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
07 Mayo 2024 - 7:00AM
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.
“These results illustrate a key pillar of our approach – to
engineer drugs for safety for rare and common diseases faster and
more efficiently compared to traditional drug discovery
techniques,” said Chris Hart, Ph.D., Co-Founder, Chief Executive
Officer, and President of Creyon Bio. “We developed predictive
models that guide our molecular engineering from the outset and
help mitigate potential safety concerns. This approach allowed us
to rapidly identify a drug candidate to target a specific genetic
variant and have a clinical study initiated within a year. This
proof-of-concept underscores the transformative potential of AI in
OBM drug engineering to rapidly create new OBMs for common and rare
diseases and target the genetic underpinning of disease anywhere in
the body.”
“Our mission in pursuing a research program with Creyon Bio was
to help our son develop to his fullest potential,” said Yiwei She,
founder of The TNPO2 Foundation. “Faced with an ultra-rare
diagnosis and a patient population ‘too small’ for academic labs
and traditional industry, developing a personalized treatment was
our only path forward. We have been encouraged by the measurable
improvements in his behavior, health, and developmental progress,
especially his reduced seizure burden. Through The TNPO2
Foundation, we will work collaboratively to create opportunities
for other rare disease families to give this same opportunity to
other children and families with rare diseases.”
Creyon has built the first and only platform capable of
engineering for safety first, creating novel OBMs with optimal
pharmacological properties engineered to minimize side effects.
Utilizing proprietary tools, AI, and custom datasets, Creyon
created 96 ASO candidates. Of those, 88 of the candidates were
demonstrated to be safe utilizing in vivo and in vitro model
systems, and several allele selective ASOs against the
disease-causing TNPO2 variant were identified that demonstrated
both safety and target selectivity in a cell model. The safest and
most effective molecule was scaled up for GMP manufacturing,
quality assessment and compounding.
Following toxicology studies and U.S. FDA permission to initiate
the clinical study granted to the clinical investigator, Dr. Nicole
Coufal, M.D., Ph.D., Associate Professor of Pediatrics at
University of California San Diego and an attending physician at
Rady Children’s Hospital San Diego, the patient received four
increasing doses up to 40mg. This treatment has been well-tolerated
with no increase in inflammatory markers in the cerebrospinal fluid
(CSF) or adverse changes on brain MRI scans. Notably, the patient
experienced reduced seizure frequency after the second dose and
regained developmental milestones including rolling, sustained
attention and gaining novel skills such as tripod sitting and
responsive babbling after the third dose.
“It has been so rewarding to care for this patient and to see
the hope and joy that this investigational treatment has brought
his family,” said Dr. Coufal.
“Seeing the patient regain several key developmental milestones
and that the investigational treatment was well-tolerated over nine
months is beyond what we dared hope for,” commented David Dimmock,
M.D., Chief Medical Officer of Creyon Bio. “Inflammatory reactions
to oligonucleotide therapies have caused serious adverse reactions
in other early clinical-stage therapies delivered directly to the
central nervous system. Our platform is designed to mitigate these
risks by leveraging AI-driven predictive models to identify and
engineer solutions that reduce the potential for such adverse
reactions.”
The oral presentation is titled “AI enabled ASO design can lead
to rapid initiation of treatment for an ultra-rare disorder leading
to allele selective knockdown of a toxic protein and consequent
clinical improvement” (Abstract 309), and the full abstract can be
viewed here.
About Creyon Bio, Inc.Creyon Bio is a clinical
stage biotechnology company developing best-in-class precision
medicines with industry-leading efficiency through molecular
engineering of Oligonucleotide-Based Medicines (OBMs). This
includes ASOs, siRNAs, and aptamers. The company is changing how we
create novel OBMs, transforming the process from drug discovery to
drug engineering. Creyon has built the first and only platform
capable of engineering for safety first, translating new target
discoveries to OBMs with optimal pharmacological properties.
Coupled with our aptamer-based delivery technologies, Creyon Bio is
unlocking the full potential of OBMs for common and rare diseases
alike. To learn more, visit creyonbio.com.
About The TNPO2 FoundationThe TNPO2 Foundation
was created to provide hope and action for families with children
facing rare diseases. Grounded in the belief that even the most
devastating diagnoses can be treatable with modern science,
medicine, and technology, the organization is dedicated to
expanding access to cutting-edge precision medicine and therapies.
The TNPO2 Foundation strives to support the challenges of those
with the greatest unmet needs, by raising and strategically
deploying funds to accelerate the research, development, and
administration of personalized treatments for children with TNPO2
mutations and other ultra-rare conditions. To learn more, visit
www.TNPO2.org.
Media ContactJon YuCreyonPR@westwicke.com