Dr. Axel Gawantka’s Post

In May 2024, the 27th Annual Meeting of American Society of Gene & Cell Therapy (ASGCT) explored the current viral vector-based therapy landscape and the emerging vectors in the field. A panel of experts discuss the trends, conversations, and innovations occurring in the viral vector-based therapy sector at ASGCT.   Read what OrganaBio’s Gabrielle Freitas told The Medicine Maker about what techniques and innovations caused most excitement and which players presented promising data and potential solutions for #CGT: https://lnkd.in/eMm9BzGT #celltherapy #genetherapy #immunotherapy #regenerativemedicine

Tune-in to our presentation this week at #ASGCT2024 where we highlight how we used AI to engineer an allele selective antisense oligonucleotide investigational therapy and an investigator-initiated clinical trial received approval to treat a pediatric patient with an ultra-rare disease within a year. 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. https://lnkd.in/dezfE_V9 American Society of Gene & Cell Therapy, Nicole Coufal, TNPO2 Foundation

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.

🎙️🎧 From cell collection to commercial contracting: Advancements in cell & gene therapy manufacturing (Part II) In this episode, Fierce Pharma's Fraiser Kansteiner sits down with Delara Motlagh, head of cell therapy at Catalent Pharma Solutions; Becky Butler Cap, senior vice president of biotherapies at Vitalant; and Kevin Kyle, CEO of Germfree. Together, they discuss cell collection, the critical first step in advanced therapy manufacturing, and the importance of locking in process decisions early and how to effectively scale projects from the lab to the clinic and eventually to the market. Listen below ⬇️🎧 #CellGeneTherapy #CGT #CellandGeneTherapies

Take a look at this article, co-authored by Zhe Liu, PhD, Labcorp staff scientist, which focuses on development of a preclinical anti-AAV antibody assay to enable screening for pre-existing AAV immunity. Discover how this assay can help gene therapy developers identify potential low-responding individuals, increase the likelihood of successful transduction and maximize the utility of the preclinical investigations. Read the full article: http://spr.ly/6040pXp5E #GeneTherapy #AAV #CellandGeneTherapy

Regenerative medicine is not a distant reality—it's a transformative force that's shaping the world of medicinal therapeutics today. At Two Labs, we’ve proudly guided manufacturers from the earliest stages of pre-clinical development through to the life-changing patient outcomes that follow. If you missed our latest insights on the current state of the cell and gene therapy pipeline, we've made it easy to catch up. Check out our comprehensive webinar recap that charts the progress and previews the breakthroughs on the horizon. Start with the blog here and access the full webinar within for a full recap of where the pipeline stands. https://bit.ly/48wftyD

新增一批生物药和CGT研发职位（Antibody Discovery，Biology，immune-oncology， cell therapy process，cell therapy Discovery，AAV gene therapy Discovery，DMPK）https://lnkd.in/gVFt2Hj5

Check out Dr Kara Poons presentation on "Characterization of gene vectors using TEM" on May 10th at the #ASGCT 2024 conference! #TEM #genetherapy #QuTEM #innovation

💡 Get insight on the therapeutics pipeline, clinical targets, developer progress, and more! The American Society of Gene & Cell Therapy recently published its final report on the 2023 Gene, Cell, & RNA Therapy Landscape🔬 What are the highlights? • The pipeline for gene, cell, and RNA therapies grew by 6% • RNA therapies saw the greatest number of approvals across the gene, cell, and RNA therapy landscape, with eight first-time approvals • Dealmaking continued to decline for advanced molecular therapy companies 👉 Download the full report to learn more: https://lnkd.in/eUJuMzjX #celltherapy #genetherapy #clinicaltrials

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