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DT-216 is a GeneTAC™ small molecule designed to specifically target the GAA repeat expansion mutation, unblock the transcriptional machinery, and restore the production of functional, natural FXN mRNA.
Lead Product(s): DT-216
Therapeutic Area: Genetic Disease Product Name: DT-216
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 14, 2023
Details:
The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response.
Lead Product(s): DT-216
Therapeutic Area: Genetic Disease Product Name: DT-216
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 07, 2022
Details:
DT-216 is a novel GeneTAC™ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation, the underlying cause of FA, and restore frataxin (FXN) gene expression.
Lead Product(s): DT-216
Therapeutic Area: Genetic Disease Product Name: DT-216
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 30, 2022
Details:
GeneTAC™ molecules are designed to target specific genomic sequences and modulate transcription without requiring invasive procedures or gene editing, potentially offering a novel option for earlier medical treatment.
Lead Product(s): Undisclosed
Therapeutic Area: Ophthalmology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 09, 2022
Details:
Design Therapeutics is developing novel small-molecule therapies, called gene targeted chimeras (GeneTACs), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.
Lead Product(s): Gene targeted chimeras
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Small molecule
Partner/Sponsor/Collaborator: Renaissance Capital
Deal Size: $228.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering March 22, 2021
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