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Avrobio
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One Kendall Square, Building 300, Suite 201 Cambridge, MA 02139
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617.914.8420

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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.


Lead Product(s): TX000045

Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: TX45

Highest Development Status: Phase IProduct Type: Large molecule

Recipient: Tectonic Therapeutic

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Merger January 30, 2024

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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.


Lead Product(s): CTNS-RD-04

Therapeutic Area: Genetic Disease Product Name: CTNS-RD-04

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation

Deal Size: $87.5 million Upfront Cash: $87.5 million

Deal Type: Divestment May 22, 2023

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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.


Lead Product(s): AVR-RD-02

Therapeutic Area: Genetic Disease Product Name: AVR-RD-02

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 09, 2023

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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.


Lead Product(s): AVR-RD-02

Therapeutic Area: Genetic Disease Product Name: AVR-RD-02

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 07, 2022

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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.


Lead Product(s): AVR-RD-04

Therapeutic Area: Genetic Disease Product Name: AVR-RD-04

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 25, 2022

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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.


Lead Product(s): AVR-RD-05

Therapeutic Area: Genetic Disease Product Name: AVR-RD-05

Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 14, 2022

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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.


Lead Product(s): AVR-RD-05

Therapeutic Area: Genetic Disease Product Name: AVR-RD-05

Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 13, 2022

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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.


Lead Product(s): AVR-RD-03

Therapeutic Area: Genetic Disease Product Name: AVR-RD-03

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 18, 2022

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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.


Lead Product(s): AVR-RD-04

Therapeutic Area: Genetic Disease Product Name: CTNS-RD-04

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 17, 2022

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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.


Lead Product(s): AVR-RD-04

Therapeutic Area: Genetic Disease Product Name: AVR-RD-04

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: University of California

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 27, 2022

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