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Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"PTC Therapeutics \/ Not Applicable"}]

Find Clinical Drug Pipeline Developments & Deals by PTC Therapeutics

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                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Upstaza (eladocagene exuparvovec) is a gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase enzyme (hAADC) deficiency in children and adults.

                          Brand Name : Kebilidi

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 13, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, indicated for the treatment of nonsense mutated duchenne muscular dystrophy , in patients aged 2 or above.

                          Brand Name : Translarna

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 18, 2024

                          Lead Product(s) : Ataluren

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                          Brand Name : PTC743

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 08, 2024

                          Lead Product(s) : Vatiquinone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.

                          Brand Name : PTC923

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 01, 2024

                          Lead Product(s) : Sepiapterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.

                          Brand Name : PTC518

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 26, 2024

                          Lead Product(s) : PTC518

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.

                          Brand Name : PTC518

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 20, 2024

                          Lead Product(s) : PTC518

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Upstaza (eladocagene exuparvovec) BLA has been accepted , it is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC).

                          Brand Name : Upstaza

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 14, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.

                          Brand Name : PTC923

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 28, 2024

                          Lead Product(s) : Sepiapterin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Upstaza (eladocagene exuparvovec) is a gene therapy expressing the human aromatic L-amino acid decarboxylase enzyme, evaluated for AADC deficiency treatment.

                          Brand Name : Upstaza

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 19, 2024

                          Lead Product(s) : Eladocagene Exuparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : ClearPoint Neuro

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          BMWS24
                          Not Confirmed
                          BMWS24
                          Not Confirmed

                          Details : Translarna (ataluren) is a protein restoration therapy under phase 3 development for nonsense mutation Duchenne Muscular Dystrophy, enabling functioning protein formation in patients.

                          Brand Name : Translarna

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          January 25, 2024

                          Lead Product(s) : Ataluren

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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