CAI’s Post

Conclusions, take home messages from the fantastic business panel with Jack O'Meara, Kathryn Golden, Lee Dunham, Michael Anstey at Innovation Forum's Healthcare 2.0 Cell and Gene Therapy conference yesterday: 1. 'The Maths doesn't work' - Michael Anstey brought the high level message home: for VCs to invest, there must be good ROI balanced by risk. In rare disease the addressable market size is small, while the RnD costs are high...How can we make the Maths work? ➗ are rare disease RnD and n=1 trials forever stuck within academia and philantropy? 2. Cost vs Price - RnD attrition + high manufacturing costs hike the price up. Jack O'Meara brought up a new pricing model for 'once and done' cures: a success-based price structure with an affordable upfront payment followed by a lifetime direct debit setup. At first this sounds just ethically wrong ('rent' your life until the day you die?!), but if we think of the price of Zolgensma making it inaccessible for children whose life prospects it significantly improves, that puts this into perspective. Would this ever work within the NHS though? 🤔 3. Automation first - Kathryn Golden raised the point that using automation during RnD phase then scaling it to manufacture establishes the mindset within the company from the get go → removes the bottleneck of having to redevelop manual processes into automated ones. BUT, there is always a but - how could an early stage biotech possibly afford this? This is where Cell and Gene Therapy Catapult can come in providing a test bed - a well set conclusion from Lee Dunham ✨ Arctoris is here to apply modular automation during the RnD on a project cost basis. The day has come where automation is not only affordable in the HTS and manufacturing context, we can use it affordably in low / mid throughput setting. Come and talk to us if you'd like to give it a go → better data will lead to better decisions and as Bradley Hardiman has so eloquently put it, VC's would lose their shirts less often, and spend less time trying to climb out of a ditch 💵

The cell and gene therapy (CGT) landscape is rapidly evolving. Here’s how we’re helping our stop loss customers navigate the cost implications of these emerging treatment technologies. If you’d like to learn more about our approach to cell and gene therapy claims, let’s connect!

Our Accelerating Science Cell & Gene Therapy Feature is now live, showcasing lab product reviews by scientists like you. Explore top reviews on key technologies from gene editing to manufacturing. #celltherapy #genetherapy #sciencecommunication #reviews #labproducts #geneediting

Significant advances in upstream biomanufacturing methods for cell and gene therapies have been achieved in recent years. However, final yields continue to be impacted by losses in downstream steps. Interferometric light microscopy (ILM) represents a fast and cost-effective method for process development teams to obtain size and concentration data within seconds. Learn more in this upcoming webinar with Cell & Gene Therapy Insights presented by Benjamin Josey: http://ms.spr.ly/6045WHOvb

Join Goodwin's Michael Shuster, Joshua Weinger, PhD, and Sarah Stoiber for the Cell + Gene Therapies Webinar: IP Due Diligence, to explore the unique IP issues faced by cell and gene therapies companies. This webinar will discuss: 🔹 Navigating patentability, freedom-to-operate, and managing third-party risks 🔹 Best practices in conducting thorough and strategic IP reviews at important inflection points of a company's growth stages 🔹 Practical examples and lessons learned from working with leading companies in the life sciences sector Register here: https://bit.ly/3TpAl5f #IntellectualProperty #LifeSciences

Little Journey partnered with a global pharmaceutical company to tailor our support solution for children with Duchenne Muscular Dystrophy (DMD) participating in gene therapy research. Our newest case study is out now, ‘Preparing and supporting children undergoing gene therapy’ 🔎 Read about the background, the challenges and our solution here 👉 https://lnkd.in/eR4d6Ezu #GeneTherapy #ClinicalTrials #DMD #CaseStudy

Revolutionizing AVV gene therapy production with cutting-edge mass photometry technology! 🚀 Discover how Rentschler Biopharma and its partners are setting new standards in quality and efficiency. #GeneTherapy #AAV #Innovation

2025 Outlook For CGT: Focus On Non-Oncology Indications, In Vivo Gene Editing, And POC https://lnkd.in/e8hy3u49 - By Erin Harris, Editor-In-Chief, Cell & Gene  The cell and gene therapy sector is poised for significant advancements in 2025 driven by three key trends.

With the current downturn and significant layoffs across the biopharma sector, outsourcing has become a vital component in ensuring future success for these impacted organizations. The CDMO space is incredibly rich in both volume of opportunities but also competition. Now more than ever these companies must innovate to differentiate… derisking sponsor projects through novel analytics and methods that deliver consistent results with increased speed and quality. CDMOs have a very unique opportunity right now to be the leaders and experts that hold the know how in accelerating significant advancements across complex development and production processes.

Please add CellReady™ to the CGT list. CellReady™ is the only CGT CDMO that is solely dedicated to G-Rex-centric manufacturing. We also believe that CellReady™ is the only CGT CDMO that has an ownership structure that is absent venture funding or any other form of ownership structure that imposes a need to generate significant financial profit. Why is this important? It frees CellReady™ to provide CGT entities what they need most and have least. TIME and MONEY How is that possible? CellReady™ is playing the Infinite Game and has a mindset of abundance. It is willing and able to run close to breakeven with minimal profit. No quarterly financial targets, no need to drive towards a lucrative financial exit, no need to do anything that makes its clients become viewed as a source of money. Where does this lead? CellReady™ is likely the only CDMO with the sole goal of openly sharing its operational and technical approaches to manufacturing efficiency with the entire field of CGT, including competitive CDMOs. By doing that, CellReady™ intends to revolutionize the business model of CGT by democratizing the information that sets it apart from others. That will help set the field free of the existing time and money trap which has caused investment to dry up. With a mindset of abundance, one initiative that CellReady™ is helping to fund is a $20 million G-Rex Grant Program available at ScaleReady which provides every recipient with up to $300,000 of free products and services to accelerate their clinical enabling manufacturing work or to make manufacturing advancements for programs that are already in the clinical. Please check it out.