TNPO2

Creyon Bio to Present Clinical Data on Rapid AI-Enabled Engineering of Oligonucleotide-Based Medicines at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

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tisdag, maj 7, 2024
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SAN DIEGO and RESEARCH TRIANGLE PARK, N.C., May 07, 2024 (GLOBE NEWSWIRE) -- Creyon Bio, Inc. ("Creyon"), a clinical stage drug development company that engineers Oligonucleotide-Based Medicines (OBMs) with industry-leading efficiency creating novel, best-in-class gene-centric medicines to treat rare and common diseases, today announced encouraging clinical data highlighting the use of its custom designed data set paired with artificial intelligence (AI) to rapidly engineer an investigational antisense oligonucleotide (ASO) therapy. Within one year of project initiation in partnership with The TNPO2 Foundation, Creyon developed a novel allele-selective Locked Nucleic Acid (LNA) treatment candidate for an ultra-rare and severe neurological disease caused by a single nucleotide variant in the Transportin-2 (TNPO2) receptor, and an investigator-initiated clinical trial received approval to dose a patient. Initial data from this trial demonstrate that the investigational treatment successfully reduced seizures, restored developmental milestones, and was well-tolerated over nine months. Creyon will share these results during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore on Friday, May 10, 2024, between 5:15-5:30 p.m. EDT in Room 307-308 of the Baltimore Convention Center.

Key Points: 
  • Initial data from this trial demonstrate that the investigational treatment successfully reduced seizures, restored developmental milestones, and was well-tolerated over nine months.
  • Creyon will share these results during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore on Friday, May 10, 2024, between 5:15-5:30 p.m. EDT in Room 307-308 of the Baltimore Convention Center.
  • “We developed predictive models that guide our molecular engineering from the outset and help mitigate potential safety concerns.
  • “Inflammatory reactions to oligonucleotide therapies have caused serious adverse reactions in other early clinical-stage therapies delivered directly to the central nervous system.