What are the challenges in gene therapy development?

Gene therapy holds the promise to revolutionize medicine by providing cures for previously untreatable genetic disorders. However, the path from discovery to commercialization is full of unique challenges. We dive into some of the most-asked questions about challenges in gene therapy.

What is gene therapy and its potential advantages for treating diseases?

Gene therapy is an approach to treat or cure diseases caused by genetic aberrations through gene addition, gene modification, or gene expression alteration. Cells can be modified within the body (in vivo) or outside (ex vivo). Viral vectors are a popular choice for gene delivery as they can enter a cell efficiently to deliver the gene product. However, to improve the safety associated with this therapy, there is an increasing interest in non-viral approaches for gene delivery such as lipid nanoparticles (LNP), electroporation, etc.

The greatest advantage of gene therapy is that it can potentially treat or cure diseases at the root cause level—which is the DNA—and can be a one-time treatment, if successful. Several rare diseases that currently have no treatment options are particularly well suited for gene therapy as they are often genetic in nature.

What are the key challenges in gene therapy development?

There are several challenges in gene therapy development.

• During Research and Development, access to technologies for determining the optimal gene editing technology and the vehicle for delivering it are critical. In addition, establishing early processes that enable quality and scalable production of the gene editing machinery can smoothen the transition down the drug development journey.
• When transitioning to Process Development, it is important to establish reproducible methods that are reliable and scalable—robust technologies and analytical methods are key at this stage. In addition, it is important to consider the regulatory requirements at this stage to prepare for the clinical phase.
• During Clinical Trials the primary concerns focus on patient safety. This requires experience in executing gene therapy clinical trials and having access to tools that can pre-emptively assess risk to patients based on data.
• During commercialization and production of a gene therapy, the focus shifts to scalability and supply chain. Key factors that determine success in this phase are consistent manufacturing practices, a robust supply chain network, as well as integrated project management to help streamline manufacturing and drug delivery.

Download our infographic to learn more on how to navigate these challenges through practical solutions and help ensure a smoother journey from research to market.

How can you navigate regulations around gene therapies and successfully commercialize them?
Specialized regulatory support can help companies navigate regulations around gene therapy development to expedite the approval process while maintaining safety standards. This support can also foster collaboration between regulatory agencies, researchers, and industry stakeholders, creating a conducive environment for innovation.

Key considerations for gene therapy commercialization include:

• Managing compressed timelines
• Addressing assay challenges and variability
• Ensuring the quality of raw materials
• Handling limited batches at commercial scale

By focusing on these areas, risks can be mitigated to help ensure advancement of the gene therapy.

How can a CDMO and CRO help support gene therapy development?
Working with an CDMO or integrated CDMO and CRO experienced with advanced therapies can help streamline the drug development process because they have the background to navigate development and commercialization challenges, the resources to execute and manage timelines, and the capabilities that can support the journey from bench to market.

The Accelerator™ Drug Development by Thermo Fisher Scientific offers innovative 360° CDMO and CRO solutions designed to expedite the drug development journey and get treatments to patients faster. In an industry where developing novel therapies is high-risk and adding multiple vendors can increase complexity and delays, our fully integrated CDMO and CRO services provide seamless support throughout the entire process.

Download our infographic now to gain insights into overcoming common challenges and help accelerate your gene therapy development process.

More resources on gene therapy
Download our eBook or read more from the Thermo Fisher Scientific team on gene therapy development in clinical trials and commercialization of a gene therapy.