This presentation will outline the quality frameworks for therapeutic oligonucleotides and peptides, emphasizing how public standards strengthen CMC development and manufacturing. Challenges in impurity control, starting material characterization, and analytical consistency across product lifecycles will be discussed. Updates will also be provided on new and upcoming USP documentary and physical reference standards, including phosphoramidite, peptide, and impurity reference materials, that provide robust tools to help support regulatory expectations, enhance process control, and advance reliable, high-quality manufacturing.

This presentation explores the unique challenges in CMC development for oligonucleotide drug products, including navigating ATMP manufacturing licenses, optimizing specialized routes of administration, and managing high formulation viscosity. Practical strategies to streamline development, ensure regulatory compliance, and enable patient-centric delivery will be discussed. Insights also highlight common pitfalls and offer guidance for efficient oligonucleotide product development and manufacturing.

In vitro transcription (IVT) for mRNA manufacturing lacks a validated, macroscopic rate law, limiting rational process design and driving costly empirical optimization. The field's benchmark model assumes IVT is always initiation-limited, resting on an aphysical elongation rate constant. We show IVT can be initiation- or elongation-limited across clinically relevant mRNA sequences, depending on DNA:polymerase ratio, with regime-dependent salt sensitivity. Extending to dsRNA formation, data reject an RNA-templated mechanism, yielding a quantitative framework for process and impurity control.

The manufacturing of Antibody-Oligonucleotide-Conjugates (AOCs) has emerged as a critical area of innovation in the development of targeted therapeutics and diagnostic tools. This novel approach is aimed at improving the efficiency, specificity, and scalability of conjugation strategies, addressing key challenges such as site-selective modification, linker stability, and product homogeneity by integrating advanced bioconjugation chemistries, optimized purification protocols, and automation-friendly workflows.

RNA interference has emerged as a powerful modality for modulating gene expression and addressing diseases with limited therapeutic options. This presentation will highlight advances in our clinical programs that utilises RNAi platform technologies designed to improve delivery, stability, and target engagement across multiple disease areas. Emphasis will also be placed on the process development, manufacturing, and analytical considerations required to translate the siRNA candidates from discovery through clinical development, supporting scalable production and consistent product quality.

This presentation will explore considerations in designing GMP facilities for oligonucleotide drug substance manufacturing. This will be seen through the lens of automation, solvent usage, and regulatory compliance. Drawing on industry benchmarks and project experience, the talk will discuss how capital facility cost is derived from these elements and highlight the future impact to oligonucleotide manufacturing facilities through emerging technologies such as enzymatic ligation and de novo enzymatic synthesis.

Many genetic diseases result from insufficient protein expression. CAMP4 is developing antisense oligonucleotides (ASOs) designed to upregulate gene expression and restore healthy protein levels using insights from the RAP Platform. This presentation will highlight the discovery strategy alongside key CMC considerations, including oligonucleotide design, manufacturability, analytical characterization, and quality attributes required to advance ASO therapeutics that increase gene expression into clinical development.

ASOs with unique n-methylacetamide (NMA)-modified chemical backbones can potentially improve efficacy, allowing patients with long-interval dosing. However, NMA chemistry is not straightforward and comes with many challenges that are unique to this moiety. In this presentation, the challenges associated with NMA ASOs will be discussed along with the developmental work to optimize the process. Moreover, a next-generation starting material will be discussed for ASOs with NMA chemistry.

Endotoxins are glycolipids shed from gram-negative bacterial membranes. Regulatory agencies require stringent control of endotoxins in drug substance and product with tighter control for intrathecally-administered drugs. Conventional endotoxin removal relies on positively charged filters that bind negatively charged endotoxins. However, these approaches are incompatible with highly anionic antisense oligonucleotides, causing yield loss. Here, we demonstrate a hydrophobic interaction–based separation strategy that removes endotoxins from ASOs while preserving high product recovery.

In 2025, Baby KJ became the world's first patient to receive personalized CRISPR therapy in just six months. The critical next step is translating this breakthrough into scalable, cost-effective standard care for thousands with rare genetic diseases. This presentation explores the strategic and technical foundations for building scalable gene editing platforms, examines regulatory innovations that enable rapid deployment, and discusses how industry is reshaping the economics of personalized CRISPR therapy.

• Discuss the unique CMC challenges associated with oligonucleotide therapeutics and emerging conjugated modalities, including antibody oligonucleotide conjugates
• Explore critical considerations for synthesis, conjugation, purification, and analytical characterization
• Highlight strategies to control product quality, impurity profiles, potency, stability, and batch consistency
• Examine how evolving regulatory expectations are shaping CMC development for increasingly complex oligonucleotide-based therapies
  

This presentation explores longevity and lifestyle medicines as a growing market, focusing on challenges in large-scale biopharmaceutical manufacturing and cost-reduction strategies. It highlights advances in peptide bioprocessing and beyond, examining whether these innovations can meet rising market demands for longevity biopharmaceuticals. Emphasis is placed on optimizing production efficiency and scalability to align with consumer expectations for effective, accessible, lifestyle and longevity therapies.

Manufacturing disulfide-bonded peptides in Escherichia coli offers a scalable and sustainable alternative to chemical synthesis but remains limited by proteolysis, complex folding requirements, and peptide-specific downstream development. CASPON technology provides a platform solution enabling high-titre soluble expression, affinity capture, and precise, scar-free tag removal suitable for biopharmaceutical production. By integrating outer membrane engineering, controlled peptide release into the culture medium further simplifies recovery and reduces downstream processing complexity. This combined approach minimizes process customization, shortens development timelines, and enables robust, scalable manufacturing of high-quality disulfide-bonded peptides for industrial applications.

Today’s peptide therapeutics increasingly rely on chemistries beyond the 20 canonical amino acids to achieve exceptional pharmacological functions. Their manufacture relies on chemical methods, generating on average 13,000 kg of waste per kg of peptide, of which >50% is organic solvents. This talk will demonstrate how genetic code expansion enables the scalable biomanufacture of peptides and proteins that incorporate multiple non-canonical amino acids, in a reprogrammed E. coli host.

Peptide co-formulation represents a strategic approach to simultaneously modulating complementary biological pathways while reducing injection burden for patients. Successful development requires an understanding of interactions of the co-formulated products to identify potential challenges in the formulation development. This presentation focuses on the thought process of studying peptide interactions in co-formulations using biophysical techniques.