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Explore BCIQ profiles by entering a target name.
BioCentury | May 11, 2021
Emerging Company Profile
DTx: Optimizing RNA therapies for cellular uptake, biodistribution
Emerging Company Profile: DTx is slated to enter the clinic next year with its fatty acid ligand conjugated RNA therapy to treat retinitis pigmentosa
BioCentury | Apr 29, 2021
Distillery Therapeutics
shRNA-based AAV gene therapy for Charcot-Marie-Tooth disease
DISEASE CATEGORY: Neurology
INDICATION: Neuropathy A PMP22-targeting gene therapy could treat Charcot-Marie-Tooth disease 1A, a demyelinating neuropathy caused by PMP22 duplication. In a rat
INDICATION: Neuropathy A PMP22-targeting gene therapy could treat Charcot-Marie-Tooth disease 1A, a demyelinating neuropathy caused by PMP22 duplication. In a rat
BioCentury | Apr 30, 2019
Distillery Therapeutics
RUNX1, RUNX3, CBFB and PMP22 as targets for neurofibromatosis
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