[{"orgOrder":0,"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"},{"orgOrder":0,"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 therapy","graph2":"Aruvant Sciences"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Sickle Cell Disease Association of America","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sickle Cell Disease Association of America and Aruvant Sciences Forge New Partnership to Educate Around Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","amount":"$517.0 million","upfrontCash":"$90.0 million","newsHeadline":"Retrophin Announces Agreement to Acquire Orphan Technologies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Orphan Technologies"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Announces Updated Data to be Presented in Oral Presentation of ARU-1801 Data at the 62nd ASH Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","amount":"$517.0 million","upfrontCash":"$90.0 million","newsHeadline":"Retrophin Completes Acquisition of Orphan Technologies, OT-58 Strengthens Pipeline of Therapies Targeting Rare Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Orphan Technologies"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Receives Orphan Designation from European Medicines Agency for Gene Therapy ARU-1801 for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Aruvant Sciences"},{"orgOrder":0,"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"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant to Participate in Multiple Conferences This 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"},{"orgOrder":0,"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"},{"orgOrder":0,"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"},{"orgOrder":0,"company":"Octapharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Octapharma Shares New Data at ISTH 2022 in Its Mission to Meet the Challenges Faced by People With Bleeding Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Octapharma"}]
Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development in SWITZERLAND
OCTA101, (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer/OCTA8) a recombinant FVIII developed for subcutaneous administration in adult patients with severe haemophilia A.
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.
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.
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.
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.
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.
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.
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).
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.