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Lead Product(s): Ravulizumab
Therapeutic Area: Neurology Product Name: Ultomiris
Highest Development Status: Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Alexion Pharmaceuticals
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 20, 2021
Details:
The IDMC recommended that the trial be discontinued due to lack of efficacy. No new safety findings were observed and the data were consistent with the established safety profile of Ultomiris.
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Lead Product(s): Elivaldogene autotemcel
Therapeutic Area: Neurology Product Name: Skysona
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 21, 2021
Details:
SKYSONA uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s own HSCs.
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