Gene Therapy Process Development & Manufacturing Support

Element’s consultative team of biopharmaceutical scientists have extensive experience supporting gene therapy programs for both IND and BLA regulatory pathways.

Not many therapeutic modalities can match the promise of gene therapies. Recently, life-changing results have been produced by gene therapies in late-stage trials, and the number of gene therapies approved by the US Food and Drug Administration (FDA) continues to increase. Yet, while the industry faces mounting pressure to reduce cost-per-dose, a critical need for many more indications persists.

Analytical testing is crucial to optimizing gene therapy development. Biopharmaceutical companies developing gene therapies need to identify a scientific partner that can meet the highest standards of safety and reliability, while meeting aggressive commercialization timelines. The ability of a contract research organization (CRO) and contract development and manufacturing organization (CDMO) to meet and accelerate commercialization timelines in support of one-of-a-kind regulatory paths with robust data collection and documentation is critical. This is particularly significant in an era of one-time functional cures, where competition within rare disease communities is common. Determine the optimal development path forward, taking into account both strategy and commitment, ensuring a speedy gene-to-clinic strategy, informed by solid and robust data delivered by Element’s experts.

Element’s consultative teams of scientific and regulatory experts understand that diverse projects have unique needs. Our flexibility, in combination with our deep knowledge base, allows us to customize our gene therapy analytical methods to suite the specific needs of each project. Our QC-centric mindset informs optimized method development, allowing us to identify and overcome problems early and leapfrog challenges before they arise. Manufacturing the doses required for a therapeutic effect is exceptionally expensive, and efficiencies throughout gene therapy process development can significantly reduce costs and increase the program’s viability. Realize significant cost and time savings as workflows begin to scale, by means of optimized and accelerated processes.

Element works with our partners to determine what assays, methods, and instruments will best help them reach their goals. We use a wide range of orthogonal analytical methods and capabilities to ensure the safety, identity, quality, purity, and strength of gene therapies, including a mammalian cell culture facility, capillary electrophoresis, stability chambers with custom settings, and chromatography and mass spectrometry, including Quadrupole Time of Flight (QTOF) and QExactive systems. Our comprehensive services support the entire product life cycle, from discovery to gene therapy process development, manufacturing, and beyond.

Gene therapy analytical methods and services

• Determination of empty/full capsid by UPLC
• Determination of vector genome and total viral particle titer by SDS-PAGE or capillary gel electrophoresis
• Determination of viral vector purity
• Method development for potency and transduction
• Peptide mapping
• Residual polyethylenimine, iodixanol, benzonase, and host cell protein detection

Ensuring safety in gene therapy process development

Unquestionably, one of the biggest challenges to the clinical success of gene therapies remains patient safety. With more than 1,000 clinical studies now underway, targeting a range of ailments, including cancer and monogenic diseases like 𝛽-thalassemia and sickle cell anemia, patient safety must remain at the center of all development activities. Thus, while an efficient in vivo or ex vivo vector-strategy is important for successful genetic transfer, these considerations must come second to patient safety.

A careful analysis of viral vectors and transduction methods remains vital. Element understands that accurate viral vector analysis, such as with adeno-associated viruses (AAVs), and development are essential to this process. We also understand that scientific confidence goes a long way towards ensuring gene therapies can hit their mark and cure difficult diseases. Our blend of deep scientific expertise, regulatory knowledge, and industry experience allows us to design custom gene therapy analytical methods and CMC strategies specific to the needs of each project while meeting the highest standards of safety and reliability.

The Element advantage

Element offers comprehensive, phase appropriate GMP services to support the development and manufacturing of leading-edge gene therapies, supporting both IND and BLA regulatory submissions. Leverage the deep regulatory expertise of our biologic experts and purpose-built labs to advance your gene therapy today.

For more information about Element’s advanced gene therapy services or to request a quote, contact us today.