Creyon Bio’s Post

Excited that Creyon’s CMO, David Dimmock, will be sharing data at #ASGCT2024 from the investigator-initiated clinical trial of a novel allele-selective Locked Nucleic Acid (LNA) antisense oligonucleotide that Creyon created. Creyon used our AI/ML-driven predictive models to identify compounds, with greater than 80% of them validated as safe, and move rapidly from whiteboard to dosing a patient with an ultra-rare disease. This would not have been possible without the hard work and dedication of the Creyon team, Nicole Coufal at Rady Children’s Hospital and UCSD, the research team at Rady Children’s Hospital, and the TNPO2 Foundation. This demonstrates the power of Creyon’s predictive models for ultra-rare disease, but we are also excited about the efficiencies that we are able to realize creating novel Oligonucleotide-Based Medicines (OBMs) for rare and common diseases as well. Creyon is committed to changing how we create OBMs - moving from trial-and-error drug discovery to drug engineering.

#Gene and #cell-based therapies have the potential to provide targeted and personalized treatments, according to Charité - Universitätsmedizin Berlin and Bayer. They are a beacon of hope for people for whom conventional therapies have failed or for whom there is no treatment so far. With Charité and Bayer, two partners for major medical progress are coming together who will establish a new center for gene and cell therapy in #Berlin, which is to be established in 2025. More information in first comment #healthforall #teambayer

“A full-length dystrophin is highly desired for the treatment of Duchenne muscular dystrophy, and it has not yet been delivered.” Our CEO, Aki Ko, joined Erin Harris on the Cell & Gene Podcast to discuss underserved segments of #DuchenneMuscularDystrophy patients and how we are working to change that with our Bobcat mRNA™ therapeutic. 🎧 Listen here: https://bit.ly/3KyPDjm #CellAndGenePodcast

"It's great to see a willingness to learn and a willingness to take some risk. We should take advantage of this partnership because we're all learning together and delivering value to patients." - Miguel Forte, Kiji Therapeutics The clinical development roadmap for cell and gene therapy is ever-changing. How can we prepare for a more integrated approach to clinical trial design and execution? Brought to you by Precision For Medicine and hosted by Endpoints News. WATCH NOW: https://hubs.ly/Q02ybr8t0 #CGTx #CGTxDay2024 #CellTherapy #GeneTherapy #transformativemedicine

Thank you Lung-I Cheng for facilitating this robust discussion at Reuters Events Pharma about the value of #patient engagement in #cell and #gene therapy development. Key takeaways included: *Innovative study designs, such as leveraging #RWE, is needed to develop treatments for rare diseases which often have small patient populations. *Leveraging information from #FDA's patient focused drug development meetings is important to understanding the patient experience. *The rapid development of #CGTs is a paradigm shift for many regulating bodies, and risk-based flexability is needed to ensure timely #patient #access. *Long-term patient follow up remains a challenge, but the value of capturing patient #data remains a priority. Fellow Panelists: Olga Granaturova, Co-founder, President, PriveBio Jeffrey Meckler, Chief Executive Officer, Indaptus Therapeutics Jason Lott, MD MSHP MHS FAAD, VP, Global Integrated Evidence Generation, Specialty Medicine, Integrated Care, and Cell-Gene Therapy, Bayer Special thanks to my colleagues American Society of Gene & Cell Therapy for supporting my participation at events on this important topic. #ASGCT #ASGCTadvocacy #CellTherapy #GeneTherapy

"It's great to see a willingness to learn and a willingness to take some risk. We should take advantage of this partnership because we're all learning together and delivering value to patients." - Miguel Forte, Kiji Therapeutics The clinical development roadmap for cell and gene therapy is ever-changing. How can we prepare for a more integrated approach to clinical trial design and execution? Brought to you by Precision For Medicine and hosted by Endpoints News. WATCH NOW: https://hubs.ly/Q02xXpMD0 #CGTx #CGTxDay2024 #CellTherapy #GeneTherapy #transformativemedicine

Designing a patient-centric clinical trial that prioritizes safety should always be top of mind. Innovative medical treatments, such as cell, gene and immunotherapies, are on the rise. Patients are in need of life-saving treatments. It’s important that sponsors have a clinical research partner who understands and adapts to the ever-evolving regulatory landscape when it comes to these trials. Download Advanced Clinical’s fact sheet to learn about our immunotherapy clinical research experience and solutions: https://bit.ly/44vt2xm

🧬 Unlocking the Potential of PARP/DDR Pathway for Drug Discovery 🌟 The DNA damage response (DDR) plays a pivotal role in maintaining genomic stability. Dysfunctions in DDR can lead to cancer and uncontrolled cell growth. Among the key players in this pathway are the PARP family of proteins, essential for gene transcription and DNA repair. At Promega, we're at the forefront of PARP/DDR pathway drug discovery. Our comprehensive suite of assays and technologies enables precise measurement of compound binding, PARP and kinase activity, and cellular health. Looking to dive deeper into PARP/DDR pathway solutions for groundbreaking drug discovery? Reach out to explore how we can assist! https://bit.ly/3SOe1m2

"It's great to see a willingness to learn and a willingness to take some risk. We should take advantage of this partnership because we're all learning together and delivering value to patients." - Miguel Forte, Kiji Therapeutics The clinical development roadmap for cell and gene therapy is ever-changing. How can we prepare for a more integrated approach to clinical trial design and execution? Brought to you by Precision For Medicine and hosted by Endpoints News. WATCH NOW: https://hubs.ly/Q02xXtRJ0 #CGTx #CGTxDay2024 #CellTherapy #GeneTherapy #transformativemedicine

Designing a patient-centric clinical trial that prioritizes safety should always be top of mind. Innovative medical treatments, such as cell, gene and immunotherapies, are on the rise. Patients are in need of life-saving treatments. It’s important that sponsors have a clinical research partner who understands and adapts to the ever-evolving regulatory landscape when it comes to these trials. Download Advanced Clinical’s fact sheet to learn about our immunotherapy clinical research experience and solutions: https://bit.ly/44vt2xm #ClinicalTrials