Red Nucleus的动态

?? Reducing the high costs of manufacturing advanced therapies is crucial to ensuring widespread patient access. Academic institutions and nonprofits play a key role in the development of complex gene therapies, but doing so requires significant investment and commercial viability concerns, and often sees the development of promising treatments for rare diseases paused before they can reach patients who could benefit. By streamlining cell therapy manufacturing and automating labor-intensive steps, throughput can be increased, costs lowered, and reliability improved – addressing many of the barriers to viability. Our CEO Jason C. Foster talked to Pharmaceutical-Technology to outline how IRO?, our manufacturing platform, can support manufacturers to scale up production and reduce costs – making therapies more affordable for providers and patients. Find out how IRO? could scale your impact. Book a demo today ?? https://lnkd.in/eXEXEGZZ Thank you Pharmaceutical-Technology for the feature: https://lnkd.in/dewsVbcd #IRO #TheNewStandard #AutomateBetterBiology #AccelerateProductDevelopment #ScaleYourImpact

We’re reflecting on a great experience at #ATW24 (Phacilitate) in Miami and grateful for the time spent together with our colleagues in the #advancedtherapies field. Here are three key insights from the conference: ?? Automation continues to be a major focus within the industry, not just in bioprocess but also in analytics. Both are bolstered by an increased interest in using #ArtificialIntelligence and #MachineLearning, since these powerful combinations can streamline development, improve cell and gene therapies’ robustness and quality, and overcome common #CMC issues that impact our industry’s progress. The industry continues to explore how these capabilities can better connect clinical outcomes and #CMC to enhance safety and efficacy of advanced therapies. ?? A sustainable analytical and quality control strategy is paramount to a therapeutic developer’s ability to understand their process and seamlessly get to market. The key is to ensure your product is well-characterized applying phase-appropriate, robust and, where possible, rapid analytical testing early in development. ?? Patient testimonials continue to be powerful reminders of why we do what we do. Patient advocate Laurie Adami shared her personal journey with cancer, and the incredible impact CAR T medicine Yescarta has had on her quality of life. More on Laurie’s journey here: https://lnkd.in/gsyhuVgb Click to learn more about our services or to request a consult from our experts: https://lnkd.in/gg7aFvBb #AdvancedTherapiesWeek #ATW #ATW24 #Phacilitate #celltherapyanalyticaldevelopment #cellandgenetherapy #cdmo #advancedtherapies #celltherapies #celltherapymanufacturing #cellandgenetherapymanufacturing #advancedtherapymanufaturing #cgtforwardprogress #WeAreOmniaBio?

We will be presenting our novel #CRISPR /high-fidelity Cas12i variant (hfCas12Max) and hfCas13Y discovered and engineered through the Company’s AI/ML-guided CRISPR-based HG-PRECISE platform to treat #Alzheimer , #ALS , #DMD , #AMD , and #MECP2 duplication syndrome, novel engineered #AAV vector carrying #mRNA for transient expression of Cas protein, AAV gene replacement therapy with a better viral vector to reduce the AAV-mediated toxicity, and an update on Company progress, at the September/October conferences: Dr. Alvin L., CEO, will present at: European Society of Retina Specialists (EURETINA) 24th?Annual Congress, 19-22 September 2024, Barcelona, Spain. World Muscle Society (WMS) 29th?Annual Congress, 8-12 October, Prague, Czechia. In addition, members of the HuidaGene Therapeutics and Synthego Corporation teams will present at: European Society of Gene and Cell Therapy (ESGCT) 31st?Annual Congress, 22-25 October, Rome, Italy. Alzheimer's Association? Alzheimer's Society Duchenne UK HEAL DMD Muscular Dystrophy Association Asociación Mácula Retina #alzheimer #ENDALZ #MeCP2 #dupmecp2 #muscle #duchenne #musculardystrophy #inheritedblindness #maculardegeneration #ALS #alzhiemers #dementia #braindisorders #genetherapy #geneediting #biotechnology #technology #innovation #pharmaceuticals #healthcare #science

?? Breakthrough Therapies or Regulatory Minefield? The Truth About ATMPs Revealed! Advanced Therapy Medicinal Products (ATMPs) are revolutionizing medicine, but navigating their development is like walking through a regulatory labyrinth. ???? Our article uncovers: ?Why ATMPs are the next frontier in treating cancer and rare disorders ?How global health authorities are fast-tracking these innovations ?The complex classification system of cell and gene products Don't miss out on understanding the future of medicine. Read our in-depth analysis now and stay ahead of the curve: https://shorturl.at/0BOc6 #ATMP #CGT #BiotechInnovation #RegulatoryAffairs

At Vivet Therapeutics we understand that living with a #raredisease such as #WilsonDisease can be physically, mentally, financially, and emotionally exhausting. Key findings published in the international peer-reviewed medical journal - Current Medical Opinion & Research (CMRO), indicate that an unmet need exists among patients with #WilsonDisease (WD) and their care partners, which underscores the need for more effective treatments to reduce the burden of disease and improve the outcomes of patients with WD. Read the full article in the link in the comments below! #Vivet is committed to working with research and development teams to create a diversified pipeline of novel gene therapies that provide treatment options to clinicians, with long lasting benefits that will improve the quality of life of those living with a rare disease. #AAVGeneTherapy #GenomicMedicines #CellandGeneTherapy #GenomeEditing #CGT

Insightful key takeaways by the team at Vivet Therapeutics, highlighting the burden and unmet need of patients with #WilsonDisease! Follow #Vivet to keep up to date with its lead program, VTX-801 currently advancing in a Phase 1/2 GATEWAY trial for the treatment of Wilson Disease - encouraging safety, tolerability and early pharmacodynamic changes observed in Cohort 1 as it advances in Cohort 2 with the dosing of its first patient with lead program VTX-801!

Our mission is to enable widespread patient access to lifesaving cell and gene therapies. Here's how we're making this a reality... Collaborations with our LEAP partners have provided invaluable feasibility data for IRO?, demonstrating performance in real-world scenarios. At this year's ISCT, Jason Bock, Founder and CEO of CTMC shared his direct experience using the platform. Here are his key insights: ?? ?? Automate Better Biology IRO? delivered higher CAR+ yields and more efficient transduction (~69% vs ~45%) versus the control. ?? Accelerate Product Development Process establishment on IRO? took <5 weeks from kick-off, and met all target release criteria by day 6 across two independent sites. ?? Scale Your Impact Vector copy number remained below the FDA-recommended <5 per transduced cell, meaning IRO? maintained safety and consistency. By empowering scientists with a new standard of tools for therapy development, we're pushing the boundaries in CGT manufacturing to enable widespread patient access and scale the impact of advanced therapies. Read more of our case study data: https://lnkd.in/gKUgg39R #IRO #TheNewStandard #AutomateBetterBiology #AccelerateProductDevelopment #ScaleYourImpact CTMC / A joint venture between Resilience + MD Anderson Cancer Center

?? Cracking the EMA Code: Essential Insights for Rare Disease Therapies in Europe ?? It's week 2, and this week's articles in our "Cracking the EMA Code" series deliver expert guidance for your #raredisease therapy's European journey: ?? Tomorrow, June 11th: Prof. Boris Kramer, a seasoned expert in Translational Science and cell therapies, shares insights on designing and executing successful rare disease clinical trials for #EMA approval. ??June 14th: An exclusive Q&A with Dr. Joshi Venugopal, General Manager of Gene Therapy and Rare Diseases, Europe at Novartis, offers invaluable perspectives on navigating gene therapy commercialization in Europe. Missed our earlier articles? Catch up on Prof. Trevor Jones' introduction to expanding into Europe and Agnes Kohl's overview of European regulatory frameworks. ?? https://lnkd.in/e2PZnDJ9 #orphandiseases #genetherapy #celltherapy #biotech #marketaccess #EU #europe #drugdevelopment #patientcentricity #clinicaltrials

The Avenna team had?a?pleasure?to?attend?ELRIG UK last week at the ExCeL Arena in London.? ? We?discovered?a wide range of topics, from mechanisms of ageing and cell & gene therapy to more practical aspects like advances in assay design, high-content imaging, target identification, and disease modelling. ? Did you know that only 5 out of every 100 cancer drugs make it from clinical trials to market? Drug discovery is tough, often involving very high costs (>$4BN) and a low likelihood of success when moving from an initial idea to a marketed drug! ? This and more, we?have?learned?during?the talk by Cathy Tralau-Stewart Executive Director at Milner Therapeutics Institute. Her insights?into?the need for a better predictive model resonated?with our current work?in the field of Inflammatory Bowel diseases.?She highlighted the challenge of addressing current limitations in understanding human biology and disease processes. By?bridging this?gap?we can improve the efficiency of drug discovery and?speed up?translation?of innovations into tangible patient outcomes.? ? Another?notable speech?was @Professor Arne Akbar of UCL, who shared compelling strategies to enhance immunity during ageing,?a vital?point to address?as we work to improve health outcomes for the ageing population. ? Let’s keep pushing the boundaries of what’s possible in drug discovery. If you are developing a drug and looking for complementary or companion diagnostics, get in touch with?our team?to learn more about our GlyHealth Technology.? ? #ELRIGDD24 #DrugDiscovery #TranslationalResearch #LifeSciences #Collaboration #Avenna #GlyHealth #Biomarkers