Personalized therapeutics, such as cell and gene therapies and personalized cancer vaccines, are highly individualized and require specialized technology solutions to ensure patients receive and benefit from these drug therapies. At the start of the year, the U.S. FDA said that it expects to receive more than 200 new cell and gene therapy applications per year by 2025, and to approve 10 to 20 new cell and gene treatments each year.
 
Cell and gene therapy technology company, Vineti has opened a new regional headquarters in Bethesda, MD, to help expand access to its Personalized Therapy Management platform in support of the increasing cell and gene therapy manufacturing facilities in the region, such as Paragon, Kite, Precigen, Bluebird, and Pfizer. Vineti’s third-party platform has undergone FDA and EMA regulatory review as part of a successful, expedited cell therapy filing.  
 
Vineti recently entered partnership agreements with CareMetx to build patient-centered digital and service solutions to streamline cell and gene therapies, and with Marker Therapeutics to optimize logistics relating to Marker’s clinical stage pipeline of therapies in a safe and efficient manner.
 
Contract Pharma spoke with Amy DuRoss, Vineti’s chief executive officer and co-founder, to discuss the key challenges cell and gene therapies face, and the advances being made to enhance safety and speed, making processes move more quickly and improving access to these highly individualized therapies.  –KB

Contract Pharma: What are the key challenges pharma/biopharma companies face in the cell and gene therapy market?
 
Amy DuRoss: Cell and gene therapies (CGT) are the most complex therapeutic processes that medicine has ever encountered. In contrast to the linear workflow of traditional medicines, the patient-centric CGT manufacturing process is more circular. A patient’s unique treatment must make it to him or her without any mistakes in Chain of Custody or Chain of Identity as it passes through numerous stages of the process. If there’s ever a mix-up and a patient receives the wrong cells, the resulting immune response and rejection could be extremely dangerous, or even fatal. Therefore, traceability is an essential patient safety component.
 
Compounding this challenge is the fact that —unlike with small molecule drugs such as Lyrica and Zoloft —CGT treatments are difficult to batch process. Specifically, in these autologous therapies, where cells come from and are returned to the same patient, each patient is considered a “batch of one.” When a patient’s own tissues represent the starting material for a treatment, the extra care required can hamper efforts to manufacture these treatments at scale and with speed if enabling technologies are not in place. Allogeneic therapies — which are based on donor cells — can also be complex, because the donor material and final drug product may need to be matched to the intended patient in some way, often at a molecular level. Allogeneic therapies may also require multiple dosing, with each dose having the same stringent traceability requirements.
 
Vineti’s Personalized Therapy Management (PTM) platform is a cloud-based, centralized solution, which provides complete orchestration of critical components in the CGT process for each patient’s unique treatment needs. The Vineti platform can track patient and donor material, workflow data, and drug product processes, in real-time, and coordinate the disparate steps of each process component so that they are fully integrated as a single therapeutic management process, instead of a fractured network of unequal parts. This orchestration is critical to patient safety and manufacturing efficiency.
 
CP: What are the challenges moving from clinical to commercial scale manufacturing for cell and gene therapies?
 
AD: Two key challenges are often at odds with each other — time and complexity.
 
In advanced therapies, trial sponsors are under pressure to receive expedited status and move as quickly as possible. Traditional clinical trial phases are often shortened or merged. If you look at the clinical pipelines of many cell and gene therapy biopharmaceutical sponsors, you’ll note that many have products in phases such as “Phase I/II” or “Phase II/III.” Trials can expand from a handful of patients to dozens or even hundreds much faster than originally anticipated. According to data compiled by the Alliance for Regenerative Medicine, more than 90 therapies have received some type of accelerated status in the U.S., E.U., or Japan.
 
At the same time, the patient journey and drug product process are more complex than ever before. Clinical trial operations and logistics require a new multi-dimensional, patient-centric paradigm that outstrips the capabilities of spreadsheets and manual management quickly.
 
Data management also takes on new complexities. With traditional small molecule drugs, the pathway to commercialization often presents challenges to drug manufacturers during the regulatory approval process, as they attempt to gather and normalize data from the entire development journey to meet regulatory requests. However, these challenges are amplified during the CGT approval process, because the data stream is more complicated and, to a greater degree, is fragmented among the various components involved in sample handling throughout the manufacturing process.
 
Approval of CGT treatments also carries a unique burden in that the software platform responsible for managing and orchestrating the therapeutic process must undergo regulatory approval as part of the drug product filing. That’s how critical a software system is to patient safety. These therapies represent the first time that regulatory authorities have added this type of systems-review requirement to the application process. 
 
CP: Where have advances been made to enhance safety and speed, make processes move more quickly and improve access to CGT? 
 
AD: There have been many advances – here are two of the most important ones.
 
Improved access and safety for patients
Digital, cloud-based solutions can greatly improve the safety — and speed — of the personalized medicine process. Systems such as Vineti allow for private and secure capture of critical patient data at each step in the value chain, such that each participant in the product journey can provide the right data at the right time to ensure product accuracy, safety and predictable product administration. At certain steps, critical data can be displayed to boost patient safety. At other steps, as appropriate or required by regulation, some data can be masked to preserve patient privacy. The simplification of this process and ease of deployment can allow a broader group of healthcare providers to prescribe and access these products for patients. The transparency built into this process can boost patient safety and confidence.
 
Digital solutions can also make the process move more quickly for patients who are often very ill. Vineti’s platform can manage and orchestrate all the necessary patient information, for example, without requiring many error-prone and time-consuming paper forms, phone calls, and phone chains through different healthcare personnel. Courier logistics can be managed through the system for greater efficiency. These features can really streamline the process and keep things moving faster.
 
Simplicity and familiarity for healthcare providers
This is one of the most significant advances towards improving access to these therapies. Personalized therapeutics require workflows that cross boundaries and push stakeholders into new roles. Busy healthcare providers are now having to manage regulated, Good Manufacturing Practices (GMP) — and they are having to do so on behalf of a rapidly growing group of biopharmaceutical manufacturers. Therefore, they need to have confidence that these therapies can be managed safely on behalf of their patients.
 
If these critical aspects aren’t as simple for them as possible, progress will slow. Digital solutions can simplify the complexity of the personalized medicine process, providing greater product accuracy, safety and predictability. The simplification of this process and ease of deployment can allow a broader group of healthcare providers to prescribe and access these products for patients.
 
The Vineti system will be in use in almost 200 leading medical centers worldwide in 2019. Healthcare providers know our platform and know that we emphasize simplicity, usability, and transparency for their teams. That familiarity helps create a deeper sense of familiarity and trust.
 
CP: How can key challenges to CGT processes be overcome?
 
AD: Standardization and industrialization
It may sound counterintuitive to standardize such deeply personalized products. But if we don’t standardize the parts of these processes that can be made predictable and repeatable, then we can’t industrialize this field. And if we can’t industrialize it, the field can’t grow to reach all the patients in need. “Mass customization” has been possible in other industries, and we need to start focusing there in cell and gene therapy.
 
Digital solutions such as Vineti’s, for example, allow for accurate, transparent, repeatable Chain of Identity and Chain of Custody throughout the entire product journey to administration. This gives patients, physicians, and regulators confidence that the correct cells are being handled appropriately and that they can see the progress of each patient-centric product through to infusion.
 
On an industry level, significant standards progress is being made in the CGT industry. The FDA-funded Standards Coordinating Body is driving ahead to get industry and agencies aligned, identify standards gaps, and start closing them. Vineti sits on the executive committee of this group and is actively helping to drive standardization, both with our clients and on an industry-wide level.
 
Regulatory support
As evidenced by the FDA’s statement in January 2019, anticipating more than 200 cell and gene therapy investigational new drugs per year by 2020 and 10 to 20 approvals a year by 2025, regulatory agencies are clearly ramping up for scale. We all need to make the work of these agencies as manageable and predictable as possible.
 
In cell and gene therapies, for the first time, regulatory agencies have required that commercial filings include a description of the traceability system of record, since it’s so critical to patient safety. The Vineti platform has now been part of filing reviews by FDA and EMA regulators, and, in both cases, the reviews have been expedited. We’re working with our biopharmaceutical partners to establish a proven baseline of patient safety infrastructure with which agencies are familiar.
 
Modernizing biotech and pharma IT options
In biotech and pharma, the emphasis has always rightly been on the science. But as the science points to next-gen therapeutics such as cell and gene therapies, leaders are recognizing that enabling digital technologies are also required. Automation and cloud-based technologies, for example, are required in the highly distributed, complex CGT ecosystem.
 
CGT also requires that many stakeholders and component systems collaborate. For example, healthcare providers need to time cell collections to be in sync with manufacturing capacity, so that collected cells don’t expire waiting for a manufacturing slot. Using orchestration tools such as Vineti’s PTM platform, the management of CGT manufacturing processes and patient populations has become increasingly more integrated and dependable. This is especially true as Vineti partners with increasing numbers of component systems, such as couriers, CROs, and hub services.
 
By developing our platform as a centralized, cloud-based solution, we have also been able to provide advanced systems support to our clients, such as “always on” reliability and advanced cybersecurity. A cloud-based, centralized system gives us more agility and speed to identify issues or threats quickly and to apply preventive measures or fixes immediately and universally. With a centralized system, we have visibility into all process components simultaneously and in real time. This advanced, enterprise-grade approach also allows us to manage multiple client data sets, while preserving each individual client’s privacy and intellectual property.
 
Better tracking of outcomes data
In cell and gene therapy, the patient journey doesn’t end at infusion. Some therapies are multi-dose. All have long-term monitoring requirements that require detailed tracking, often for many years.
 
These cell therapies are expensive due to their personalized nature; however, they can be very efficacious and possibly curative. The reimbursement landscape is ever shifting, especially in the United States, and outcomes-based reimbursement is still a developing concept. This reimbursement mechanism will require the proper data capture to ensure that all parties receive the required information to trigger reimbursement.
 
Outcomes data can also provide critical insights that will help make these products better over time. However, these complex data need to be captured in ways that are manageable and usable. It’s one thing to have the data — it’s another to truly make use of it and cycle it back into R&D learnings.
 
Integrating existing healthcare systems with these new cloud-based technologies, tasked with managing the product outside electronic health records, can bring the data into one accurate and single view for regulators, payer, and manufacturers.
 
CP: What are some of the services provided under your partnership agreements?
 
AD: Many of our partners are looking for expert input and collaborative solution development. We collaborate with our partners to understand their challenges, help map out their workflows, and develop the right solution for them. Vineti works with its partners to map their workflows, determine which modules they need, and outline how those modules should be configured. Our engineering teams enable and test the solution each partner needs. Also, GMP validation is essential for these systems, and Vineti handles this in partnership with our clients. We then provide training and work with our clients to get the system readied for “Go Live.”

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Amy is Co-founder and CEO of Vineti, a software technology company with the first commercial, cloud-based platform to expand patient access to life-saving cell and gene therapies. Amy is a long-time leader in biotechnology innovation. Prior to Vineti, Amy was Managing Director of GE Ventures, General Electric’s venture capital subsidiary, where she focused on creating new healthcare businesses in addition to venture investments. Before joining GE Ventures, Amy was Chief Business Officer at Navigenics, a genomics company sold to Life Technologies in 2012. Amy was also the Co-founder and Executive Director of Proposition 71, California's $3 billion stem cell research initiative passed in 2004, as well as Chief of Staff for the resulting state grant oversight agency, the California Institute for Regenerative Medicine.