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[{"orgOrder":0,"company":"Can-Fite BioPharma","sponsor":"Fondazione Telethon","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Can-Fite Enters Rare Genetic Disease Field Based on Impactful Findings of a Fondazione Telethon and Naples University Researcher Showing Piclidenoson is Efficacious in Treating Lowe Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Can-Fite BioPharma"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jaguar Gene Therapy Launches with Mission to Accelerate Breakthroughs in Gene Therapy for Patients Suffering from Severe Genetic Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Jaguar Gene Therapy"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Eli Lilly","pharmaFlowCategory":"D","amount":"$139.0 million","upfrontCash":"Undisclosed","newsHeadline":"Jaguar Gene Therapy Closes $139 Million Series B Funding Co-Led by Eli Lilly and Company and Deerfield Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Jaguar Gene Therapy"},{"orgOrder":0,"company":"Jaguar Gene Therapy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jaguar Gene Therapy Presents Preclinical Data of JAG101 in Type 1 Galactosemia at American Society of Gene and Cell Therapy 25th Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Jaguar Gene Therapy"},{"orgOrder":0,"company":"SpliSense","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SpliSense Presents Preclinical Data for SPL23-ASO, mRNA-Altering Drug Candidate for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"SpliSense"},{"orgOrder":0,"company":"Quris","sponsor":"Welltech Ventures","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Quris Closes $28 Million in Seed Funding From Top Institutional Investors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"ISRAEL","productType":"Undisclosed","productStatus":"Undisclosed","date":"January 2022","url1":"","url2":"","graph1":"Undisclosed","graph2":"Quris"}]

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            Details:

            The agreement aims for the development of CF101 (piclidenoson), a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule, orally bioavailable drug, for the treatment of Lowe Syndrome, a high medical need with no drug available.

            Lead Product(s): Piclidenoson

            Therapeutic Area: Genetic Disease Product Name: CF101

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Fondazione Telethon

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement August 24, 2023

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            Preclinical studies support continued development of JAG101, an investigational gene therapy designed to address the root cause of Type 1 galactosemia and reduce multiple toxic metabolites.

            Lead Product(s): JAG101

            Therapeutic Area: Genetic Disease Product Name: JAG101

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 16, 2022

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            The funding will fast-track Quris’s novel drug research and development, team expansion and partner-building efforts as it scales to rapidly scan 100s of drug candidates to train its AI engine in its mission to revolutionize the drug development process.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Welltech Ventures

            Deal Size: $28.0 million Upfront Cash: Undisclosed

            Deal Type: Financing January 11, 2022

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            Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein.

            Lead Product(s): SPL23-ASO

            Therapeutic Area: Genetic Disease Product Name: SPL23-ASO

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 11, 2021

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            Jaguar will use Series B funding proceeds to continue to advance its initial pre-clinical pipeline, which utilizes the proven and well-characterized AAV9 vector to target diseases in larger patient populations with significant unmet need.

            Lead Product(s): JAG101

            Therapeutic Area: Genetic Disease Product Name: JAG101

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Eli Lilly

            Deal Size: $139.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing April 13, 2021

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            Details:

            Jaguar Gene Therapy is focused on advancing its initial pre-clinical pipeline of AAV9-based gene therapies which includes JAG101, JAG201, JAG301, AXV101 focusing on key therapeutic areas such as galactosemia, autism spectrum disorder, Type 1 diabetes and BBS1 respectively.

            Lead Product(s): JAG101

            Therapeutic Area: Genetic Disease Product Name: JAG101

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 25, 2021

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