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 developing and 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 developing individualized 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
GemicelTM technology platform
• Targeted drug delivery to the lower gastrointestinal (GI)
tract is difficult to achieve
• Assembly Biosciences’ GemicelTM is 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
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