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            Details:

            Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.

            Lead Product(s): Sirolimus

            Therapeutic Area: Rare Diseases and Disorders Product Name: Sirolimus

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: University College Cork

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 12, 2024

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            Niagen (nicotinamide riboside chloride) is an oral supplement which significantly increases NAD+ levels, it is being investigated in Ataxia Telangiectasia.

            Lead Product(s): Nicotinamide Riboside Chloride

            Therapeutic Area: Rare Diseases and Disorders Product Name: Niagen

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 07, 2024

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            MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage clinical trial studies for the treatment of LAMA2-congenital muscular dystrophy.

            Lead Product(s): MDL-101

            Therapeutic Area: Rare Diseases and Disorders Product Name: MDL-101

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 07, 2024

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            MIV-711 is a potent and selective inhibitor of cathepsin K, the principal protease involved in breaking down collagen in bone and cartilage. It is being evaluated for the treatment of Legg-Calvé-Perthes Disease.

            Lead Product(s): MIV-711

            Therapeutic Area: Rare Diseases and Disorders Product Name: MIV-711

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 25, 2024

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            Rezolute candidate RZ358, which is an insulin receptor allosteric site activator. Currently, it is being evaluated in the preclinical trial studies for the treatment of Non-Islet Cell Tumor Hypoglycemia.

            Lead Product(s): Ersodetug

            Therapeutic Area: Rare Diseases and Disorders Product Name: RZ358

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 06, 2024

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            The funding will accelerate the development of Neurenati's NEU-001, which is being evaluated in the preclinical studies for the treatment of Hirschsprung’s disease.

            Lead Product(s): NEU-001

            Therapeutic Area: Rare Diseases and Disorders Product Name: NEU-001

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Genson Capital

            Deal Size: $1.2 million Upfront Cash: Undisclosed

            Deal Type: Funding February 29, 2024

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            GC1126A is a novel ADAMTS13 mutein designed to evade autoantibodies with an extended half-life, which is investigated for the treatment of thrombotic thrombocytopenic purpura.

            Lead Product(s): GC1126A

            Therapeutic Area: Rare Diseases and Disorders Product Name: GC1126A

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 05, 2023

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            Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

            Lead Product(s): Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: $1,000.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 20, 2023

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