Preclinical

With systemic toxicity challenges plaguing the AAV field, low-dose CNS indications are an ideal target for gene therapy. The plethora of neurological indications dominating gene therapy pipelines vary from rare neurological disorders to more common diseases including Parkinson’s, epilepsy and neurodevelopmental disorders. As programs advance globally, the 3rd Annual Gene Therapy for Neurological Disorders Europe meeting is the field’s definitive forum to showcase the European landscape’s progress and specific regulatory challenges. Gene therapy developers from all of the world, including Europe’s brightest minds in CNS gene therapy, will unite in Paris to share their research progress and host an industry-dedicated discussion.

In the context of unprecedented gene therapy investment and clinical progress, with mounting regulatory scrutiny, Gene Therapy Analytical Development Europe will unite innovative biotech and pharma organisations to develop robust analytical tools to guarantee the consistency, quality, and safety of gene therapy products Whether you’re looking for specific characterisation, potency or infectivity approaches to improve your current analytical toolbox or if you’re seeking to understand how gene therapy analytics differ from other biologics you’ve worked on in the past, this focused, technical event will provide insights specifically on the unique analytical challenges posed by gene therapy vectors, delivered by ‘boots on the ground’ scientists grappling with these challenges first-hand. Join 150+ analytical pioneers to discover the realities of working with tools like dPCR, AUC and HPLC, as well as how and where these tools can be most effective in process development, and what the European regulators expect in order to grant approval. Whether you’re working with AAV or lentiviral vectors, this is your opportunity to enhance your existing analytical methods and explore innovative new tools to support safe and effective gene therapy development.

The Gene Therapy for Muscular Disorders Summit took place in April this year, and is the field’s only industry-focused event specifically designed to help gene therapy developers successfully target the muscle. With a disease-agnostic agenda, but highlighting novel research in Duchenne’s (DMD), Limb-Girdle (LGMD), Pompe Disease and X-Linked Myotubular Myopathy (XLMTM), the expert industry speakers from the likes of Solid Biosciences, Sarepta Therapeutics, Astellas Gene Therapies and Pfizer spent 4-days discussing the fields hottest topics from navigating toxicity to state-of-the-art muscular targeting.

As drug developers are increasingly prioritizing their later stage assets and bringing them through to approval, the Gene Therapy
Comparability Summit will unite large pharma and innovative biotechs to ensure you can establish comparability at every stage of development, supporting a seamless transition to the larger scales required for commercialization. Focused specifically on enhancing gene therapy comparability approaches, this event will ensure you can maintain safety, quality, consistency and efficacy while adapting manufacturing processes, methods and platforms, to ensure you satisfy regulatory demands and avoid costly delays to clinical programs.

In the face of evolving regulatory guidelines and mounting scrutiny, Gene Therapy Analytical Development Europe Summit will discuss ways you can apply contemporary analytical tools to guarantee the consistency, quality, and safety of your gene therapy products. We will join leading CMC, regulatory, QC, analytical and process development experts to gain access to novel data and recent case studies by industry professionals from AstraZeneca, the MHRA, Novartis, Sarepta Therapeutics, Roche, Sanofi, and many more.

Whether you are focusing on specific characterization methods, enhancing your genome sequencing, advancing your understanding of full and partial particles, or advancing your early-stage bioassays, with 4 tracks, 8 pre-conference workshops and a post-conference focus day, the 4th Gene Therapy Analytical Development Summit will encompass all aspects of analytical development, giving you the chance to address and overcome challenges. If you work in quality control, quality assurance, or process development – we’ve listened and we’ve answered. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Talks include enhancing the knowledge transfer between departments, bridging between analytical methods with regards to QC/PD, and enhancing in-process development support. Whether you’re working with AAV, non-viral vectors or lentiviral vectors, this is your opportunity to enhance your existing analytical methods and explore innovative new tools to support safe and effective gene therapy development.

As newly published data show the first Gamma Delta T cell therapy’s clinical efficacy in patients, the race to market for the first allogeneic and solid tumor-targeting γδT has begun. Join us at the 3rd Gamma Delta T Therapies Summit alongside 120+ of the world’s leaders in the field to discuss discovery to manufacturing and clinical development of these ‘holy grail’ therapies in allogeneic and solid tumor settings. Developed with key opinion leaders such as Adrian Hayday, and experts from Adicet Bio, Lava Therapeutics, IN8 Bio, and many more, attend this meeting to engage in interactive discussions and gain the actionable insights to advance bench to commercial bedside development of γδT-based therapies. Gather in person for the very first time with industry pioneers harnessing GDT cells to bring to market more cytotoxic, clinically safe therapies for solid tumors. From modulating cell activity through genetic engineering and antibody engagement to enhancing the functionality of GDT cells through an improved understanding of their intrinsic biology, this is a conversation you won’t want to miss.

Over the past couple of years, the engineered biology market has seen fledgling companies rise to IPO smashing power players, as the demand for high-quality, engineered biomolecules and controllable therapeutics grows exponentially. The therapeutic potential of novel synthetic units is huge, but challenges remain in demonstrating proof-of-concept, developing assays, functional characterization, manufacturing scale-up, and identifying therapeutic applications for commercial development. as the Synthetic Biology-based Therapeutics Summit) is the leading industry-defining forum made exclusively for leaders in the synthetic and engineered biology space, focusing on the technical challenges that lie ahead in the path towards commercialization.Learn from industry trailblazers at Arsenal Biosciences, bit.bio, MIT, Novome Bio, Strand Therapeutics, Synlogic and more, to support your efforts in developing safe and effective synthetic biology-based therapeutics.  

Recognised with a Nobel Prize in 2020, CRISPR has opened the floodgates for a new era of gene editing therapies, promising to transform genetic disease treatments. With a well-established early field of genomics research, drug developers and researchers have been busy establishing the next-generation CRISPR toolkit, including Cas nucleases, base and prime editing technologies, and innovative delivery platforms. The CRISPR 2.0 Summit is devoted to showcasing only the latest and greatest innovations by uncovering how the next generation of CRISPR tools are radically enhancing efficacy, minimizing off-target effects and enabling efficient tissue-specific delivery to help you progress your program into and through the clinic.

From the earliest stages of characterization to the final steps of preparing for release and commercialization, our diverse program provides a comprehensive and unparalleled journey through end-to-end potency assay considerations. With a focus on practical insights, this summit equips you with the knowledge and expertise to ensure you’re prepared to excel in your role, which ultimately will bring better therapies to patients faster.