March 16, 2021 PAO-03-21-NI-01
Fabian Stöcker, VP Global Strategy and Innovation, SCHOTT Pharmaceutical Systems
A: We see a couple of trends that will shape the industry in the coming years. One of these is the topic of smartness in the healthcare industry, which will be driving new innovations. On the one hand side, this is accompanied by the increasing trend to digitalize processes in production, on the other it is driven by digital services, which are increasingly expected between suppliers and customers. Another trend is the further developments and steep increase in new biologics, as well as highly sensitive drugs. These unique and highly advanced formulations require new packaging variants specialized for their individual needs. Throughout these developments, the importance of patient safety and drug stability will remain a key focus for the entire industry. Another significant development is the increasing use of ready-to-use (RTU) solutions and particularly RTU vials. We expect this trend to increasingly penetrate the European and North American markets, while also moving to Asia and especially to the Chinese market. Lastly, COVID-19 vaccines will continue to have a great impact on the pharma industry. While the various vaccine projects are being scaled up, the industry will be taking initial steps for the preparation of transferring the vaccines from vials to prefilled syringes in the future.
Isabelle de Cremoux, CEO and Managing Partner, Seventure Partners
A: I believe that the focus on the microbiome will significantly increase in 2021 and beyond. The pandemic hardly disrupted microbiome research, and 2020 saw an increasing level of investment and partnering in this area. The number of microbiome-focused patents and clinical trials is on the rise, and last year the first phase III studies concluded. 2021 is likely to see the first market authorization of a microbiome-related drug, while the range of indications for microbiome research is expanding rapidly. Also, there are now 40 new studies focusing directly on the link between COVID-19 and the microbiome.
A: I believe we will see an emphasis on precision medicine this year, as the industry focuses on a diagnostic and therapeutic approach that not only helps put each patient in the center of their medical care, but also helps physicians and payers make informed decisions in this unprecedented time. Tools like precision imaging radioligands are powerful in not just assessing the disease, but also monitoring the disease and selecting appropriate targets for therapy. They can allow patients to receive personalized treatment, while minimizing or eliminating unnecessary treatment. For physicians, precision imaging radioligands could enhance their ability to provide a more accurate diagnosis, select optimal therapies, and monitor treatment response and disease progression. For payers, they may reduce time and costs associated with suboptimal treatments. Through this combined approach, Advanced Accelerator Applications (AAA), a Novartis company, is leading the development of new complementary oncology drug pairings targeting the same marker or receptor expressed on a tumor that may be used to both image and treat cancer, potentially maximizing the possible therapeutic impact for patients. While this approach has historically only been utilized in niche disease areas, the advent of new targets, new radioisotopes and rapidly increasing clinical experience is leading to broader exploration of radioligand therapy and imaging in multiple tumor types. At AAA, we are committed to expanding the research and development of targeted radioligand therapies in 2021, and I believe the industry is already starting to embrace this innovative approach to cancer care.
Tim Roberts, Vice President, Global Sales, Clinical Services | PCI Pharma Services
A: I think there will be two main growth vectors in 2021 and beyond. To start, at a micro level, the expansion of direct-to-patient service directly to patients’ homes. With COVID as a driver and the subsequent closure (through lockdown) of medical facilities and clinics, there will be required distribution to patients’ homes to ensure continuity of trials. The future of this personalized supply chain strategy will likely continue even after this year, with new business models and processes in place. Additionally, from a macro perspective, there will be a general growth in clinical trials, since there continues to be a boom in both small and large pharma investment, with R&D being a focus for life-changing therapies in newer therapeutic areas, such as rare disease and cell and gene therapy. These areas, combined with the increased number of oncology trials in both small molecule and immunology, will drive the market forward with new solutions for patients.
A: The viral vector–based platforms that produced many of the COVID vaccines (AstraZeneca, Sputnik, and Johnson & Johnson) have proved that the technology is flexible and responsive and has the potential to be applied much more broadly beyond vaccine production. We anticipate that these platforms will be used to accelerate treatments for a wide variety of diseases, such as immuno-oncology, cardiovascular disease, and autoimmune diseases. Cell and gene therapies are also starting to gain traction, with significant investments being made by industry players to gain scale and secure capabilities. The scale of investment made in 2020 is huge and is likely to continue in the coming years as the industry players fill gaps in their portfolios needed to bring therapies to market. The Alliance for Regenerative Medicine moreover announced last November that the global financing for the regenerative medicine sector set an annual record of $15.9 billion through just the first three quarters of 2020. There are currently 97 ongoing phase III clinical trials and regulatory decisions expected on multiple product candidates in the coming months. Finally, we also expect that pharmaceutical and drug manufacturers will continue to examine their supply chain and find ways of producing closer to the market as a way to secure supply regionally. CDMOs and CMOs are going to play a significant part in supporting this trend.
A: There’s no denying that the world has changed in the past 12 months. We’ve witnessed the almost miraculous development of new vaccines in timelines previously thought unattainable. Developers, regulators, supply chain managers, and myriad other stakeholders rose to the challenge with a resounding spirit. This is not some new cavalier attitude toward compromised safety; it was the invention unleashed by necessity. The year 2021, therefore, will be remarkable for the cultural shift emerging from users of conventional tools and technologies to a rethinking of development and manufacturing approaches — we have a new meter stick by which to measure our successes. On the development side, new therapeutic approaches, such as mRNA, will become more prevalent, requiring new raw materials, manufacturing methods, and facilities, and possibly new distribution infrastructure. Cell line development technologies, to keep pace with therapeutic development demand, will increasingly deploy automation and rely more heavily on automation, image analysis, and machine learning. On the manufacturing side, rapidly deployable and reconfigurable production capacity will start to replace conventional brick and mortar manufacturing infrastructure to address the need for adaptable capacity for the rapidly evolving therapeutic landscape. With the increased flexibility afforded by these modular facilities and workflows, process analytical technologies and automation, together with enterprise-level data control and management, will also be more broadly adopted over the coming year.
Marrie Groeneveld, CCO, SkyCell
A: Firstly, 2021 will see greater controls and regulation being brought in for the pharmaceutical supply chain due to the fragility that has been made evident in 2020. This includes shortages of the correct packaging needed to keep vaccines viable in transit and issues surrounding flight availability. We will also see an increased use of data to combat these issues, as containers that can track deliveries and the condition of the contents will be in greater demand as we move into 2021 and beyond. We are already doing this at SkyCell through our data-driven Lane Risk Assessment that compares the risk profile of each lane based on data gathered from previous shipments.
To help combat this, not only should there be a move toward more sustainable and long-term packaging solutions, but greater data flow integration will ensure the end-to-end visibility of the supply chain. This can be achieved through the use of Internet of Things (IoT) devices that track, monitor, and alert pharmaceutical shipment companies of any potential issues. This links to the second development the industry will see: with the biologics market expected to reach a value of more than $1.2 trillion by 2030, with a compound annual growth rate of above 15%, there will be an industry-wide drive to improve the capacity of temperature-controlled transport.
Finally, something that is not specific to the pharmaceutical industry but will certainly affect how it works will be the continued trend of working from home. Although some roles, such as ground handling, will have to be done on site, a strong IT infrastructure will enable companies to work entirely remotely without compromising on quality
Alen Guy, Technical Director, the Pharmaceutical Business Group of IMCD
A: 2021 will be unlike any other year in one clear way: digitalization. I believe the utilization of AI (artificial intelligence) and machine learning will make big advances in 2021, particularly with respect to shortening development times for drug products. More collaborations will spring up around a group of high-demand companies. These companies will be agile data analysis specialists. They will help the industry structure data from many sources. In this way, they will be able to start to unlock the potential for AI. Shortening time to market has always been something of a dream for pharmaceutical companies. The prospect of gaining more exclusivity for sales from the patents on their molecules will be highly attractive. I think we will see a quantum jump in accelerating through phases for many drug molecules, based upon use of AI.
The second element of digitalization is the way we approach our customers. Behavior has changed, and I have seen reports that, in some segments of pharmaceutical marketing, a majority of doctors want to stay “virtual” and use digital communications as their preferred channel in their detailing of drug products. Virtual seminars, webinars and even one-to-one meetings will need to develop digitally so the people that need the information, from manufacturer to prescribing physician, get what they need. This will require an agile transformation across large parts of the industry.
A: Personalized medicine is coming of age and will have a significant impact on how drug research and development is conducted and refined in the future. Personalized medicine isn’t a new approach, as illustrated by the approval of the monoclonal antibody, Herceptin, over 20 years ago for breast cancer patients that overexpress the HER2 protein.
The concept of personalized medicine is based on a patient being diagnosed and treated according to their own genetic profile. Genetic screening techniques and molecular profiling are tools that can be used to inform on individual susceptibility to disease(s). These tools can also be used to predict whether a specific treatment will be more or less effective and safer in individual patients or a subgroup of the patient population. Increasingly, in clinical trials, patients are enrolled and selected based on their own genetic profile — especially in oncology but this has expanded into other indications. This process of selecting therapies based on a patient’s own genetic profile thus has the potential to transform the traditional drug research and development paradigm into a more tailored approach, both from a clinical research perspective as well as in patient care settings.
Its success in 2021 and beyond relies on continued scientific developments (e.g., improving on existing technologies and earlier stage screening), as well as new discoveries that further the sector’s understanding of disease. In addition, all stakeholders (scientists, healthcare professionals, policymakers, payers, and industry) must continue to collaborate and evolve in order to successfully deliver these customized therapies.
A: We envisage that the pharmaceutical industry will continue to innovate and systematize the unprecedented collaboration that has led to faster development of COVID-19 vaccines and treatments.
These partnerships and collaborations have been simplified and streamlined by the use of technology, and the use of remote technologies for virtual audits and inspections is likely to increase throughout 2021 as travel restrictions continue. At Catalent, we host or perform more than 500 audits per year across over 45 sites globally, and, as the sophistication of the remote inspections continues to rapidly evolve, for example using 360° video to give customers and regulatory agencies — including the FDA — virtual tours of facilities, we expect them to be more widely adopted.
While the technology is unlikely to replace onsite audits and inspections completely, the industry and regulators have recognized the time- and cost-saving benefits of remote inspections. Protocols are continuing to be written that balance the capabilities and limitations of current technology, and it is unlikely that it will ever go back entirely to the old ways of doing these things.
We also anticipate that there will be greater use of augmented reality and virtual reality, and see tremendous potential value in using technologies such as HoloLens, which allows text, graphics, or video to be overlaid on the user’s view. At Catalent, for instance, we have begun to look at its potential in employee training and onboarding for some standard operating procedures that involve high-risk processes and can see this being rolled out more widely.
A: As years pass and technology evolves, it becomes increasingly difficult to single out a specific area or topic as to be the most significant development for Industry. However, a clear tendency towards digitalized systems has been in constant development and has a strong impact on the pharmaceutical industry’s present and future development.
We are in the midst of the transition from traditional paper-based systems, which are becoming costly to maintain and effectively review and update in the current pharma regulatory environment. This is mainly due to the increasing requirements to which pharma manufacturing has to adhere to ensure drug efficacy and patient safety, from the manufacture of the needed raw materials, product manufacture, final drug storage, and shipment to the associated pharmacovigilance activities. The humongous amount of data generated through the product life cycle needs to be registered, monitored, and evaluated.
This is where the computation advances come in — an AI capable of effectively tracking and interconnecting all this data and metadata, in a real-time scenario.
A boost in the development and interconnection of digital AI systems, cloud computing, and monitoring systems is expected for the current year, which will pave the way for new drug development strategies, personalized medicine, patient follow-up, and business development and marketing strategies.
A: One of the areas to keep an eye on is the level of funding for biopharmaceutical companies, which reached record levels in 2020, driven not only by VC, with other funding vehicles growing. While, of course, COVID-19 was a factor here, it contributes to a long-standing trend of innovation in pharma being driven by small and emerging companies. There is a record number of early-phase candidate compounds in the pipeline today, with some 80% of these assets held by such small and even virtual companies. Lonza Small Molecules continues to adapt our business models and operating practices to better serve the needs of these companies, which means refining and expanding our service offerings, particularly for preclinical to first-in-human studies (Lonza SimpliFiH® Solutions). Consolidation in our industry continues, driven in part by the aforementioned factors. We see signs that M&A activity has adapted to the constraints of COVID-19 with some notable deals in the industry among both innovators and service providers (CROs/CMOs/CDMOs).
A: 2020 brought significant growth within the development and manufacturing of biologics. Apart from projects driven by COVID-19, we witnessed advances across the entire biologics spectrum. Protein therapeutics are moving further to more complex formats, and cell and gene therapies, as well as mRNA-based treatments, have been established as important therapeutic platforms in the market, growing significantly. This is a significant game-changer, far beyond COVID-19 vaccines. It is evident to the experts in the field, but in certain parts also to the public, that these new complex therapies require much more pharmaceutical development toward a robust drug and are far behind robust technologies like monoclonal antibodies.
To accommodate this growth, there is a need to increase the available sterile manufacturing capacity. Furthermore, the industry needs to focus on providing an integrated pharmaceutical development approach for sterile drug products of the various therapeutic modalities, encompassing a holistic view on the various aspects of formulation and stability, manufacturing operations, and, finally, administration to patients. Looking at the development in the area of administration, we see an increased interest in self-administration and drug–device combination products, such as prefilled syringes, wearable devices, or autoinjectors, reaching commercialization. These trends will undoubtedly continue throughout 2021.
A: First, I think of the lightning speed at which developers were able to deploy capacity and start clinical trials for vaccines. This was unprecedented and underlined the importance of CDMOs to offer the ability to scale up or down fast and as needed. Another key benefit of tapping into CDMOs’ manufacturing capabilities was the ability to manufacture across the globe, sometimes locally: disrupted supply chain networks require production to take place at multiple locations, which is enabled by working with companies like Lonza that have a readily available global manufacturing footprint to address a multitude of therapy and vaccine technologies.
As a cell and gene therapy manufacturer, we intimately understand the criticality of the supply chain, raw materials, and cold chain logistics due to the personalized nature of these therapies. Especially when dealing with shipping live cells across long distances and in a timely manner, we need strong, reliable partnerships and logistical suppliers to ensure that the therapies can be shipped at a specific temperature and within a precise timeframe. We are faced with a similar situation on a global scale. Some of the therapies and vaccines being developed for COVID-19 require very low temperatures in addition to strong supply chain networks and very specialized storage capabilities. This makes shipping these drugs to remote places across the globe very challenging. I think we will see more specialized supply chain networks established and reaching more areas of the world.
A: When I think about significant developments in the industry this year, I agree it goes beyond COVID-19 vaccines and therapeutics, but they still are indicators of an important shift in our industry — the continued adoption of novel modalities and technologies. Take for example the use of mRNA: it has been incubating for several years, but now mRNA has found its first real-world application in COVID-19 vaccines. Most importantly, the past months have proven that mRNA vaccines are both safe and efficacious, providing a clearer regulatory pathway, subsequently opening the door for more applications across indications. Of particular notice is also the speed and flexibility with which these vaccines have been developed and are already being tested against the mutated virus.
The success of mRNA COVID-19 vaccines also highlights another important development that directly impacts other novel modalities and their pathways to success. That is the focus on lipid nanoparticles as a unique platform to deliver nucleic acid payloads to the target. This has exciting implications for the entire field of cell and gene therapy, for example, as it offers a complementary route of delivering the novel modalities currently being developed to their targets within the body.
Other modalities, such as monoclonal antibodies and antibody–drug conjugates, will also keep up their momentum and will expand into new indications, especially in oncology.
Bringing this all together, this rise of novel modalities, technologies, and complementary tools helps us to assemble a powerful toolkit with the potential to be game-changing in the fight against numerous devastating diseases and global health challenges.
A: The last few years, ATMPs (advanced therapy medicinal products), have been the focus of much attention and capital investment. However, even with the large number of products in late-stage clinical trials, the number of FDA-approved therapies has remained low. I believe we are poised to finally see a number of approved therapies hit the market in 2021, as seen with the approval of Breyanzi® at the beginning of the year.
As a side effect of the COVID-19 pandemic, the industry has seen a significant increase in the number of facility projects and schedule acceleration. The manufacturing life cycle had already been shrinking — but a pandemic has pushed us to accelerate it even further. We have learned how to deliver mRNA vaccines in record time. The new norm for our industry is going to be accelerated, fast-paced projects with integrated project teams that question traditional approaches to project delivery.
Up until this point, ATMPs have been typically developed and delivered with more manual, laboratory-like processes, which require scale-out to meet market demand. Advances in automation and robotics, along with new approaches to project delivery, are going to be needed to drive our industry forward into the future and support the new therapies that are coming to market.
A: In 2020, the biotech industry saw major advances in cell therapy. Cell therapies currently hold immense potential in oncology and have already proven their value in the treatment of hematologic cancers, but, despite incredible advancement in treatment strategies, solid tumors remain as an area of significant unmet clinical need, representing nearly 80% of new cancer diagnoses. Challenges, such as overcoming the tumor microenvironment, among others, have prevented cell therapies from demonstrating significant efficacy in treatment and have served as major barriers to successful clinical development. In 2020, we witnessed potentially transformative advancements in cell therapies, particularly the emergence of novel technologies and approaches that promise a new generation of clinical cell therapy candidates with the potential to overcome these long-standing challenges. A key advancement in this case will be the optimization of cell receptor profiles through innovative screening and selection processes. For example, at Acepodia, we are hyperfocused on solid tumor treatment and believe that we have unlocked the ability of natural killer cells to engage tumors by selecting for the highest concentration of tumor-activated receptors. We aim to treat these indications by uniting these cell lines with antibodies through conjugation. While still evolving, we expect to see many of these programs, including our own, advance in the clinic and potentially even show early signs of efficacy in 2021, which could change the treatment paradigm for these patients and elevate the therapeutic potential cell therapies beyond hematologic cancers.
A: It’s been incredible to watch the advancements that have happened in the antiviral disease space in 2020. Our colleagues in these fields have worked overtime in the hopes of providing much-needed COVID-19 vaccines and therapies. What’s also been encouraging is that innovation in other fields, such as gene therapy, neuroscience, and oncology, hasn’t slowed down during this time.
At BeyondSpring, we are focused on developing better treatments for oncology patients, and it’s been inspiring to see a number of important breakthroughs in different aspects of the field that really benefit patients. We look forward to seeing this continue in 2021. One important endeavor where we could see significant impact is in the development of combination treatment regimens in immuno-oncology. A multi-faceted approach that targets the immune system in a number of key steps in the immune cascade is expected to provide the most optimal benefit from immuno-oncology therapies. These treatments have the potential to give long-term benefit and the hope of cure for these patients.
The other advancement that we look forward to seeing is the design of combination treatments without added toxicity. Adding different agents together as combination therapy typically results in added efficacy but also in potentially added and unacceptable toxicity. This means that we need combination treatments that will tackle cancers with greater efficacy and fewer adverse effects.
We're looking forward to being part of all of the exciting innovation that is sure to come in oncology and beyond this coming year
A: A relevant topic in the healthcare space that has arisen amid the COVID-19 pandemic is the alarming drop in cancer screening rates globally due to limited access of patients to hospitals settings and cancellations of non-elective procedures, which will inevitably result in increased cancer deaths in coming years. The pandemic has reemphasized the relevance of the advancement of patient-friendly solutions that can help diagnose the warning signs of cancer effectively, while also reducing the need of hospital visits and invasive procedures. I believe that, in 2021, we will continue to see efforts from biotech and medical technology companies to improve performance of current patient-friendly screening tools, to offer increased sensitivity and specificity while ensuring patient compliance. For some cancers, such as colorectal cancer, this is particularly relevant, since screening for and later removal of benign colorectal polyps can in fact prevent development of the disease and later cancer.
Other developments I foresee in the medical technology space include a continuous effort to advance technology solutions that enable patients access to a personalized digital health, including data analytics and coaching, to improve patient compliance to medication and health screenings. This will include features that enable direct interconnection with patients’ cell phones and virtual reading centers, which will expedite diagnosis and treatment.
A: I believe the most significant development in the biopharmaceutical industry can be summed up in one word: cooperation. In all my years as a physician and biotech executive, I have never seen such a strong collaborative effort among professionals from industry, academia, and government institutions working together to attack a common foe. These partnerships within the healthcare industry are the key factor to improving the overall response to the global pandemic. Countless lives have been and will continue to be saved due to this remarkable cooperation among various stakeholders.
Can we apply these same methods of cooperation and technology to develop life-changing therapies for diseases not well served by current therapies? We can for the many diseases caused by infection. Although therapies like CAR-T cells to treat cancer and lifesaving organ transplants are remarkable and sexy, they are only applicable in rich countries with finely developed medical systems. The biggest problems globally remain disability and death due to infections. These range from the dramatic to the mundane, but they cause pain, suffering, and death while keeping communities from reaching their full potential. Vaccinations work! Now, with the new mRNA technology showcased in the development of the COVID-19 vaccines, the process is fast and effective. Who would have thought we would develop not one, but two vaccines against COVID-19 in less than 12 months that are 95% effective. This is breathtaking! If these technologies are turned on the infectious disease problems of the world… change will occur… for the better.
A: We’ve learned from the pandemic that biopharma must prioritize formulating drugs in easily administered forms wherever possible. Much of the world may be subject to some degree of quarantine for at least several more months; we may also face epidemics or other catastrophes in the future where traveling to a doctor’s office to get important treatments delivered by injection from a professional just won’t be feasible.
Innovation is needed in order to reduce the frequency of patient visits to healthcare centers and minimize the risk of exposure to COVID-19 while also maintaining the access to therapy.
Keeping in mind that traveling to the doctor’s office to get important treatments delivered by injection from a professional might not be feasible, our industry needs to adjust and adapt accordingly in order to keep patients’ safety and well-being top of mind. Through developing therapies in oral or intranasal forms, companies can facilitate easy access to treatment and potentially improve patient compliance. I expect to see many biotech and biopharma companies in 2021 developing new formulations to not only provide patients with comfort and convenience, but also better prepare and handle potential disruptions similar to what we experienced in 2020.
A: One thing the COVID-19 pandemic has taught our industry is that R&D has to be flexible in unprecedented times in order to adapt and continue advancing relevant products that serve the needs of patients with cancer, rare diseases, and many other diseases who can’t afford to wait until the pandemic comes to an end. We have seen outstanding efforts from many companies, including X4, to amend clinical trial design, management, and planning to accommodate our patient communities, ensure their safety, and enhance convenience as they participate in our studies. This includes establishing virtual visits and assessments and enabling home visits or in-home drug delivery, which have been far more accessible for patients who are immunocompromised. I anticipate these practices will become the new normal moving forward for some companies, especially those advancing oral formulations or representing immunodeficient and immunocompromised patients with the highest risk.
I have also been pleased to see an increased investment in recent years for the development of rare disease treatments. As new genetic testing tools become more accessible to patients and the research community continues to gain a better understanding of the genetic mechanism of disease through the increasing advancement of genomic tools, we will continue to find novel therapeutic targets and develop innovative drugs to address them. We should see more therapies for rare diseases entering product pipelines in 2021 and beyond, and hopefully some of them will become the first disease-modifying treatments for many of these patients in the near future.
A: The rise of cell and gene therapies is astonishing, considering the first approvals were only in 2017 — not only does this represent a huge advancement in treatment, but also a uniquely challenging area to develop, regulate, scale, and effectively integrate into the healthcare industry
On the small molecule side, moving more toward efficient processing, specifically with continuous manufacturing of solid oral doses, pushes pharma into a modern manufacturing scheme, which is more efficient from a business perspective, less wasteful from a sustainability perspective, and better to control from a safety perspective.
Finally, pharma continues to move into more personalized medicines, and this starts with the modern biologic treatments based on ones’ own cells and ranges to 3D printed formulations made on demand, and these are really exciting techniques to see come to market.
Fabian, having joined SCHOTT AG in 2012, is Vice President Global Strategy & Innovation for the SCHOTT Business Unit Pharmaceutical Systems Within the management team, he is responsible for strategy, product management, product development, IP management and market intelligence covering ampoules, cartridges, pre-filled syringes and vials for injectable use. Prior to this, he was heading the corporate strategy department of SCHOTT AG, being responsible for corporate development, strategic planning, and portfolio management of the whole group. Fabian studied business administration at the University of Cologne/Germany and the Corvinus University of Budapest/Hungary and holds an MBA from the University of Cologne. He is a Chartered Financial Analyst (CFA) charterholder from the CFA Institute, Charlottesville/USA.
Many of the change agents I have seen in 2019 are derived from changes in regulatory law, commercial downscaling, and impact from patent expiry strategies. The largest external regulatory change came from the issuance of the long-awaited EMEA Annex I, clarifying which technologies are required and acceptable, when and why.
The change in operational focus, from clinical scale-up to commercial scale-down, is enabling use of smaller, modular, flexible fillers with self-contained isolators. In parallel with the approval of biosimilars and biobetters, there is strong industry focus on individualized micro-batches, for CAR-T solutions and gene therapy products. The use of process automation and robotics have increased in all fill-finish unit operations. Widespread implementation of ready-to-use/ready-to-sterilize components and single-use (SUT) in upstream and downstream (SUS) through final fill designs have changed how facilities are planned, reducing plant size and changing warehouse space to accommodate densely packaged plastics goods.
Filling modalities have also been changing; bags that can be mated to lock-luer fittings with pre-sterilized needles and blow-fill-seal/form-fill-seal are re-emerging as processes that offer potential unit cost reduction. Traditional vial and syringe container designs are also changing as suppliers improve standardize offerings while having options including clear plastics.
The most exciting technological or scientific advancement that has influenced our business strategy in 2019 is our novel epigenetic regulator program. Unlike gene therapies, which target and modify DNA directly by inserting specific genes into patient’s cells, epigenetic regulators control or modify gene expression through processes that do not alter the sequence of DNA directly. Our lead asset DUR-928 is a small endogenous molecule that plays an important role in regulating cellular functions such as lipid homeostasis, inflammation and cell survival, crucial pathways involved in many acute and chronic diseases. DUR-928 has shown positive results in a phase IIa trial for the treatment of alcoholic hepatitis, a devastating acute condition with high mortality rates and limited therapeutic options. We are also advancing programs in other indications that could benefit from DUR-928, such as non-alcoholic steatohepatitis (NASH) or psoriasis. We believe that epigenetic regulation is a powerful and untapped treatment approach for many challenging diseases.
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