[{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-02","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-01","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-02","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-02","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Avrobio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"Lentiviral gene therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Avrobio","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Avrobio"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-01","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"AVR-RD-01","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Avrobio \/ Morgan Stanley","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Morgan Stanley"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AVR-RD-06","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"AVR-RD-01","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Hematopoietic stem cells","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"AVR-RD-01","moa":"","graph1":"Genetic Disease","graph2":"Phase 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molecule","year":"2021","type":"Collaboration","leadProduct":"Humanized Anti-CD117 Monoclonal Antibody","moa":"CD117","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avrobio \/ AVROBIO","highestDevelopmentStatusID":"4","companyTruncated":"Avrobio \/ AVROBIO"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Lentiviral gene therapy","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Not 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Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ University of California","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ University of California"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"CTNS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Not 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Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-04","moa":"CTNS","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-02","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"AVR-RD-02","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Not Applicable"},{"orgOrder":0,"company":"Avrobio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"CTNS-RD-04","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Avrobio","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"","sponsorNew":"Avrobio \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"7","companyTruncated":"Avrobio \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Avrobio","sponsor":"Tectonic Therapeutic","pharmaFlowCategory":"D","therapeuticArea":"Pulmonary\/Respiratory Diseases","country":"","productType":"Large molecule","year":"2024","type":"Merger","leadProduct":"TX000045","moa":"","graph1":"Pulmonary\/Respiratory Diseases","graph2":"Phase I","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Respiratory Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ AVROBIO","highestDevelopmentStatusID":"6","companyTruncated":"Avrobio \/ AVROBIO"},{"orgOrder":0,"company":"Avrobio","sponsor":"Tectonic Therapeutic","pharmaFlowCategory":"D","therapeuticArea":"Pulmonary\/Respiratory Diseases","country":"","productType":"Large molecule","year":"2024","type":"Merger","leadProduct":"TX000045","moa":"","graph1":"Pulmonary\/Respiratory Diseases","graph2":"Phase I","graph3":"Avrobio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Respiratory Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Avrobio","highestDevelopmentStatusID":"6","companyTruncated":"Avrobio \/ Avrobio"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Through the merger with AVROBIO, Tectonic will be focused on advancing a pipeline, including TX45 (TX000045), which is being evaluated for Pulmonary Hypertension in patients with Heart Failure.

                          Brand Name : TX45

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          June 20, 2024

                          Lead Product(s) : TX000045

                          Therapeutic Area : Pulmonary/Respiratory Diseases

                          Highest Development Status : Phase I

                          Recipient : Tectonic Therapeutic

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : TX45

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          January 30, 2024

                          Lead Product(s) : TX000045

                          Therapeutic Area : Pulmonary/Respiratory Diseases

                          Highest Development Status : Phase I

                          Recipient : Tectonic Therapeutic

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : CTNS-RD-04

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $87.5 million

                          May 22, 2023

                          Lead Product(s) : CTNS-RD-04

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Novartis Pharmaceuticals Corporation

                          Deal Size : $87.5 million

                          Deal Type : Divestment

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : AVR-RD-02

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 09, 2023

                          Lead Product(s) : AVR-RD-02

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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 ...

                          Brand Name : AVR-RD-02

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 07, 2022

                          Lead Product(s) : AVR-RD-02

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : AVR-RD-04

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          October 25, 2022

                          Lead Product(s) : AVR-RD-04

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : AVR-RD-05

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 14, 2022

                          Lead Product(s) : AVR-RD-05

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : AVR-RD-05

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 13, 2022

                          Lead Product(s) : AVR-RD-05

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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 ti...

                          Brand Name : AVR-RD-03

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 18, 2022

                          Lead Product(s) : AVR-RD-03

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : CTNS-RD-04

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 17, 2022

                          Lead Product(s) : AVR-RD-04

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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