Cell therapies are driving clinically meaningful benefit in some haematological malignancies. Extending this success to solid tumors has proved challenging, in part due to the complex and hostile tumor microenvironment (TME) that limits the activity of cell therapies.

This presentation will provide insight into AstraZeneca’s approach to overcome these challenges, including our novel preclinical framework and innovative strategies to ‘armour’ cell therapies to resist immune-suppression in the TME. By advancing a broad portfolio of CAR-Ts and TCR-Ts, we are unlocking a breadth of intra- and extra-cellular targets in cancer treatment, and I will share preclinical and clinical data demonstrating their early promise in hard-to-treat solid cancers. Data will also be presented showing the exciting potential of a dual-targeting CAR-T in haematology.

Looking to the future, there are still many challenges that prevent the widespread adoption of cell therapy and I will share AstraZeneca’s vision for overcoming this, with the goal of bringing cell therapy to more patients globally.

• The forgotten millions: overview of the immense unmet need facing the rare disease community.
• How investment fuels recent breakthroughs, especially within cell & gene therapy
• The necessity of partnerships between researchers, investors, the industry, and patient advocacy groups to transform healthcare for rare diseases.

• How to effectively move through the ‘valley of death’ between therapeutic discovery and commercial readiness
• Transitioning product into industry: preparation and education to prevent bottlenecks
• Strategies to travel through the middle ground between small scale production and commercial need, to meet patient needs
• The power of partnerships, preparation, and regulatory readiness: when to build own GMP space, when to rent or use CDMO?

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

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NextCell Pharma is a clinical cell therapy company working in the autoimmune and inflammatory disease space. Their lead drug candidate, ProTrans is comprised of umbilical cord mesenchymal stromal cells (UC-MSC) selected using the company’s proprietary selection algorithm and pooled to generate the cryopreserved therapy. ProTrans has been used in Phase I and II trials for the treatment of type 1 diabetes and in severe lung hyperinflammation due to viral pneumonia.

Dr Davies is the Chief Scientific Officer for NextCell Pharma and will present the company’s current clinical portfolio for type 1 diabetes. The company has conducted a Phase I open label dose-escalation study with a primary endpoint of safety, as well as a Phase II double-blinded, randomised and placebo-controlled study with a primary endpoint of Δ-change in C-peptide area under the curve for a mixed meal tolerance test at 1 year following ProTrans/placebo infusion compared with baseline performance prior to treatment.

Dr Davies will share data demonstrating the efficacy of ProTrans treatment on preserving endogenous insulin production in newly diagnosed type 1 diabetes patients and the company’s long-term follow-up indicating that a single infusion of ProTrans results in a disease altering effect, with a slowing of disease progression for >3.5 years.

The company is now participating in a Phase II paediatric trial for the treatment of newly diagnosed diabetes in individuals aged 7-21. An update on the current trial status and vision for the future will be discussed as part of this presentation.

Practical solutions to achieve a 'get it right the first time' bioprocess, from the experience of running a multi-national, multi-product clinical cell therapy manufacturing portfolio. Early drug development decisions are critical to the commercialisation pathway, and understanding the healthcare landscape, treatment setting and infrastructure are necessary to design a competent CMC strategy for growth. However, drug manufacturing to meet the scale and agility necessary to support clinic-driven development is highly challenging. Real-world solutions to starting material selection, and technology intensification for downstream and fill finish solutions will be discussed to elucidate on building a successful CMC production strategy to meet patient demands and global supply. Manufacturing solutions to support early-through-late stage cell therapy clinical studies are not readily available, with a technological chasm occurring between early-phase 'academic' solutions and commercial-ready high capital unit operations. Here, practical solutions have been derived, and are in clinical GMP operations, for an off-the-shelf cell therapy portfolio to highlight development pathways for novel therapies within a small biotech company. Specifically, solutions to address batch-to-batch variability, to support patient demand and global supply, and to present a novel fill/finish scalable solution will be shown: all designed to bridge the technological chasm and provide bioprocessing throughout clinical development and beyond. Clinical success can be streamlined through value-driven early CMC decisions, with choices led by patient and clinic-led decisions. Drug development must be supported by a robust and agile bioprocess, with the solutions presented here aiming to bridge clinical phase production strategies.

• What steps can be taken during earlier development to prepare for commercialization?
  • Formulation to ensure scalability
  • Process development for reproducibility
• The importance of a robust supply chain for advanced therapies
• Fortifying the supply chain, ensuring reliable stakeholders and suppliers, efficiency for production
• Effective learning around implementation of new tools

Cytokine Release Syndrome (CRS) is a potentially life-threatening side effect associated with many modern immunotherapies such as CAR T cell therapy and T cell engaging bispecific antibodies. Despite incidence rates of up to 95% and significant escalation of medical costs very few treatment options are available, with only tociluzumab approved for CAR T cell induced CRS within the US. No preventative methods exist despite significant physician and patient needs. A range of investigational and repurposed drugs have been evaluated but have failed to produce sufficient reductions in CRS levels to impact patient management plans. A number of MAPK inhibitors have now been shown to enhance cell therapies and offer an ideal method to attenuate hypercytokinaemia associated with CRS without impairing immunotherapy efficacy. POLB 001 is an oral p38 MAPK inhibitor in development for the prevention of CRS. It has been evaluated in a Phase 1b LPS challenge study showing potency in dramatically reducing IL-6, TNFa, IL-8 and other proinflammatory cytokines along with meaningful reductions in heart rate, temperature and C-reactive protein increases. Phase 2 trials are planned with the intent to enable broader and safer uptake of immunotherapies in the clinic.

The advancement of allogeneic therapies presents a promising frontier in regenerative medicine, offering the potential for scalable, off-the-shelf treatments. However, the development of these therapies faces significant challenges, including the complexity of manufacturing processes and the need for consistent product quality. This abstract outline an approach to streamline allogeneic product development through the integration of platform automation in both 2D and 3D cell culture systems.

By leveraging automated platforms, Bayer aims to industrialize the efficiency and reproducibility of allogeneic cell production. Automation technology facilitates control over the cell culture environment, ensuring consistent cell quality and yield. In 2D systems, automation supports the standardization of monolayer cell cultures, while in 3D systems, it enables the scalable production of clusters or spheroids, which more closely mimic the in vivo cell environment.

The implementation of platform automation in allogeneic product development not only accelerates the production process but also reduces the risk of human error and variability. This approach promises to lower the barriers to entry for allogeneic therapies, making these innovative treatments more accessible to patients in need.

Whether you're increasing your company profile, launching a new product or focusing on new business development opportunities, collaborate with us to identify custom solutions to help you reach your goals.

Contact us today to learn more: Partners@informaconnectls.com

• Algorithms for optimization – how can AI predict optimal conditions to maximize yield and quality?
• Predictive analytics for cell growth, differentiation, and impurity forecasts to enhance process control
• Data-driven decision making
• How data analytics can help with heterogeneity of patient cells

• Compared to usual biologics, CGT processes are inherently variable in its starting material.
• This leads to batch to batch variability. Hence, we need tools to assess the input material uncertainty and develop a compensatory control strategy to achieve constant product quality.
• In addition, especially for autologous processes, we have much less runs to develop or validate our process. Lot 1? Therefore we need to be very conscious of building up platform knowledge from other products and deploy this knowledge in a predictive manner.
• This contribution will tools how platform knowledge can be obtained and deployed to overcome input material uncertainty and predict product outcomes using digital twins. This includes Physics Informed AI.