Seqens Seqens

X
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Entity","date":"June 2022","url1":"","url2":"","graph1":"Oncology","graph2":"Preclinical"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Receives Positive CHMP Opinion for Use of CRYSVITA\u00ae (burosumab) for the Treatment of Tumour-Induced Osteomalacia (TIO)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"MEI Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MEI Pharma and Kyowa Kirin Report New Clinical Data on Zandelisib at American Society of Clinical Oncology Annual Meeting 2022","therapeuticArea":"Oncology","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Oncology","graph2":"Phase III"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Announces Discontinuation for Developing KW-6356","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"MEI Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MEI Pharma and Kyowa Kirin Announce Publication in The Lancet Oncology of Data from Phase 1b Clinical Study of Zandelisib in Patients with Relapsed or Refractory B-cell Malignancy","therapeuticArea":"Oncology","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Oncology","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Oxford Biomedica","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Oxford Biomedica Initiates New Project With Orchard Therapeutics Utilising LentiStable\u2122 Technology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Receives European Commission Approval for Use of CRYSVITA\u00ae (burosumab) for the Treatment of Tumour-Induced Osteomalacia (TIO)","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Announces Multiple Presentations at 2022 ESGCT Annual Congress Showing the Potential of HSC Gene Therapy in Several Therapeutic Areas","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Ardelyx","pharmaFlowCategory":"D","amount":"$520.5 million","upfrontCash":"$10.5 million","newsHeadline":"Ardelyx Collaboration Partner, Kyowa Kirin, Announces Submission of New Drug Application for Tenapanor for Hyperphosphatemia in Japan","therapeuticArea":"Nephrology","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Nephrology","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces First Neurocognitive Data from OTL-201 Study in MPS-IIIA Accepted for Oral Presentation at ASH 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"MEI Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin and MEI Pharma Announce Topline Data from the Phase 2 MIRAGE Study Evaluating Zandelisib in Patients with Indolent B-cell non-Hodgkin\u2019s Lymphoma in Japan","therapeuticArea":"Oncology","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Oncology","graph2":"Phase II"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Grunenthal","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$82.7 million","newsHeadline":"Grunenthal and Kyowa Kirin International Announce Joint Venture Collaboration for Established Medicines Portfolio","therapeuticArea":"Neurology","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"MEI Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MEI Pharma and Kyowa Kirin Announce Discontinuation of Zandelisib Development Outside of Japan Following Recent FDA Meeting","therapeuticArea":"Oncology","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Oncology","graph2":"Phase II"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Promising Early Neurocognitive Outcomes from Ongoing Proof-of-concept Study of OTL-201 in MPS-IIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IH","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"New Therapies Council","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Agreement Enabling Reimbursed Access to Libmeldy for All Eligible MLD Patients in Sweden","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"$188.0 million","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Strategic Financing Totalling up to $188 Million","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Will Present New Rocatinlimab Phase 2b Data in Atopic Dermatitis at the American Academy of Dermatology Annual Meeting 2023","therapeuticArea":"Dermatology","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Dermatology","graph2":"Phase III"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Reata Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Announces Phase III Study Results of bardoxolone methyl (RTA 402) in Japan and Discontinuation of Development","therapeuticArea":"Nephrology","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Nephrology","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Guggenheim Securities, LLC","pharmaFlowCategory":"D","amount":"$188.0 million","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Second Closing of Strategic Financing, Resulting in $34 Million of Additional Capital","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD and Receives Priority Review","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"$387.4 million","upfrontCash":"$387.4 million","newsHeadline":"Kyowa Kirin to Acquire Orchard Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"$477.6 million","upfrontCash":"$387.4 million","newsHeadline":"Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Beneluxa Initiative","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"San Raffaele-Telethon Institute for Gene Therapy","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$100.0 million","newsHeadline":"BridgeBio Pharma and Kyowa Kirin Announce Partnership with an Upfront Payment of $100 Million for an Exclusive License on Infigratinib in Skeletal Dysplasias in Japan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives FDA Approval of Lenmeldy\u2122 (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Post Hoc Analysis from MAVORIC Trial Sheds Light on the Burden of Cutaneous T-Cell Lymphoma on Health-Related Quality of Life","therapeuticArea":"Oncology","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Oncology","graph2":"Approved"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kyowa Kirin Announces First Patient Enrolled in the Phase 2 Clinical Trial Evaluating Tivozanib Eye Drops in Patients with Diabetic Macular Edema","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Phase II","country":"JAPAN","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Phase II"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Boehringer Ingelheim GmbH","pharmaFlowCategory":"D","amount":"$441.8 million","upfrontCash":"Undisclosed","newsHeadline":"Boehringer Ingelheim Licenses a New Compound as Part of its Commitment to People Living with Fibro-Inflammatory Diseases\u200b from Kyowa Kirin","therapeuticArea":"Immunology","highestDevelopmentStatus":"Undisclosed","country":"JAPAN","productType":"Undisclosed","productStatus":"Undisclosed","date":"January 2024","url1":"","url2":"","graph1":"Immunology","graph2":"Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            Details:

            Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells. It is approved for the treatment of early-onset metachromatic leukodystrophy.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Lenmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 20, 2024

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            Lenmeldy (atidarsagene autotemcel) is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells. It is approved for the treatment of early-onset metachromatic leukodystrophy.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Lenmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 18, 2024

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            Under the terms of the agreements, Kyowa Kirin has an exclusive license to develop and commercialize BGJ398 (infigratinib) for the treatment of achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan.

            Lead Product(s): Infigratinib

            Therapeutic Area: Genetic Disease Product Name: BGJ398

            Highest Development Status: Phase III Product Type: Small molecule

            Recipient: BridgeBio Pharma

            Deal Size: Undisclosed Upfront Cash: $100.0 million

            Deal Type: Licensing Agreement February 07, 2024

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            KHK4951 (tivozanib) is a small-molecule vascular endothelial growth factor receptor (VEGFR)-1, -2, and -3 tyrosine kinase inhibitor. It is being evaluated in phase 2 clinical trials for the treatment of diabetic macular edema & neovascular (wet) age-related macular degeneration.

            Lead Product(s): Tivozanib

            Therapeutic Area: Ophthalmology Product Name: KHK4951

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 06, 2024

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            OTL-203 is an investigational hematopoietic stem cell gene therapy which uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. It is being evaluated in Phase 3 clinical trials for MPS-I Hurler Syndrome.

            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: San Raffaele-Telethon Institute for Gene Therapy

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 05, 2024

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            The agreement aims for enabling reimbursed access to Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Beneluxa Initiative

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement January 25, 2024

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            Through the acquisition, Kyowa expands its portfolio, enables the development of promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Rare Diseases and Disorders Product Name: Libmeldy

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

            Recipient: Orchard Therapeutics

            Deal Size: $477.6 million Upfront Cash: $387.4 million

            Deal Type: Acquisition January 24, 2024

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            Under the terms of the agreement, Boehringer Ingelheim will receive exclusive, worldwide rights from Kyowa Kirin to develop novel, first-in-class treatments for fibro-inflammatory diseases.

            Lead Product(s): Undisclosed

            Therapeutic Area: Immunology Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Boehringer Ingelheim GmbH

            Deal Size: $441.8 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement January 05, 2024

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            OTL-200 (libmeldy or atidarsagene autotemcel), is a hematopoietic stem cell (HSC) gene therapy approved by Swissmedic for the treatment of early-onset metachromatic leukodystrophy (MLD).

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 11, 2023

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            Poteligeo (mogamulizumab-kpkc), a humanized, afucosylated monoclonal antibody targeting CCR4, is a treatment for adults living with the rare blood cancers mycosis fungoides and Sézary syndrome, two subtypes of cutaneous T-cell lymphoma.

            Lead Product(s): Mogamulizumab-kpkc

            Therapeutic Area: Oncology Product Name: Poteligeo

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 11, 2023

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