[{"company":"Lonza Group","sponsor":"Aruvant Sciences","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Aruvant Chooses Lonza to Manufacture ARU-1801","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lonza Group"},{"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Receives Orphan Drug Designation for ARU-1801 for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene 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Spring","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Announces Oral Presentation at American Society of Gene and Cell Therapy (ASGCT)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Announces Data Presentation at European Hematology Association 2022 Hybrid Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"company":"Orphan Technologies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphan Technologies Receives Rare Pediatric Disease Designation from FDA for OT-58","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Orphan Technologies"},{"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","amount":"$517.0 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Details:
The data shows that ARU-1801 offers potential long-term improvement of the primary symptoms seen with SCD without the toxicities and resource utilization associated with fully myeloablative chemotherapies.
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
June 10, 2022
Details:
Data demonstrate that ARU-1801 gene therapy may not only be able to reduce severe vaso-occlusive events but also reduce days in hospital for SCD patients, which could provide a clinically meaningful benefit for patients and help reduce health care costs.
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
May 16, 2022
Details:
ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative chemotherapy.
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
April 19, 2022
Details:
The agreement aims to support ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). Aruvant has chosen Lonza to help develop and manufacture ARU-1801 for its upcoming pivotal trial.
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:
Aruvant Sciences
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Partnership
January 18, 2021
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.
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:
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.
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:
Travere Therapeutics
Deal Size: $517.0 million
Upfront Cash: $90.0 million
Deal Type: Acquisition
November 12, 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).
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:
Agreement adds OT-58, a novel enzyme replacement therapy in Phase 1/2 development for the treatment of classical homocystinuria.
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:
Travere Therapeutics
Deal Size: $517.0 million
Upfront Cash: $90.0 million
Deal Type: Acquisition
October 22, 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.
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:
Orphan Technologies is now eligible to receive a priority review voucher that may be utilized for subsequent human drug applications.
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
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