Drug Discovery and Development

The therapeutic potential of messenger RNA (mRNA) therapeutics has an unprecedented ability to target previously undruggable diseases. The poly(A) tail drives the therapeutic efficacy of the mRNA molecule. Using Droplet Digital PCR (ddPCR) technology to analyze poly(A) tail length and integrity yields unrivaled sensitivity, enabling developers to ensure high poly(A) tail integrity and, ultimately, functional therapeutic mRNAs.

In December 2022, a groundbreaking law was passed granting the U.S. FDA the authority to consider data from new alternative methods in the preclinical testing phase for new drug development. Among the new methods are 3D cell cultures that mimic human tissues and organs, known as microphysiological systems (MPS). The new law aims to improve clinical outcomes while decreasing reliance on animal testing. In this article, we discuss MPS and review data showing how they can synergize with Bio-Plex Assays.

Immunotherapy has revolutionized cancer treatment, yet the drug discovery process to develop new biotherapeutic antibodies remains arduous. However, significant advances in antibody technology such as phage display have enabled fully human antibodies to be developed from animal-free libraries for just about any antigen.

Inadequate quality control assays shouldn’t limit cell and gene therapy applications. Instead, quantifying the potency of cell and gene therapies is essential for successful therapeutics. In this article, we describe multiple leading potency assays developed using Droplet Digital PCR (ddPCR) technology, including an assay that quantifies transgene integration and expression in one step.

Cell and gene therapies have seen record growth due to their potential to address the underlying causes of genetic and acquired diseases. Non-viral delivery methods are increasing in popularity due to their safety, cost-effectiveness, reduction in manufacturing time, and flexibility compared to viral vector delivery methods. Electroporation is a simple, broadly applicable method capable of delivering molecules to a wide range of cell types for either cell or gene therapy. This article outlines the top advantages of using electroporation in cell and gene therapy and why it might be a good approach for your own research group.

Disparate systems, distributed teams, and overly complex technologies are a recipe for inefficiency in research and development (R&D) laboratories, especially in drug development. With the pressure to meet challenging deadlines and strict regulations, it is easy for working practices to become disconnected. To improve the efficiency, accuracy, and consistency of data generation, acquisition, and management, R&D organizations are looking to streamline their lab practices by investing in technologies that automate data generation, accelerate data collection speed and storage, and enhance the quality and reliability of the information produced. Lab automation presents exciting opportunities for future research, promising novel advances in machine learning and artificial intelligence (AI), liquid handling robotics, and cloud-based data workflows.

The CAR T-cell manufacturing process involves high potential for variability. Learn how sensitive ddPCR assays can be used to ensure safety and efficacy.

As more of the cell and gene therapies revolutionizing healthcare are approved for use, innovator companies usually get the credit. But many therapies would never make it to market without the expertise and capabilities of contract development and manufacturing organizations (CDMOs) operating behind the scenes. Learn more about how CDMOs enable new cell and gene therapies to come to market, and how Droplet Digital PCR can help.

Research into human diseases has led to the development of novel monoclonal antibody drugs. These monoclonal antibody therapies have been successful because of their specificity and selectivity. However, ongoing development and successfully launching a new mAb drug require robust and reliable methods to screen for drug targets, demonstrate drug safety and efficacy, and scale up manufacturing to meet the stringent regulatory guidelines required for approval.

While the FDA has approved Abecma, the first CAR-T cell therapy in its class, regulatory approval is just the beginning for cell and gene therapies. Such therapies’ safety and financial viability rest on a robust manufacturing and quality control program with the flexibility to scale up and out. Droplet Digital PCR is a sensitive biopharmaceutical quality control tool, offering absolute quantification of nucleic acids and other molecules in a sample.