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PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.
Lead Product(s): Sepiapterin
Therapeutic Area: Genetic Disease Product Name: PTC923
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 28, 2024
Details:
Upstaza (eladocagene exuparvovec) is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC). It is being evaluated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
Lead Product(s): Eladocagene Exuparvovec
Therapeutic Area: Genetic Disease Product Name: Upstaza
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: ClearPoint Neuro
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 19, 2024
Details:
Translarna (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. It is under phase 3 clinical development for the treatment of nonsense mutation Duchenne Muscular Dystrophy.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 25, 2024
Details:
Translarna (ataluren) is a dystrophin expression stimulator small molecule drug candidate, which is currently being evaluated for the treatment of duchenne muscular dystrophy from a nonsense mutated dystrophin gene, in patients aged 2 or above via oral suspension.
Lead Product(s): Ataluren
Therapeutic Area: Genetic Disease Product Name: Translarna
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 05, 2023
Details:
Under the agreement, Royalty Pharma acquires additional royalties on Evrysdi (risdiplam), a survival motor neuron 2 splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency.
Lead Product(s): Risdiplam
Therapeutic Area: Genetic Disease Product Name: Evrysdi
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Royalty Pharma
Deal Size: $1,500.0 million Upfront Cash: $1,000.0 million
Deal Type: Agreement October 19, 2023
Details:
PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Mitochondrial Disease Associated Seizures.
Lead Product(s): Vatiquinone
Therapeutic Area: Genetic Disease Product Name: PTC743
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2023
Details:
PTC518, is a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression.
Lead Product(s): PTC518
Therapeutic Area: Genetic Disease Product Name: PTC518
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 21, 2023
Details:
Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia.
Lead Product(s): Vatiquinone
Therapeutic Area: Genetic Disease Product Name: PTC743
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 23, 2023
Details:
PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products and has the potential to treat the broad range of PKU patients.
Lead Product(s): Sepiapterin
Therapeutic Area: Genetic Disease Product Name: PTC923
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 17, 2023
Details:
Waylivra (volanesorsen), an antisense oligonucleotide designed to inhibit the formation of apoC-III, is a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology. Waylivra has received conditional marketing approval in the EU as a treatment for FCS.
Lead Product(s): Volanesorsen Sodium
Therapeutic Area: Rare Diseases and Disorders Product Name: Waylivra
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 01, 2022
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