UBriGene at SAPA 2024 for Cell and Gene Therapy

🕺🎵 Everyday I'm shuffling ... Or not⁉️ In the dynamic field of gene therapy, optimizing Adeno-Associated Virus (AAV) vectors is crucial for effective therapeutic delivery with two options: 𝐆𝐮𝐢𝐝𝐞𝐝 𝐑𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝐃𝐞𝐬𝐢𝐠𝐧 or 𝐃𝐢𝐫𝐞𝐜𝐭𝐞𝐝 𝐄𝐯𝐨𝐥𝐮𝐭𝐢𝐨𝐧 (random suffling). At WhiteLab Genomics, we believe 𝐆𝐮𝐢𝐝𝐞𝐝 𝐑𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝐃𝐞𝐬𝐢𝐠𝐧 is Gaining Ground. Here’s why: ✅ Using in-depth knowledge of AAV capsid structures, coupled with advanced computational models, we can make targeted modifications and predict outcomes with high accuracy. This minimizes wet lab experiments and accelerates development. ✅ By focusing on precise, data-driven changes, we reduce the development timeline, bringing therapies to clinical application faster with better target product profile. ✅ Rational design offers scalable solutions, facilitating broader applications in gene therapy. ❌ 𝐃𝐢𝐫𝐞𝐜𝐭𝐞𝐝 𝐄𝐯𝐨𝐥𝐮𝐭𝐢𝐨𝐧 (random suffling) relies on creating a vast library of random AAV variants and extensive screening to find effective ones. This method is labor-intensive and time-consuming, often associated with prolonged timelines. More importantly, ultimately we struggle to reach our target product profile. 🔍 Which approach do you think holds the most promise for the future of gene therapy? What do you think is the future of AAV vector development ? Let’s dive into this debate! 🧬! Share your thoughts, experiences in the comments below. 👇

I had the privilege of attending the Bridging the Gap seminar series: Connecting Science, Industry, & Cell & Gene Therapy today, and it was truly insightful. The esteemed panel consisting of #DrSiddharthaMukherjee, #TomWhitehead, #DrPatrickHanley, and #AlbertRibickas shared their valuable insights. What resonated with me the most was the story behind the story - the human resilience and tenacity displayed by the workers who built the #Immuneel #GMP facility during the challenging times of the COVID-19 pandemic. Despite facing limited resources s, these workers diligently continued their work, even going so far as to build a village around the facility. Their unwavering dedication is truly commendable. There were several other key takeaways from the seminar: The #FDA recognizes the impact of cell and gene therapies and is committed to accelerating the approval process and improving patient access. This understanding is crucial for the advancement of these modalities. The #regulatory aspects of cell and gene therapies are equally important, particularly in terms of #standardization and global adoption. Ensuring regulatory compliance is essential for the success and widespread acceptance of these therapies. #AI and #machinelearning have the potential to revolutionize the development of new technologies and expedite #drugdiscovery. By analyzing #geneticdata, identifying patterns, and assessing #riskfactors and patient profiles, these technologies can assist physicians in identifying the most suitable candidates for therapy. Overall, the seminar provided valuable insights into the intersection of science, industry, and cell and gene therapy. It highlighted the resilience of individuals and the importance of regulatory considerations and technological advancements in driving progress in this field. Thankyou #GeorgeEastwood for moderating the panel. #celltherapy #genetherapy #insights #Emilywhiteheadfoundation #bridgingthegap #olgabukatova #patientaccess #inspiration #Immuneel

Size matters when we talk about gene length. Remember those iconic images comparing the size of hard drives from 40 years ago to today? What once was the size of a vehicle can now fit in your hand. In #GeneTherapy, VHHs are making similar strides. Traditional gene therapy often struggles with the large size of genetic material that needs to be inserted into viral vectors. 🟣 Isogenica's VHH antibodies are only one-tenth the size of classical antibodies, and half the size of scFvs. For gene therapies, the coding DNA required is significantly shorter than for conventional antibody fragments, offering key advantages: · Increased Vector Capacity: This allows for additional genetic elements, boosting the therapeutic effect or adding extra functionality such as bi-specificity. · Improved Delivery: smaller genetic payloads enhance viral transduction rates, ensuring more target cells receive and express the therapeutic gene. · Correct Folding: VHHs, being simple molecules, are more likely to fold correctly when expressed in vivo, which is critical for safety and efficacy. ✅ Just as modern memory cards revolutionized data storage, we're backing a transition from enormous genetic sizes to compact ones, offering great solutions in gene therapy. #biotech #ViralVector #VHH #Isogenica #AAV

𝐑𝐄𝐆𝐈𝐒𝐓𝐄𝐑 𝐍𝐎𝐖, 𝐓𝐈𝐌𝐄 𝐈𝐒 𝐑𝐔𝐍𝐍𝐈𝐍𝐆 𝐎𝐔𝐓 Institute for Advanced Clinical Trials for Children is excited to announce tomorrow's educational webinar on the intricate world of cell and gene therapy! Join us as we delve into the science, applications, and ethical considerations of this transformative field. Equip yourself with the knowledge to avoid misunderstandings or misconceptions that impact a family’s ability to make an informed decision about potentially participating in a cell and gene therapy trial. Webinar Objectives ·Understand the fundamental principles of gene manipulation. ·Identify different types of cell and gene therapies. ·Explain the delivery methods for therapeutic genes. ·Identify potential applications for treating various diseases. ·Comprehend the ethical considerations surrounding Cell and Gene Therapy. ·Identify the long-term challenges of Cell and Gene Therapy. Guest Speakers: Daniel Eisenman PhD, CBSP Carolyn Riley Chapman, PhD MS 𝐌𝐚𝐫𝐢𝐭 𝐒𝐢𝐯𝐞𝐫𝐭𝐬𝐨𝐧, 𝐉𝐃 https://lnkd.in/ewJrhJFx #Ethics #CGT

In Cell & Gene Therapy, we hide behind the "hubris of science", but we still have a lot of operational challenges to overcome before we can really impact the lives of patients. This reality check comes from Sanjay Srivastava, PhD, Managing Director of Accenture's Centre of Excellence for Cell & Gene Therapy. With over a decade in cell and gene therapy, including launching the first CAR-T therapies, Sanjay shares his expert insights in the latest episode of the PharmaSource podcast. Key takeaways include: - Less than 5% of eligible patients globally receive CAR-T therapies. We must do better. - Bringing therapies to patients, not vice versa, is crucial. Think mobile apheresis labs and point-of-care manufacturing. - Automation isn't a cure-all. It's about gaining deeper insights into process biology, not just cost-cutting. - "Go slow to go fast." Fine-tune processes before scaling up to avoid future headaches. Rushing to clinic doesn't guarantee commercial success. Your thoughts? How else can we bridge the gap between scientific breakthroughs and operational excellence? 🎧 Listen to the full episode here: https://lnkd.in/ebfi8Cuh #CGT #CellandGeneTherapy #PersonalizedMedicine

“We are at an exciting point in the development of cell and gene therapies. A lot is evolving in this space, and I am extremely positive that operational scale and new technologies will help us reduce the cost per dose for advanced therapies. If during 2018-2022 we have been in a period of irrational optimism, and from 2023 till now we have been in a phase of irrational pessimism, we are now entering an era of rational realism: good science is getting funded and reaches the patients and the market. No one is questioning any more the value of cell and gene therapies. We have proven that they can save lives in unprecedented ways. Collaboration and partnerships can do great things both for the industry and for society, and we share a common passion to bring therapies to life.” There is a lot to be excited about for cell and gene therapies in 2024. Alberto Santagostino, SVP of Cell and Gene Therapies at Lonza, recently gave his thoughts on the outlook for the sector, and some of the technologies which could play an important role in bringing advanced therapies to more patients as a participant in Endpoints News’ Cell & Gene Day 2024. #AHealthierWorld #CellTherapy #GeneTherapy #PatientAccess #Innovation

Cell and gene therapy is at another pivotal moment. While challenges remain, ARM’s 2024 Cell & Gene Meeting on the Mesa outlined ideas on how the sector can mature into a new phase for manufacturing and commercialization. Inside Precision Medicine covers more on how industry leaders at #CGMesa24 are shaping the future for cell and gene therapy. https://lnkd.in/ez9QJzM8 #cellandgenetherapy

Introducing Cell and Gene Therapy in Clinical Trials Learning Module! https://lnkd.in/eGy4gDXT Embark on a transformative journey into the realm of innovative therapies with our comprehensive learning module designed to equip you with basic knowledge and skills needed to prepare for navigating the complexities of #cell and #gene therapy clinical trials. 👉 Gain insights into the fundamental concepts behind cell and gene therapy, understanding their revolutionary potential in transforming patient care. 🔍 Explore the intricacies of trial processes and delve into the #regulatory landscape to ensure #compliance and success. 💡 Unlock a wealth of resources, including regulatory guidelines, #exRNA Atlas, and more, empowering you to leverage the latest tools and insights for optimal trial outcomes. With the #patient at the helm, our learning module fosters a patient-centric approach, prioritizing their well-being and ensuring that every step of the trial journey is guided by #compassion and #dedication. Shaping the future of medicine with cell and gene therapies. Let's innovate together! 💪 #CellTherapy #GeneTherapy #ClinicalTrials #Innovation #PatientCentricity #CGT #exvivo #MRNA #allogenic #autologous #stemcellresearch #cohortmanagement #raredisease #goodclinicalpractice #opentowork #lifesciencestechnology #biotechtechnology #biotechtech #AI #ML #artificialintelligence #techinvestors #lifesciencescollaboration #lifescienceshub #learning #knowledge #clinicaloperations #clinicaldevelopment #cellandgenetherapy

𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐟𝐨𝐫 𝐇𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚: A New Era of Treatment 🚀 - Precision Medicine 🧬: Gene therapy targets the root genetic cause of hemophilia by delivering functional copies of the defective genes, potentially offering long-term solutions beyond traditional treatments. - Clinical Advances 👩⚕️👨⚕️: Recent trials show promising decreases in bleeding episodes and reduction of factor replacement therapies, bringing hope for improved quality of life. - Innovative Techniques 🛠️: Technologies such as CRISPR and viral vectors are being harnessed to boost delivery efficiency and safety, paving the way for broader application. - Regulatory Milestones 📜: Approvals and fast-track designations signal increasing support for gene therapy, encouraging further research and development in the field. 🔍 To delve deeper into cutting-edge gene therapy research, browse the latest reviews on https://www.sciqst.com. #GeneTherapy #Hemophilia #Biotech #BiomedicalResearch

Friends - An interesting article from My friend Sanjay Srivastava, PhD Sharing my thoughts - what do you think about the challenges he outlines? I believe it’s not so much that we are ‘hiding behind the science,’ but rather that many in the field are blinded by it. There are hundreds of firms embarking on this journey without the necessary experience to manage these products or the strategic thinking required to operationalize them in the market. In my conversations with these companies, I’ve noticed a consistent theme: they are so engrossed in the science that there’s little room for other crucial conversations. The reality is that developing the operational capabilities to scale precision medicine therapies demands as much design, testing, and resources as the therapies themselves. It’s often said that ‘the process is the product,’ and that rings especially true here. Much of the critical intel needed to fully understand and enable these processes only becomes clear in the commercial stages—a stage that many of these companies have yet to reach. Even for large players, entering the CAR-T space for the first time can be daunting. Sanjay’s insights are valuable, and I would add my own analogy: it’s like describing the ocean to someone who has never experienced water. It’s not that these companies don’t want to understand; they simply lack the framework to begin comprehending the complexities involved. The operational models necessary to launch and sustain these therapies often lie outside the conventional practices of biopharma and require the design and integration of processes that are almost unrecognizable compared to traditional methods. When you factor in new-to-industry academic founders and VC-backed partners, the challenges become even more pronounced. While some approved therapies have managed to deliver acceptable approaches, even they would admit that the puzzle is far from solved, despite significant investments and remarkable efforts.