Drugs in Dev.
Genetic Disease
Phase I/ Phase II
Switzerland 
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Lead Product(s) : DFT383
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
DFT383 in Pediatric Participants With Nephropathic Cystinosis
Details : DFT383 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Cystinosis.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
April 04, 2025
Lead Product(s) : DFT383
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ACI-24.060
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
AC Immune Reports Interim Data from Phase 1b/2 ABATE Trial of ACI-24.060 in Down Syndrome
Details : ACI-24.060, a potential first-in-class, beta amyloid A4 protein modulator, therapeutic vaccine candidate, which is being evaluated for the treatment of down syndrome in adults.
Product Name : Undisclosed
Product Type : Vaccine
Upfront Cash : Inapplicable
December 10, 2024
Lead Product(s) : ACI-24.060
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ITU512
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Details : ITU512 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
August 09, 2024
Lead Product(s) : ITU512
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : NXT007
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : NXT007 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
August 14, 2023
Lead Product(s) : NXT007
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : OCTA8,OCTA12
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
July 04, 2022
Lead Product(s) : OCTA8,OCTA12
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aruvant Announces Data Presentation at European Hematology Association 2022 Hybrid Congress
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 10, 2022
Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Assess Safety and Efficacy of VAD044 in HHT Patients
Details : VAD044 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Telangiectasia, Hereditary Hemorrhagic.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
June 06, 2022
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aruvant Announces Oral Presentation at American Society of Gene and Cell Therapy (ASGCT)
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 16, 2022
Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aruvant to Participate in Multiple Conferences This Spring
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 19, 2022
Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Aruvant Sciences
Deal Size : Undisclosed
Deal Type : Agreement
Aruvant Chooses Lonza to Manufacture ARU-1801
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.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
January 18, 2021
Lead Product(s) : ARU-1801
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Aruvant Sciences
Deal Size : Undisclosed
Deal Type : Agreement
