Advanced Therapies 2022

Poster Sessions

Advanced Therapies 2022


1. Painting a Digital Landscape, Digital Lab Consulting
Digital Lab Consulting have supported the digital transformation journey of cell therapy companies to unleash the power of their data to drive projects forward.
Using our Digital Blueprint assessment, DLC were able to identify and prioritise key areas to improve data integrity and compliance; improve data management and traceability; and facilitate data sharing and collaboration.
Understanding the current pain points across businesses allows us to recommend where to improve a company's digital landscape, including a digital roadmap of capabilities to meet urgent needs.
With a clear vision, DLC were able to support businesses identify a solution through a technology selection process using our proprietary vendor database and market expertise to allow cell therapy companies identify the best fit for their requirements.
Implementation of a laboratory informatics system is the start of a digital transformation process to leverage the power of data!
2. Identification of genetic signatures in bone metastasis of breast and prostate cancer, OncoBone Therapeutics
Bone metastases (BM) cause high mortality and are present in 70-90% of advanced breast and prostate cancer patients. Cancer is a complex genetic disease, and no single gene has been shown to be solely responsible for the initiation, growth or progression of cancer. Therefore, more complete understanding of the multiple genes responsible for disease progression, and more specifically, for the development of BM is needed to support development of diagnostic tools and novel therapeutics for BM. The aim of this study was to identify genetic signatures specific for BM in solid tumors, more specifically in breast cancer (BC) and prostate cancer (PC).
3. Digital Bioprocessing: Efficient ways to combine Process Knowledge and Process Data to accelerate ATMP development, Novasign GmbH
Generating valuable and sufficient process understanding for ATMP development in the QbD concept is a cumbersome task. Hybrid modeling and advanced design space screening methods reduce the required experimental effort and the overall development timelines. Conserving process understanding and adaptive learning enables model transfer from early-stage development until manufacturing.
The poster will show the highlights of model-assisted process characterization, process optimization, implementation of soft-sensors ranging from microbial to complex ATMP processes and should spark a discussion of new concepts to advance the development of current ATMP bioprocesses. Finally, the modeling results of our modeling toolbox, which was used for several showcases, and the incorporated Digital Twins will be shown live.
4.Upgrading from cleanroom to isolators: tips and tricks for a smooth transition in ATMP applications, COMECER
Advanced therapy medicinal products (ATMPs) pose specific manufacturing challenges beyond those typically addressed by pharmaceutical chemistry. Often in current ATMPs applications, a change in approach is introduced at some point in the development process out of convenience or necessity, which then results in a change in technology. This work analyzes the possibility of transferring a cell and gene therapy (C>) process from the cleanroom approach to an environment based on isolation technology or, in other words, of moving the process from an open space manipulation to a closed and segregated space concept, considering benefits and mitigating risks for the most flawless transition.
5.Improving woman health with vaginal microbiome analyses, Yoni Solutions
Yoni Solutions is a Healthcare Company exploiting the untapped potential of vaginal microbiome to improve the treatment of recurrent vaginal infections and the outcome of In Vitro Fertilisation procedures after multiple failures.
6.Co-Stimulatory CAR-T Approach In γδ T Cells: Avoiding The On-target Off-Tumour Toxicity: Avoiding The On-target Off-tumour Toxicity, TC BioPharm
γ9δ2 T cells are capable of effectively killing unhealthy cells (via the isopentenyl pyrophosphate/butyrophilin – IPP/BTN – signalling axis) in an MHC-unrestricted manner (unlike αβ T cells), highlighting their unique safety profile in the treatment of cancer and infectious disease, in an allogeneic setting. This study assesses the potential of γδ T cells equipped with novel CAR designs using the so-called co-stimulatory approach ('costim'), to improve potency against unhealthy target cells, whilst displaying safety towards healthy cells expressing the CAR target antigen (avoiding on-target off-tumour related toxicity).
7. Traceable Impedance-Based Dispensing and Cloning of Living Single Cells, SEED Biosciences
Single-cell cloning is essential in stem cell biology, cancer research, and biotechnology. Regulatory agencies now require an indisputable proof of clonality that current technologies do not readily provide. Here, we report a one-step cloning method using an engineered pipet combined with an impedance-based sensing tip. This technology permits the efficient and traceable isolation of living cells, stem cells, and cancer stem cells that can be individually expanded in culture and transplanted.
8.Cellular phenotyping facility “The Stem Cell Hotel” Projects & Science Translation, Stem Cell Hotel - KCL
The “Stem Cell Hotel” is a centre of expertise for cell characterisation using high throughput imaging and high content analysis of cellular models. It’s a collaborative phenotyping space with users across academia as well as from the industry sector. The stem cell hotel provides guidance, expert advice and bespoke services to users, including assay development and assistance in overcoming technical challenges.  It advises researchers in the best practice of sample preparation for imaging, imaging expertise, analysis pipeline design and quantification of the results. The centre contains cell culture facilities that allow the preparation of samples in situ, minimising costs of logistics. Additionally, the range of instruments available allows not only imaging of fixed cells, but also in real-time, within an incubator or with temperature and CO2 settings within the dedicated high throughput microscope.
With five chimeric antigen receptor (CAR) T cell immunotherapy products currently approved by the FDA for the treatment of hematological cancers, there are increasing efforts to develop novel and better manufacturing technologies and processes for cell therapies to improve efficacy, reduce variability, and reduce cost. Knowledge in the field about how to optimize cell expansion for consistent and reproducible cell-based treatments is improving, but major challenges still exist in experiment reproducibility and robustness during process development. To address this gap, Erbi Biosystems has developed the Breez™ True Perfusion™ bioreactor. This fully closed sterile single-use perfusion bioreactor operates at a 2 mL working volume and can operate outside of a biosafety cabinet to replicate bench scale perfusion processes to industrially relevant cell densities in excess of 100e6 cells/mL. The advanced microfluidics, including bubble free mixing, on-line cell density control, and automated dO/pH control allow the Breez™ to achieve superb cell growth in a 2 mL scale, reducing labor and bench space required. 
10. Pin-pointTM: A Versatile Editing Platform Driving Cell Therapies, Horizon Discovery, a PerkinElmer Company
Horizon’s modular Pin-point base editing system efficiently and precisely converts target nucleotides in the genome by recruiting DNA modifying deaminases via an aptamer encoded in the guide RNA (gRNA) of the sequence-targeting Cas component. We optimized design and delivery conditions of synthetic gRNAs and mRNA to apply multiplex base editing to the development of engineered CAR-T cells. We targeted a set of therapeutically relevant genes (B2M, CD52, TRAC and PDCD1) using APOBEC1 combined with Cas9 nickase and achieved greater than 70% knockout efficiency and high purity at all sites simultaneously. Engineered CAR-T cells retain their proliferative and cytotoxic activity in vitro, and, compared to Cas9, multiplex gene knockout with Pin-point is associated with less gRNA-dependent off-target editing and negligible occurrence of chromosomal translocations.
11.Next-Generation T cell Activation and Expansion, Nanotein Technologies
Cellular therapies are the cancer treatment of the future. These therapeutics work by efficiently seeking cancer cells for targeted destruction, while leaving healthy cells intact. CAR-T cells are the most clinically and commercially advanced of anti-cancer cell therapies. While incredibly effective, they have faced multiple challenges including manufacturing failures, long manufacturing times, and short-lived or poor treatment outcomes. Nanotein’s novel platform technology can help overcome these challenges by accelerating the speed of CAR-T expansions, enhancing expansion to grow more cells per treatment, and shifting the growth profile of CAR-T cultures to favor the most long-lived and clinically effective CAR-T cell sub-populations, the stem-like central memory T cells (Tscm). Here we show that Nanotein’s two formulations STEM-T and APEX-T achieved enhanced expansion of total live T cells, CD8+ Tscm cells, and CD4+ Tscm cells during a 10-day expansion of donor peripheral blood T cells.
12.Tissue specific gRNAs for increased safety in in-vivo CRISPR/Cas gene-editing, Helex Inc.
One of the biggest challenges posed due to CRISPR/Cas gene-editing mechanisms, particularly in therapeutic settings, is the off-target effects both within the genome and in unintended tissues in case of in-vivo editing. Helex’s proprietary Hele-GUIDE platform enables tissue-specific CRISPR/Cas gene-editing. The Hele-GUIDE platform leverages core cell and tissue specific markers leveraging epigenetics to design unique, tissue-specific guide RNAs (gRNAs) that prevent editing in unwanted tissues to enhance safety. We have experimented on liver-specific editing by identifying unique liver-specific markers, and designed gRNAs accordingly. We compared the edit efficiency of the tissue-specific gRNAs in target hepatocellular carcinoma (HCC) cells versus non-target immortalized lung and colon cells. The results displayed higher editing in liver cells in comparison to non-target cells when tissue specific markers were considered while similar edit efficiencies were obtained across cell lines for targets where tissue specific markers were not considered. This shows promise for leveraging epigenetics and crucial cell specific markers for enabling tissue specific editing. This work is critical for enabling higher safety, and acts as a doubly padded layer of protection in case of leaky delivery for in vivo gene editing.
13. Analytical Performance of the iCS-digitalTM PSC test, Stem Genomics

The iCS-digitalTM PSC 24-probe test is a multiplexed digital PCR assay for the identification of the most recurrent Copy Number Variants (CNVs) occurring in cultured human Pluripotent Stem Cells (hPSC)1-2. This test is based on digital PCR technology which enables sequence-specific detection and absolute quantification of nucleic acids. Use of the iCS-digitalTM PSC test for routine testing purposes requires validation to ensure proper quality control of hPSC genome integrity. Herein, we present the results of an analytical performance study of the iCS-digitalTM PSC test.



Get involved with Advanced Therapies Congress


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Jessica Robinson
+44 208 164 3086



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Ashlea Foster
+44 208 164 3032