American Society of Gene & Cell Therapy’s Post

This is a really important discussion hosted by the American Society of Gene & Cell Therapy. In order to improve patient access to transformative #CGT medicines, we need to consider the downstream effects of how trial designs inform drug labels, which are later referenced by payers making reimbursement determinations that may ultimately exclude some patients. We need thoughtful drug development and evidence generation strategies in both the pre- and post-approval settings as a means of effectively expanding access. #ASGCTPatientEducation

Hoping to see you soon at our upcoming webinar!

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

Great to be supporting more research into ground-breaking cell and gene therapy by funding this important network for another four years. The £17.9m in funding will bolster clinical trial infrastructure in the UK and help maintain our reputation as a location of choice for advanced therapy research. Professor Marian Knight, Scientific Director for NIHR Infrastructure said: “The NIHR is committed to ensuring that the UK provides a research environment to enable rapid assessment of new advanced therapies with the potential to transform health and care. "Partnerships such as these, linked with existing NIHR research infrastructure, will help ensure that the UK public is able to benefit from these ground-breaking new treatments." Find out more 👇 https://lnkd.in/egxMFP7U 💡 Innovate UK 🧬 Cell and Gene Therapy Catapult

Heading to #ATW24 this year? Let’s discuss how we can partner to simplify your cell and gene therapy research and manufacturing processes by providing the consistency, scalability, and automation you need to streamline your path to commercialization. #advancedtherapiesweek

⚛️ Cell and gene therapy is primed to have a big year in 2024, and here is a collection of interesting predictions prepared by Mass General Brigham. Unsurprisingly, there is a clear focus on FDA approval and ensuring safety for future participants. As we have seen in other pieces on similar topics though, paramount to the success of these therapies is not only ensuring that they are indeed safe, but also educating patients and healthcare providers to the extent they feel confident about their safety. #celltherapy #genetherapy #crispr #genomics https://hubs.ly/Q02f2Klp0

"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

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.

📢 HTID#6 News 📢   Join us at HTID#6 for a compelling discussion on "Success & Frustration: Personalized Medicine, Cell and Gene Therapy Trends"    This session will feature insights from Frédéric Desdouits (TreeFrog Therapeutics), Anne Moore, PhD (Roland Berger), Miguel Forte (Kiji Therapeutics) and David Venables (Laverock Therapeutics). Moderated by Elsy Boglioli Hofman, this session promises to delve into the latest trends and challenges in personalized medicine and advanced therapies.    Make sure not to miss out on the invaluable insights offered by these European experts !   #HTID6 #HealthcareInnovation #CellTherapy #GeneTherapy #MedicalAdvancements