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Foundation"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"John Ballantyne","pharmaFlowCategory":"D","upfrontCash":"Undisclosed","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","date":"January 2023","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","upfrontCash":"Undisclosed","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","date":"January 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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Study Phase : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Details : These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 y...

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          March 16, 2021

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Sanofi Company Banner

                          02

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Evotec will collaborate with Pfizer to discover, and research potential new first-in-class therapeutic approaches for the treatment of metabolic and infectious diseases.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          July 10, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Pfizer Inc

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          03

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Under the collaboration, Sanofi has exercised its option for a second target as part of the companies' research collaboration to develop CRISPR-based therapeutics using Scribe’s CRISPR technologies.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $40.0 million

                          January 03, 2024

                          Lead Product(s) : CRISPR-based Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Sanofi

                          Deal Size : $1,240.0 million

                          Deal Type : Expanded Collaboration

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                          04

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Under the collaboration, GC4K will develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56), a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          November 28, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Genetic Cures for Kids

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          05

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The collaboration aims to explore mechanisms of metabolic improvement and weight loss from an intrapancreatic gene therapy leveraging GLP-1-based transgenes for long-duration treatment of type 2 diabetes (T2D) and obesity.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          November 16, 2023

                          Lead Product(s) : Pancreatic Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Michigan Medicine

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          06

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The collaboration aims to combine Genevant’s proprietary LNP technology with innovative mRNA-based megaTAL technology to develop an in vivo gene editing treatment for hemophilia A.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          November 06, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Novo Nordisk

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          07

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Under the collaboration, the Company will produce a prototype drug utilising Poolbeg's licensed oral delivery technology. Poolbeg believes that this technology can help to optimise the delivery of its partner's novel drug to its ideal site of therapeutic...

                          Brand Name : Undisclosed

                          Molecule Type : Vaccine

                          Upfront Cash : Undisclosed

                          October 17, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Undisclosed

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          08

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The collaboration aims to evaluate the opportunities for peptide-based therapeutic candidates across multiple indications through the well-established phage and mRNA display platforms.

                          Brand Name : Undisclosed

                          Molecule Type : Peptide

                          Upfront Cash : Undisclosed

                          June 22, 2023

                          Lead Product(s) : Peptide Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Merck & Co

                          Deal Size : Undisclosed

                          Deal Type : Expanded Collaboration

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                          09

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The proceeds will support the advancement of KateTx’s next generation AAV-based gene therapies for muscle and heart disease programs, including myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are the leading causes of adult-...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          June 08, 2023

                          Lead Product(s) : AAV-based Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Westlake Village BioPartners

                          Deal Size : $51.0 million

                          Deal Type : Series A Financing

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                          10

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The collaboration will combine the use of novel Huntington's disease drug targets from Aitia's Gemini Digital Twins with UCB's expertise in drug research and preclinical model systems in neurodegeneration to accelerate the discovery of new therapies for ...

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          March 15, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : UCB Pharma S.A

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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