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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: 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:
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
Partner/Sponsor/Collaborator: Kyowa Kirin
Deal Size: $477.6 million Upfront Cash: $387.4 million
Deal Type: Acquisition January 24, 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:
OTL-203 is an IDUA gene Transference cell and gene therapy drug infused intravenously, it is being investigated for the treatment of Hurler subtype of mucopolysaccharidosis type I.
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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 30, 2023
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: Genetic Disease Product Name: Libmeldy
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Kyowa Kirin
Deal Size: $387.4 million Upfront Cash: $387.4 million
Deal Type: Acquisition October 05, 2023
Details:
Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity.
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 September 18, 2023
Details:
Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity.
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 August 03, 2023