapy),for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.
AVR-RD-03, AVROBIO’s gene therapy for Pompe disease, includes a proprietary Glycosylation-Independent Lysosomal Targeting -tag which consists of a short peptide sequence linked to the therapeutic protein and is designed to enhance uptake in targeted tissues.
AVR-RD-04 (CTNS-RD-04), an investigational gene therapy for cystinosis, genetically modifies patients’ own hematopoietic stem cells (HSC) to express a functional version of cystinosin, the protein that is deficient in people living with cystinosis.
AVR-RD-04 is an investigational, lentiviral-based gene therapy designed to potentially halt or reverse the progression of cystinosis with a single dose of the patient’s own hematopoietic stem cells.
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