Novel Therapies

What truly novel therapies in the earliest stages of discovery and development do you believe will have a significant impact in the future?

Manufacturing Technology

What advances in manufacturing technology are most enabling the scale-up and commercialization of next-generation therapies?

Ethical Practice

Given the ongoing evolution of the pharma industry today, how does your organization incorporate ethical concerns into your business practices?

Artificial Intelligence

What do you believe the role of artificial intelligence and other advanced technologies will be in driving the development of the pharmaceutical industry over the next decade?

Innovative Technologies

What innovative technologies had the greatest impact on your company this year? What were the impacts?

Roundtable Topics

Novel Therapies

Manufacturing Technology

Ethical Practice

Artificial Intelligence

Innovative Technologies

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Drug discovery has traditionally been focused on small molecule drugs designed to bind directly to target proteins to decrease their activity. However, around 80% of protein targets are still very hard targets or even undruggable through this approach. A major and still underexploited area for drug discovery is mRNA. RNAi technologies have been developed to “knock down” mRNAs to prevent protein production. Recently, synthetic mRNA has been suggested as a drug to treat diseases where lack of protein is the problem, but it faces delivery problems and other issues that limit its widespread use. Small molecules targeting mRNA biology is one of the most promising areas for the discovery and development of new medicines. In this space, Anima’s Translation Control Therapeutics is the first and only platform for the discovery of small molecule drugs that control the translation of mRNA by ribosomes as a novel strategy against a wide range of hard and undruggable targets. Unlike other approaches that target the mRNA itself, we identify small molecules that can bind to the proteins that regulate translation. Through this approach, we can decrease or increase the production of almost any target protein, leading to drugs for the broadest range of diseases.

Yochi Slonim
Co-founder and CEO, Anima Biotech

There are few things as transformative to the pharmaceutical industry as the advent of biologics. Everything from monoclonal antibodies to cell and gene therapy technologies are transforming the way patients are treated and even fully cured. From a pharmaceutical formulator’s perspective, we continue to rely on older technology for administering these types of large molecule formulations — namely, as parenterals. The manufacturing and dosing of these formulations rely on excipients, surfactants and processing aids that are often out-of-date or not designed for large molecule manufacturing or dosing purposes. The cutting edge is where scientists aim to administer biologics using more patient-friendly means; for example, inside of an oral capsule or through the skin as a micro-needle patch. These are revolutionary ideas for administering large molecules, but they rely heavily on having tools, techniques and excipients to bring these ideas into reality. Often, in pharma, we wonder where the new molecules are. Well, the new molecules are here today, but they are fundamentally different and far more sensitive to external forces than their small molecule predecessors. Therefore, the innovation will come not just from the molecule itself, but also how it is formulated and administered to the patient.

Frank Romanski, Ph.D.
Global Technical Marketing Manager, Pharma Solutions, BASF Corporation

Currently, cell and gene therapies, along with advances in gene editing through CRISPR, offer impacts to patients that in many cases have not been effectively treated in the past, including rare disease patients. The broader impact and potential for these therapies is yet unrealized. Additionally, CAR-T therapies and their potential impact to treat cancer patients, as an example, with personalized therapies, has also not yet been fully realized.

Tiffani A. Manolis
Senior Director, Pharma Strategic Program, Agilent Technologies Inc.

One truly novel technology that could have significant impact on the lives of patients in the future is the use of RNA editing. Targeted RNA editing has the potential to directly correct the causes of genetic conditions. While therapies are still in the early stages of development, RNA editing potentially combines the benefits of both gene therapy and small molecule therapies. Like gene therapy and gene editing, RNA editing can specifically repair a disease-causing gene mutation but can do so without permanently changing a person's genetic code, reducing the chance of permanent off-target effects. Additionally, like small molecule drugs and unlike gene-editing therapies, small molecule RNA-editing therapies can often be administered in simple fashion and delivered without viral vectors. There are thousands of diseases caused by genetic mutations that can be corrected with RNA editing, and we have only just begun to witness the impact these types of therapies can have on the lives of people suffering from devastating genetic conditions.

Daniel A. de Boer
Chief Executive Officer, ProQR Therapeutics N.V.

In the past few years, antisense oligonucleotides (ASO) have become a trendsetting class of marketed drugs. The best known ASO is nusinersen/Spinraza®, developed by Ionis and Biogen for spinal muscular atrophy (SMA). The same oligonucleotide sequence can be used for all patients with SMA. This is a life-changing drug, as infants with the most aggressive form of SMA have a projected life span of less than a year. Instead of being on a respirator, infants are thriving and meeting developmental milestones. The success of nusinersen has opened up opportunities for other diseases of the central nervous system (CNS), especially life-threatening congenital diseases with rare genetic causes. Charles River is working with a number of early stage companies on both cellular and IND-supporting studies to advance this novel drug class. The second critical technology that is enabling treatment of genetic diseases is the increasing access to whole genome sequencing (WGS), allowing neonatalogists and genetics professionals to learn the exact mutation driving a patient’s disease. There are a number of synergies here that can reduce the time between diagnosis and drug treatment. The sequencing data allows a precision diagnosis, but also defines the target sequence for oligo drug development for that patient. Studies on the patient’s own cells can be used to screen ASOs in vitro for the best cellular genetic and functional readouts. Additionally, the ASO chemistry (the “backbone”) can be kept similar to nusinersen, which provided good penetration of CNS tissues and duration of activity. These are key accelerations for treating ultrarare diseases, even when only a single patient with a given mutation has been identified. As we learn more about these rare conditions, the data obtained from a single patient might be cross-applied to multiple patients with related but nonidentical mutations. The cost and time involved in WGS has come down to a few thousand dollars, and less than a month’s time — we can only hope that more patients will have access to this key technology soon, so that treatments can start as early as possible and prevent more life-threatening diseases from developing.

David Fischer
Executive Director, Charles River Laboratories

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I see the biggest impacts enabling scale-up and commercialization of next-generation therapies in the near term as small tweaks on existing platforms: items like incremental advances in closure of single-use systems enabling a completely closed cell therapy process, improvements in plastics creating more robust integrity at cryogenic temperatures, or working out nuances of adapting isolator technology to operations requiring a high level of dexterity and high turnover. Automated robotic systems have great potential but are still not having the impact of simpler automated devices that combine peristaltic pumps and pinch valves. The next-generation antibodies (e.g., Fabs, bispecifics) could be made with the same process trains as mAbs, but continuous processing will enable them to reach commercialization with less capital investment.

Erich Bozenhardt
Lead Process Engineer and BioProcess Specialist, IPS

Next-generation processing is quickly gaining traction in the industry because of the significant impact it will have on bringing therapies to patients. This manufacturing evolution will intensify the manufacturing process from multiple unit operations to a continuous flow-through process. At MilliporeSigma, we start process intensification by updating and upgrading outdated unit operations, then connecting these processes to run in a continuous flow-through fashion and ultimately reaching what we call “contiGuous” manufacturing — that is, a process that is continuous, connected and digitally enabled with all the suitable software and automation, run as an orchestrated production train. However, while continuous processing is the future of drug manufacturing, customers face challenges today in terms of speed to market, facility flexibility or cost of goods. We’ve designed the BioContinuum™ Platform to feature next-generation technologies that provide incremental process benefits now, with a mind to the “contiGuous” process of the future. The platform will revolutionize drug manufacturing by setting the standard for improvements in process efficiency, simplified plant operations and consistency in manufacturing. We predict that by 2020, approximately 20 percent of today’s molecular pipeline will be manufactured using elements of next-generation bioprocessing, or “contiGuous” manufacturing.

Andrew Bulpin
Head of Process Solutions at MilliporeSigma

It is important to discuss two key areas in manufacturing science that are developing in the pharmaceutical industry. The first is not necessarily scale-up, but rather streamlining manufacturing by pushing pharmaceutical manufacturing into the 21st century. For example, continuous manufacturing processes utilizing engineering techniques long used by other industries. Unlike what was once exclusively a batch process, with a multitude of limitations (inefficiencies, higher costs, higher risks of failed batches), drugs may now be produced in continuous manufacturing lines, coupled with precise monitoring through technologies like PAT (process analytical technology). It is important to note that a paradigm shift in manufacturing technology such as this will require manufacturers and suppliers to work side-by-side to design safe and effective processes. Next, instead of scale-up, next-generation therapies are likely to be scaled down, as a result of the personalization of medicine. We are not far off from walking into a local pharmacy and having a precise dosage form tailored to an individual patient, whether it is a 100-kg adult or a 2-year-old child. One such example would be the use of a 3D printer, mixing APIs and excipients as liquids, solids or melts to form exact dosage forms essentially on demand.

Frank Romanski, Ph.D.
Global Technical Marketing Manager, Pharma Solutions, BASF Corporation

Manufacturing across many industries, including pharma, is evolving into more digital, automated environments. As this occurs, the use of artificial intelligence (AI) to optimize the manufacturing environment, including better predictive tools for instrument performance and maintenance, could provide pharma with significant efficiency gains in productivity and is a compelling technological advancement in manufacturing. AI could also be deployed to optimize complex manufacturing lines, including biologics where process monitoring and feedback will determine the final product quality characteristics and can ultimately provide faster scale-up. Additionally, adoption of the Internet of Things (IoT) into manufacturing will also provide the industry with tighter, real-time controls, accelerating scale-up and release testing for small molecule and biologics manufacturing.

Tiffani A. Manolis
Senior Director, Pharma Strategic Program, Agilent Technologies Inc.

A large number of next-generation therapies are personalized drugs and formulations, which target smaller patient groups. Consequently, they are typically produced in small batches, which requires manufacturers to turn toward flexible manufacturing operations. This is where ready-to-use (RTU) containers come into play, and more specifically, standardized platform solutions. By working closely with pharma companies, machine manufacturers, and elastomer component suppliers, we introduced our iQ™ platform, which standardizes the tub format of RTU syringes, vials and cartridges to allow the different containers to run on the same filling line. Due to the standardization, fewer change parts are necessary when switching from one container to another, enabling pharma companies to fill various drug/container configurations on the same line with minimized changeover times. This enables the short time to scale-up and commercialization of these drugs.

Anil Busimi
Senior Global Product Manager for iQ Platform at SCHOTT

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The biopharma sector has been growing incredibly fast over the past few years. Biologics are now the fastest growing class of therapeutics in the world, with the market growing at 7–8% in the United States and Europe and around 18% in India and China. However, there are still some major challenges around affordability and access that need to be tackled before biologics become a realistic treatment option globally. Most of the manufacturing capacity is still located in the traditional Western markets, and we need to develop fast, flexible and cost-efficient manufacturing solutions that spread biologics production more evenly, as a local presence has a central role in driving the increased availability of these treatments.

Olivier Loeillot
Senior VP, BioProcess, GE Healthcare

Transparency is the best policy. I learned from a consultant that, if you want to set yourself apart from your competition, you need to identify what people hate about your industry, and then find ways to correct that problem within your organization. We know people hate negative surprises like delays and out-of-control costs, and just not knowing the status of their projects. That happens a lot with other CMOs.

Ed Price
CEO, SEQENS North America CDMO (previously PCI Synthesis)

The pharmaceutical industry is continually evolving — from laboratory and research advances in life sciences to the rapidly expanding use of new technologies such as artificial intelligence, advanced analytics and robotics. The one thing that has remained constant is our mission — to enable our customers to make the world healthier, cleaner and safer. This is the foundation for all that we do. We offer integrated solutions that help our customers bring medicines to the market.

Franco Negron
President, Commercial Operations, Thermo Fisher Scientific, Pharma Services

Catalent formally introduced a corporate responsibility commitment in 2017 that cements our belief that ethical business practices are essential to fulfilling Catalent’s mission of helping people live better, healthier lives. Our corporate values, including integrity and ethics, are at the foundation of our culture.

Shannon Trilli
Director of Corporate Responsibility, Catalent

As a company, SGS takes ethics very seriously and conducts itself to the highest standard of professional behavior. The SGS Code of Integrity defines the main principles of professional integrity for the organization, and all staff — from the senior management to entry-level employees — must complete the corporate integrity training program each year.

Jessica Martin
Global Head of Marketing, SGS

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We are at a pivotal time in the pharmaceutical industry — advancing technologies, including the use of advanced analytics and artificial intelligence are transforming the development and manufacturing of new medicines. Analytics, using the enormous data sets generated by drug development, are being used to develop systems that have the potential to predict the properties of new drug candidates.

Franco Negron
President, Commercial Operations, Thermo Fisher Scientific, Pharma Services

AI is poised to play an increasingly important role, as the pharmaceutical industry understands itself more and more as a data- and knowledge-intensive enterprise — and as it engages in the full “healthcare package” of prevention, diagnosis, personalized treatment and maintenance.

Andreas Persidis, Ph.D.
Co-founder and CEO, Biovista Inc. & President, Hellenic BioCluster

We believe that artificial intelligence (AI) and machine learning will play an increasingly important role in the diagnosis and prediction of disease — and will eventually become part of routine practice.

Ben Newton
Chief Digital Officer, GE Healthcare Life Sciences

AI and robotics will help with analytics. The science we help clients with is getting increasingly complex, which means that the demand for increasingly sophisticated equipment, technology and training will continue to be important. We’re constantly evaluating new equipment and approaches to see if they can handle the complexity, speed up processes and reduce overall costs.

Ed Price
CEO, SEQENS North America CDMO (previously PCI Synthesis)

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Colorcon continues to provide solutions for pharmaceutical technology and manufacturing trends to meet the industry needs for coating flexibility and productivity while addressing patient adherence by making tablets easier to swallow.

Ali Rajabi-Siabhoomi, Ph.D.
Vice President/Chief Scientific Officer, Colorcon

The low cost and availability of IoT (internet-of-things) technology continues to have a significant impact on our company this year. Snapdragon develops chemical manufacturing processes that rely on the integration of a wide range of mechanical and electronic components. We use IoT technology to coordinate the working of complex flow systems. We are also able to continuously harvest rich data sets from these systems with IoT-enabled sensors and process analytical technologies (PAT). This combination of automated equipment and sensors in our laboratory systems enables automated experimentation. We can rapidly perform large numbers of experiments and gather incredibly rich data sets from every experiment. This capability delivers much greater process knowledge and leads to more rapid process development and ultimately more efficient and controlled processes for drug substance and fine chemical manufacturing.

Matthew M. Bio, Ph.D.
President and CEO, Snapdragon Chemistry, Inc.

Green chemistry is at the forefront of minds within the chemical industry, and utilizing our enzyme technology is becoming the norm rather than the exception. Within Arran Chemical Company (acquired by Almac in 2015), our AdaPT (Arran Deploys Advanced Product Technologies) strategy and through a multi-million Euro investment and application of our selectAZyme™ technology, we have remained at the forefront of innovation to ensure our customers receive best-in-class solutions. Our team has researched more than 30 projects in the last year involving multi-disciplinary research on enzyme optimization and chemical process design. Another key milestone was winning the Irish Industrial Chemistry Award for the achievement of complete commercialization of an enzyme immobilization process involved in a medical device destined for the U.S. market.

Michael Cannarsa, Ph.D.
Director of North American Business Development, Almac

As a busy CDMO, we are always looking for new opportunities to expand our toolbox, and this year was no different. There are several reasons for this. The first is to make development of cGMP materials faster, cheaper and more efficient for our sponsors. To that end, we carefully evaluate new technology exhibited at the industry meetings we attend. For example, this year we have augmented our capabilities in cryogenics, micronization, large-scale chromatography and lyophilization, to name just a few.

Ed Price
President and CEO, PCI Synthesis

Although continuous flow has been utilized in similar industries for over a century, the pharmaceutical industry has been slow to adopt this technology for a variety of historical and regulatory reasons.

Shawn Conway, Ph.D.
Director of Engineering, R&D, Cambrex

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