PLATFORM TECHNOLOGY

PLATFORM TECHNOLOGY – Pilot Plant Scale Up

• A platform is a group of technologies that are used as a base upon which other applications, processes or technologies are developed
• It is an environment for building and running applications, systems and processes
• They can be viewed as toolsets for developingand operating customized and tailored services

Introduction

• Platform technologies are considered a valuable tool to improve efficiency and quality in drug product development
• Furthermore, such a platform enables a continuous improvement by adding data for every new molecule developed by this approach, increasing the robustness of the platform
• Technological platforms aid in efficient drug discovery and development
• These platforms range from vast chemical libraries, ultra-high throughput screening and huge genetic databases in discovery, to predictive toxicology platforms, cutting-edge ‘omics’ and even deep-seated knowledge of particular therapeutic areas in development
• All these ‘platforms’ have two things in common: They can be used on any (or many) development candidate assets, and they cost huge sums to establish in the first place
• Hence they are restricted to the largest pharmaceutical companies
• Only when you have hundreds of active projects can you justify the cost of creating and operating these platforms

It is access to these platforms that keeps the big companies ahead in the race to discover and develop the best medicines

Paper- technology platform

• Paper is predominantly composed of cellulose fibers that have an inherent ability to wick fluids by capillary action; it provides an interesting diagnostic platform that is inexpensive, easily obtained, and eco-friendly
• Paper has been used in various types of biologically relevant applications including paper-based molecular assays, paper- based ELISA (P-ELISA), paper-based nucleic acid assays, and paper-based cell assays
• Paper is not only very suitable for diagnostics but could provide a more advantageous platform than current plastics- based platforms for drug discovery, and would be useful for accomplishing in vitro pre-compound screening steps while offering a possible solution to several economic obstacles inherent in the pharmaceutical industry

ADVANCELL- technology platform

• ADVANCELL’s technology enables new and improved formulations of existing active principles by encapsulating them in nanosystems based on its technology, to improve delivery of drugs to the appropriate site and opens up new routes of administration
• Encapsulation of active molecules is also preserving their integrity and activity
• The technology has been initially developed at the University of Santiago de Compostela in Spain and is extensively patent protected
• ADVANCELL has further developed and expanded the application areas of the technology so that it can now be applied on an industrial scale
• The technology can significantly improve the bioavailability of complex molecules due to its sub-micrometric size and adhesive systems for a higher time of contact to skin
• It is also flexible, encapsulating a broad range of active principles and its systems can be adjusted to achieve desired properties
• In addition, the technology is robust and versatile
• Chemical stability and solubility of the active molecule
• High drug loadings can be achieved
• High encapsulation efficiency
• Developed industrial process and scalability
• Stable, simple and solvent-free technologies
• Reformulation of drugs near patent expiration
• Development of drugs previously thought impossible
• New administration routes for a variety of molecules

Arcelis® technology platform

• Argos Therapeutics is an immune-oncology company developingindividualized  immunotherapies  for the treatment of cancer using its Arcelis® technology platform to capture  mutated  and  variant  antigens  that  are  specific  to each patient’s disease
• This precision immunotherapy technology is potentially applicable to a range of different cancers and is designed to overcome many of the manufacturing and commercialization challenges that have impeded other personalized, cell-based immunotherapies says
• The Arcelis process uses only a small tumor or blood sample and the patient’s own dendritic cells, which are optimized from cells collected by a leukapheresis procedure
• The activated, antigen-loaded dendritic cells are formulated with the patient’s plasma and administered via intradermal injection
• A single production run makes enough product to continuously treat the patient for several years, and Argos has developed an automated manufacturing process to support post-launch commercial demand
• Argos believes its Arcelis technology platform can also be used to create immunotherapies for other chronic infectious diseases that don’t respond to current treatments

Gemicel^TM technology platform

• Targeted drug delivery to the lower gastrointestinal (GI) tract is difficult to achieve
• Assembly Biosciences’ Gemicel^TMis a patent-pending platform technology that allows for targeted delivery of a range of agents in an oral capsule to the GI tract, including the colon delivery technology to be straightforward, efficient, and cost-effective
• Gemicel’s novel formulations, coating and encapsulation technology, and dual-release system are designed to enable oral targeted delivery of live biotherapeutics, such as vegetative bacteria and bacterial spores, vaccines, complex macromolecules, and genetic materials, as well as small molecules and other agents
• Human clinical scintigraphy studies performed by Assembly has confirmed that Gemicel can successfully deliver bolus doses to specific regions of the lower GI tract
• The scale-up and manufacture of the Gemicel delivery technology to be straightforward, efficient, and cost-effective
• Gemicel capsules do not require refrigeration or special handling
• Gemicel achieves its targeting effects by leveraging parameters that vary in different parts of the GI tract, especially changes in pH
• The Gemicel capsule is formulated to release its therapeutic payload in targeted sections of the GI tract based on their characteristic pH levels
• Gemicel capsules have inner and outer compartments that can be designed to dissolve at different pH levels, making it possible to deliver two doses of drug in two locations, or to deliver two different therapies to different parts of the GI tract using a single capsule

ThermoDox® technology platform

• Celsion is an oncology company dedicated to the development and commercialization of cancer drugs based on two clinical-stage technology platforms
• The most advanced program is a heat-mediated, tumor- targeting drug delivery technology that employs a novel heat- sensitive liposome
• The technology is engineered to address a range of difficult-to-treat cancers
• The first application of this platform is ThermoDox®, a lyso-thermosensitive liposomal doxorubicin (LTLD), whose novel mechanism of action delivers high concentrations of doxorubicin to a region targeted with the application of localized heat above 40°C, just above body temperature
• In one of its most advanced applications, LTLD, when combined with radio frequency thermal ablation (RFA), has the potential to address a range of cancers
• It is currently in a Phase III clinical trial for the treatment of primary liver cancer

PETization technology platform

• PETization is the proprietary platform technology of veterinary therapeutic biologics company Nexvet
• It rapidly converts monoclonal antibodies (mAbs) among species, with the end products being 100% species-specific
• Nexvet has clinically validated PETization in dogs, cats, and horses with its portfolio of anti-nerve growth factor (NGF) mAbs
• Dogs, in particular, are susceptible to many of the same diseases and pathologies that afflict humans, such as diabetes, cancer, and inflammatory disease
• Clinical studies in animals with disease, therefore, can be a useful intermediary between traditional preclinical models and human clinical trials
• These proofs-of-concept are usually performed with mouse mAbs in engineered mouse models of disease with sometimes questionable validity for human disease
• “PETization enables rapid production of fully caninized mAbs, with high affinity and low immunogenic potential, suitable for testing in dogs with spontaneous/natural disease”

Atlantis™ technology platform

• Marine organisms present potent compounds with potential to be the next blockbuster cancer drug, the newest weapon in humanity’s fight against antibiotic resistance or a novel non-opioid pain medicine
• But identifying these unique compounds and understanding how they might work as a drug is a complex task requiring specialized training and equipment
• After obtaining biological samples from remote marine environments, scientists must isolate and understand the chemistry of novel marine-derived compounds
• Marine compounds tend to be more chemically unique and less amenable to lab synthesis or fermentation
• To significantly accelerate the process of marine-inspired drug development, biotechnology company Sirenas has developed the Atlantis™ platform, a data-driven approach to documenting, analyzing, and synthesizing compounds that show promise as a medicine
• Sirenas works with research institutions to collect samples of specific organisms — usually sponges, cyanobacteria, and algae

Vical Inc technology platform

• Vical Incorporated develops biopharmaceutical products for the prevention and treatment of chronic and life-threatening infectious diseases
• The company’s core technology platform is based on plasmid DNA vectors designed to express various proteins of interest after injection into muscle tissue
• Plasmid vectors are the vehicles used to drive recombinant DNA into a host cell and are a key component of molecular cloning ii
• Vical Incorporated develops biopharmaceutical products for the prevention and treatment of chronic and life-threatening infectious diseases
• The company’s core technology platform is based on plasmid DNA vectors designed to express various proteins of interest after injection into muscle tissue
• The plasmid DNA contains gene expression elements to regulate high-level expression of any gene sequence that is genetically engineered into the vector
• Plasmid DNA is manufactured by a relatively simple fermentation process in E. coli and doesn’t require handling any pathogen
• A powerful manufacturing attribute is that only a single manufacturing process is required regardless of which gene sequence is encoded by a plasmid DNA.
• Second, vaccine stability (a limitation with some live- attenuated vaccines) is not an issue with plasmid DNA vaccines as they can be stored frozen for long periods with minimal loss of potency
• Finally, plasmid DNA vaccines can elicit both arms of the adaptive immune response (T-cell- and antibody-mediated responses) in contrast to inactivated and protein subunit- based vaccines that generally elicit high levels of antibody responses

FAQ

What is platform technology in the context of pharmaceutical manufacturing?

Platform technology refers to a standardized set of processes, equipment, and methodologies that can be applied across various drug products within a pharmaceutical company. It streamlines manufacturing and scale-up processes.

How is platform technology applied in pilot plant scale-up?

Platform technology is applied by using standardized processes, equipment, and formulation templates to efficiently scale up the production of various drug products, reducing development time and costs.

What are the key advantages of using platform technology in pilot plant scale-up?

The advantages include increased efficiency, cost savings, enhanced quality control, and flexibility in adapting processes for different drug formulations.

Are there any challenges associated with platform technology in pharmaceutical manufacturing?

Challenges may include the need for specialized processes for certain products, ensuring regulatory compliance, and staying up-to-date with technological advancements and regulatory changes.

How does platform technology contribute to cost savings during pilot plant scale-up?

Platform technology reduces costs by standardizing processes and equipment, allowing for equipment reusability, and optimizing resource utilization.

Is platform technology suitable for all types of pharmaceutical products?

While platform technology is highly beneficial, some specialized products may require unique processes that cannot fully leverage this approach. Each product’s specific requirements must be considered.

How important is regulatory compliance when using platform technology in pharmaceutical manufacturing?

Regulatory compliance is crucial. Pharmaceutical companies must ensure that standardized processes meet the regulatory requirements for each specific product manufactured using platform technology. Documentation and validation are key components of compliance.

Can platform technology be adapted to new and emerging drug formulations?

Yes, platform technology is designed to be flexible and adaptable. It allows pharmaceutical companies to modify and adapt processes as needed for different drug formulations without starting from scratch for each product.

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