Elliott Berger, Vice President of Global Marketing and Strategy, Catalent Pharma Solutions
A: In the world of drug delivery technologies, a year is really a blink in time. It usually takes well over a decade for a new technology to be adapted, be proven, and to make a difference in real medicines. Over the last couple of years however, Catalent has seen a couple of trends in this space. One has been the development of technologies that help improve the targeted delivery of more challenging molecules with more complex profiles. For example, 2016 has seen the first FDA approval of a non-animal derived softgel capsule with a sustained release profile. Targeted delivery of capsules with enhanced functional benefits could aid a broad array of products. Another trend is advances in in silico modeling, high-throughput screening and development techniques to shorten the process of identifying the preferred formulation and dose-form approaches for each individual molecule, ideally enhancing the targeted patients’ outcomes. Accelerated parallel screening approaches that quickly help overcome formulation challenges, such as Catalent’s newly expanded OptiForm® Solution Suite, can help innovators optimize their molecules and advance them to clinic faster, with better chances of success.
Paul Valero, Director, Process Technology/Associate, IPS-Integrated Project Services, LLC
A: The market for specialized injectable medicines continues to move toward high-value products with smaller batch sizes and less campaigning, including the capability to process personalized medicine. In 2017, drug manufacturers exhibited new qualified systems capable of highly flexible, efficient and compliant fill-finish technologies to meet these challenges.
On a small scale, compact robotic fillers within aseptic isolators enable safe delivery of just a few vials of a unique drug tailored to one person. At larger scales, flexible fillers allow manufacturers to process vials, syringes and cartridges, including lyophilized products, all on one footprint. In parallel to improving equipment capability, drug manufacturers and suppliers have partnered to increase the availability of ready-to-fill components to make flexibility possible. These collaborations not only provide competitive advantage for trailblazing market leaders, but their efforts also benefit the industry at large, which can immediately utilize these advances.
The trend of isolated filling systems to be more compact and affordable, a result of the above developments, affects the future of our industry. Capital-lean manufacturers historically delaying investment upgrades in legacy facilities with traditional clean rooms will have the opportunity to upgrade to state-of-the-art processes in smaller spaces at a lower cost. Upgrade of aging facilities will ultimately benefit patients.
Dr. Lorenz Mayr, Chief Technology Officer, GE Healthcare Lifesciences
A: During the course of year 2017, we have seen further refinement and successful implementation of a number of groundbreaking, innovative technologies in the global healthcare industry. Three technologies should be mentioned in particular:
a) Precise Genome Editing (PGE) with CRISPR/Cas9 technology
b) Novel therapeutic modalities with ASO and modRNA
c) Novel diagnostic tools with liquid biopsy/ctDNA
The CRISPR/Cas9 technology enables precise engineering of DNA in plants, animals and humans. The technology has been adapted at an unprecedented pace toward applications in basic and applied biomedical research, and is now used in more than 20 ongoing clinical trials. It is widely accepted that this technology will enable therapeutic intervention in previously non-druggable human diseases.
Significant progress has also been made in the development and clinical use of antisense oligonucleotides (ASO) and modified RNA (modRNA) as novel therapeutic modalities, hereby complementing the existing toolbox of small molecule chemical compounds and large molecule recombinant proteins. Recent progress in clinical trials based on the improved design, delivery and targeting of these agents seems to enable a cost-effective and safe alternative for the treatment of human disease.
The combination of next-generation sequencing (NGS) technologies with powerful bioinformatics tools for analysis (‘big data’) has enabled significant advances for blood-based diagnostics (‘liquid biopsy’). These technologies facilitate fast and reliable detection of circulating DNA (ctDNA) from tumor cells, boost clinical trials and enable numerous other blood-based technologies for genomic analysis.
Professor Tom Moody, Ph.D., VP Technology and Commercialization, Almac Sciences & Arran Chemical Company
A: Enzyme technology and its applications are developing at a significant pace and are becoming prevalent in many areas of drug development. At Almac we are applying enzyme technology to API development in many ways. We are also seeing increasing numbers of innovator companies with enzyme technology as a key differentiator.
Antibody-drug conjugates (ADCs) are innovative therapeutics benefiting from enzyme technology. We have been involved in the selective modification of antibodies and attachment of key linker-payload moieties for ADC development. Enzyme technology has the potential to be site specific and/or lower the losses of linker-payload needed to obtain the desired final product. We have also been applying [14C]-technology to ADC projects by synthesizing the linker or payload, or both, with the radioactive [14C]-center. We have used ultrasound-assisted flow apparatus to aid in fermentation production of recombinant peptides and proteins. Ultrasound technology can aid in the soluble expression of protein and also in the up-regulation of certain pathways for metabolite production. Applying this technology and utilizing the multidisciplinary team of radio chemists, analysts and biologists at Almac minimize time and cost for clients by eliminating the need for multiple vendors.
Marga Viñes, Business Development Manager, Contract Manufacturing, Grifols
A: Implementation of fully automated technologies (robotics) to manufacture injectable solutions becomes an imperative to minimize quality problems and risk of contamination.
Given the increasing number of recalls due to contamination by visible particulates in parenteral drugs and the heightened concern of the FDA and other regulatory agencies, manufacturing companies will pay more attention to automation in order to improve manufacturing operation and enhance its existing quality programs — resulting in more safety products for patients.