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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: ApprovedProduct 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
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: ApprovedProduct 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
Details:
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 IIIProduct Type: Small molecule
Recipient: BridgeBio Pharma
Deal Size: Undisclosed Upfront Cash: $100.0 million
Deal Type: Licensing Agreement February 07, 2024
Details:
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 IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 06, 2024
Details:
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 IIIProduct 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
Details:
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: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Beneluxa Initiative
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement January 25, 2024
Details:
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: ApprovedProduct Type: Cell and Gene therapy
Recipient: Orchard Therapeutics
Deal Size: $477.6 million Upfront Cash: $387.4 million
Deal Type: Acquisition January 24, 2024
Details:
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: UndisclosedProduct Type: Undisclosed
Partner/Sponsor/Collaborator: Boehringer Ingelheim GmbH
Deal Size: $441.8 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement January 05, 2024
Details:
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: ApprovedProduct 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
Details:
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: ApprovedProduct 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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