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            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 years and older.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: Global Blood Therapeutics

            Deal Size: $353.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement March 16, 2021

            Sanofi Company Banner

            IPS 2024

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            Details:

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

            Lead Product(s): CRISPR-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Sanofi

            Deal Size: $1,240.0 million Upfront Cash: $40.0 million

            Deal Type: Expanded Collaboration January 03, 2024

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            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.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Genetic Cures for Kids

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 28, 2023

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            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.

            Lead Product(s): Pancreatic Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Michigan Medicine

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 16, 2023

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            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.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: Novo Nordisk

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 06, 2023

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            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 action.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Vaccine

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration October 17, 2023

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

            Lead Product(s): Peptide Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Peptide

            Partner/Sponsor/Collaborator: Merck & Co

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Collaboration June 22, 2023

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            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-onset muscular dystrophy.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Westlake Village BioPartners

            Deal Size: $51.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing June 08, 2023

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            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 Huntington's disease.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Undisclosed

            Partner/Sponsor/Collaborator: UCB Pharma S.A

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration March 15, 2023

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