Drugs in Development
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Lead Product(s): FLT180a
Therapeutic Area: Genetic Disease Product Name: FLT180a
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 13, 2020
Details:
Updated FLT180a data in the B-AMAZE study in severe Haemophilia B patients demonstrated the potential for sustained normal FIX activity levels.
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Lead Product(s): LentiGlobin
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 12, 2020
Details:
LentiGlobin™ gene therapy for adult and adolescent patients with sickle cell disease (SCD) show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS).
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Lead Product(s): OTL-201
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 27, 2020
Details:
First patient has been dosed in an open-label, investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of mucopolysaccharidosis type IIIA.
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Lead Product(s): FLT190
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 10, 2020
Details:
A 3 to 4-fold increase in plasma αGLA activity was achieved by week 4 and sustained through the data cutoff at week 20.
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Lead Product(s): FLT190
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 12, 2020
Details:
As of the data cutoff date, there have been no infusion-related adverse events. Two Grade 1 or 2 serious adverse events have been reported in the study.
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