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Lead Product(s): ARU-1801
Therapeutic Area: Genetic Disease Product Name: ARU-1801
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 16, 2020
Details:
Preliminary clinical data from Aruvant's ongoing Phase 1/2 study that demonstrates ARU-1801, administered with only reduced intensity conditioning, can achieve durable reductions in disease burden.
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Lead Product(s): OT-58
Therapeutic Area: Genetic Disease Product Name: OT-58
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Retrophin
Deal Size: $517.0 million Upfront Cash: $90.0 million
Deal Type: Acquisition November 12, 2020
Details:
Orphan Technologies' lead product OT-58 is a novel investigational human enzyme replacement therapy being evaluated in Phase 1/2 development for the treatment of classical homocystinuria.
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Lead Product(s): ARU-1801
Therapeutic Area: Genetic Disease Product Name: ARU-1801
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 04, 2020
Details:
Data presented at ASH is from the MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD).
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Lead Product(s): OT-58
Therapeutic Area: Genetic Disease Product Name: OT-58
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Retrophin
Deal Size: $517.0 million Upfront Cash: $90.0 million
Deal Type: Acquisition October 22, 2020
Details:
Agreement adds OT-58, a novel enzyme replacement therapy in Phase 1/2 development for the treatment of classical homocystinuria.
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Lead Product(s): ARU-1801
Therapeutic Area: Genetic Disease Product Name: ARU-1801
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Sickle Cell Disease Association of America
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership October 12, 2020
Details:
In partnership with SCDAA, Aruvant is working to educate patients about gene therapy, while gaining critical insights from the patient community for its ARU-1801 SCD development program.
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Lead Product(s): OT-58
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 26, 2020
Details:
Orphan Technologies is now eligible to receive a priority review voucher that may be utilized for subsequent human drug applications.
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Lead Product(s): ARU-1801
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 January 22, 2020
Details:
Aruvant's ARU-1801, received FDA Orphan Drug designation for its efficacy to deliver a highly potent gene payload with enhanced anti-sickling properties, for a Reduced Intensity Conditioning regimen.
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