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Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 19, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful change in both the primary and secondary endpoints.
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Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.
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Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: BPS-804
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 24, 2020
Details:
In Mereo’s Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in addition to being safe and well tolerated in adults with OI.
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Lead Product(s): OTL-103
Therapeutic Area: Genetic Disease Product Name: OTL-103
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Orchard Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement July 15, 2020
Details:
Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.
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Lead Product(s): Human retinal progenitor cell therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: 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 29, 2020
Details:
The Company reports that the latest data continue to demonstrate the efficacy of the therapy (hRPC stem cell therapy) , with a clinically meaningful benefit being observed at all time-points.
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Lead Product(s): SAR422459
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Sanofi
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement June 09, 2020
Details:
Oxford Biomedica has been informed by Sanofi that following completion of a company-wide portfolio review, it intends to return to OXB the rights to the following ophthalmology gene therapy programmes: SAR422459 for Stargardt disease & SAR421869 for Usher’s Syndrome type 1b.
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Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2020
Details:
IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of NPC in a multinational clinical trial (IB1001-201).
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Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 14, 2020
Details:
Setrusumab demonstrated a dose dependent increase in both failure load and stiffness at the radius at 12 months, achieving statistical significance in the high dose cohort.
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Lead Product(s): Tideglusib
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 09, 2020
Details:
Trial to assess AMO-02 in treatment of congenital myotonic dystrophy to begin patient enrollment in early 2020.
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