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Find Clinical Drug Pipeline Developments & Deals by Orchard Therapeutics
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.
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.
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.
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).
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.
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).
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.
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.
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.
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.
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