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Lead Product(s): Verbrinacogene setparvovec
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 December 14, 2020
Details:
Study results show durable Factor IX activity up to a period of nearly three years, with no bleeds reported requiring FIX supplementation, and support selection of a dose and immune management regimen that has the potential to deliver FIX activity in the normal range.
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Lead Product(s): OTL-201
Therapeutic Area: Genetic Disease Product Name: OTL-201
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 December 08, 2020
Details:
Preliminary results from first patient treated with OTL-201 show over expression of the therapeutic enzyme and decrease in substrate levels three months following gene therapy.
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Lead Product(s): OTL-201
Therapeutic Area: Genetic Disease Product Name: OTL-201
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 November 05, 2020
Details:
The oral presentation will highlight data from the first patient treated in the ongoing proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA.
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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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