Bio Platforms
GENE-EDITING TECHNOLOGY - How CRISPR-Cas9 Technology Will Play a Vital Role in the Future of Human Therapeutics & Drug Discovery
Kevin Holden, PhD, explores how this technology can be utilized in research efforts toward the development of new therapies and how it will play a vital role in the future of biopharma and drug discovery.
ANTIBODY THERAPEUTICS - Teneobio’s Next Generation of Multispecific Antibody Therapeutics
Omid Vafa, PhD, MBA, reviews unique technologies, including a transgenic rat platform expressing human heavy chain antibodies, and a state-of-the-art sequence-based discovery engine, to create novel multispecific antibodies for various therapeutic indications.
CANCER IMMUNOTHERAPY - Building on Initial Successes to Improve Clinical Outcomes
Allan B. Haberman, PhD, provides an updated discussion of approved and clinical-stage agents in immuno-oncology, including recently approved agents. He also addresses how researchers and companies are attempting to build on prior achievements in immuno-oncology to improve outcomes for more patients.
ONCOLOGY DIAGNOSTICS - Advancements Paving the Way for More Tailored Drugs
Divyaa Ravishankar, MS, says pharmaceutical companies have been subjected to a wide variety of external forces compelling them to get innovative about development of new platforms, liaise with new partners, leverage big data toward precision and predictive diagnosis, and identify new markers.
GENE & CELL THERAPY - Its Growing Potential to Disrupt Drug Research & Healthcare Delivery
Jeff Galvin believes as the pace of gene and cell therapies accelerates over this next decade, potential cures for chronic diseases, cancer cures, and autosomal defect cures will result, and the efficacy of new therapeutics may move as much as $500 billion from traditional pharmaceuticals to gene technologies.
SPECIAL FEATURE - Platform Technologies - Derisking & Transforming Drug Development
Contributor Cindy H. Dubin, in this second annual report, speaks with several exciting and innovative companies whose platform technologies are transforming drug development.
NANOPARTICLES - A Revolution in the Development of Drug Delivery Vehicles
Tim Leaver explains how his company has developed a proprietary technology for the rapid development of nanoparticles and seamless scale-up for clinical studies and commercial production, and how it is transforming the development and manufacturing of a range of nanoparticle formulations from a hit-and-miss affair to a standardized process, accelerating novel nanomedicines from the bench to the clinic.
GALECTIN-DIRECTED THERAPIES - Targeting Galectin-3 Protein in Drug Development
Peter G. Traber, MD, believes we are only at the beginning of understanding the full potential of gal-3 targeted therapy, and the future likely holds additional high affinity, specific, galectin inhibitors that are bioavailable by routes other than the two currently in development.
EXECUTIVE INTERVIEW - PCT: Manufacturing the Future of Cell Therapies
Robert A. Preti, PhD, discusses his company’s critical distinction from most other manufacturing partners, steady growth, and the primary challenges facing the cell therapy industry.
EXECUTIVE INTERVIEW - Oasmia Pharmaceutical: Commercializing Technologies While Pursuing the US Market
Julian Aleksov, Executive Chairman of Oasmia Pharmaceutical, discusses his company’s efforts to enter the US market, its strategy to increase commercial adoption, and why it believes its underlying drug delivery system technology is significant not only within the oncology sector, but the entire pharmaceutical industry.
ANTIMICROBIAL RESISTANCE - MGB: The Minor Groove Binder
Dawn A. Firmin, MSc, PhD, explains how MGB has dedicated its focus to the development of a new class of small molecules, with specific antibacterial activity against susceptible and resistant bacteria.
AAV VECTOR MANUFACTURING - Challenges & Opportunities in the Manufacturing of AAV Vectors Used in the Delivery of Gene Therapy Treatments
Daniel C. Smith, PhD, indicates there remains a clear need for improved process productivities, and the need to develop manufacturing processes that can be applied to a wide number of AAV-based viral vector therapeutic candidates.
CELL THERAPY - PLX-R18: Cell Therapy for Treatment of Acute Radiation Syndrome & Bone Marrow Failure Diseases
Racheli Ofir, PhD, and Noa Sher, PhD, report on studies showing that PLX-R18 is a strong candidate for the treatment of H-ARS as well as a plethora of bone marrow failures with similar symptomatology.
THERAPEUTIC FOCUS - GSNOR Inhibition to Stabilize & Improve Mutant CFTR Processing
Steven Shoemaker, MD, reviews how cavosonstat represents a safe and effective option for patients with CF with at least one copy of the F508del-CFTR mutation; and that when used with correctors and potentiators, improves patient outcomes including lung function.
EXECUTIVE INTERVIEW - Viral Gene: Protein-Targeting Cancer Vaccine Could Boost Survival Rates
Harry A. Arena, MBA, President & CEO of TDT, Chris Kim, President & General Counsel, Viral Gene, Inc., and Dr. Scott Waldman, Professor & Chair of Sidney Kimmel Medical College’s Department of Pharmacology & Experimental Therapeutics at Thomas Jefferson University, discuss the unique characteristics of the vaccine, the patients who will benefit the most, and how a research team captured the attention of investors.
PLATFORM TECHNOLOGY - The 3DNA® Platform for Targeted Drug Delivery
Robert C. Getts, PhD, and Jessica Bowers review how the 3DNA platform is composed entirely of noncoding DNA assembled through the sequential hybridization of single strands of DNA into a network of double-stranded nucleic acid having a controlled architecture, and multiple attachment sites for drug and targeting molecules.
PROTEIN THERAPEUTICS MARKET - Technology Advances Spur Market Growth of Protein Therapies
Laurie L. Sullivan and Shalini S. Dewan, BCC Research Analysts, believe with the advent of genetic engineering and recombinant DNA technology, it is now possible to produce a wide variety of human proteins, and that these novel technologies have lifted the market for therapeutic proteins to new heights.
SPECIAL FEATURE - Platform Technologies: Not Just for Big Pharma
Contributor Cindy H. Dubin recently spoke with several companies that are debunking the theory that access to the latest technological platforms to aid efficient drug discovery and development is limited to Big Pharma, which can more easily justify the costs of creating and operating innovative platforms.
CARBON NANOTUBES - MGMR™ - A Medical-Grade Carbon Nanotube Designed for Medical Applications
Joseph S. Dillon, PhD, MBA, and Lainie Mulvanny discuss the transformation of CNTs into a unique composition of matter, marking a complete departure from the dirty, tangled micron bundles of CNTs that frustrated medical researchers for years.
PROTEIN CRYSTALS - Reshaping Traditional Biotherapeutic Formulations
Don Paul Kovarcik, MBA, and William Wittbold, MS, indicate that while protein therapeutics have enjoyed considerable commercial success throughout the past 3 decades, there still remain formulation and delivery challenges.
What are Bio Platforms?
Platforms (or asset-independent technologies to capture all kinds of capabilities that can be leveraged across many different drug candidate assets rather than just discovery tools that the term ‘platform’ immediately brings to mind) are ubiquitous in modern pharma. They are the product of an arms race, to secure access to the best capabilities in key areas.
Platform technologies are considered a valuable tool to improve efficiency and quality in drug product development. The basic idea is that a platform, in combination with a risk-based approach, is the most systematic method to leverage prior knowledge for a given new molecule. 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.
But it has often been said that access to the latest technological platforms to aid efficient drug discovery and development is limited to Big Pharma, which can more easily justify the costs of creating and operating these platforms.
Benefits of Bio Platforms
Platform technologies have the ability to radically improve upon current products and generate completely novel products. In this sense, they open up new arenas for drug discovery and development, potentially increasing the number of therapeutic options for patients. Once a single compound or therapeutic has been generated and demonstrates a clinical benefit in patients, it is more likely this platform technology can successfully be applied to other therapeutic areas, derisking future compounds/products.
Complex drugs by their very nature are challenging and costly to manufacture. This, in turn, translates into higher costs for patients and other payers. In order to provide safe and effective therapies at a reasonable price, it is necessary for the industry to develop manufacturing technologies that reduce costs and provide a consistent product. While the initial investment may be larger, manufacturing costs will be lower over time as the manufacturing process is solidified.
Scale and Investment of Bio Platforms
Despite the initial upfront costs, platform technologies inevitably provide pragmatic solutions to production challenges, while yielding safer and more effective therapeutic products. It has often been said that one of the key features that distinguishes “Big Pharma” from biotech is access to the latest technological platforms to aid 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, and in a few cases each time they are used as well. Hence their restriction to the largest pharmaceutical companies (and a few of the so-called “big biotechs” that are, in many ways, indistinguishable from the old-guard pharma).
Only when you have hundreds of active projects can you justify the cost of creating and operating these platforms. Or so the mantra goes. It is access to these platforms that keeps the big companies ahead in the race to discover and develop the best medicines (or at least counterbalance the disadvantages of being large and slow-moving, depending on your point of view). But is that just an assertion? How much evidence is there to support the proposition that the efficiency gains due to these platforms outstrips the cost of creating and maintaining them?
Keeping these technologies “cutting edge” has become so expensive that increasingly we hear pharma companies talking of “pre-competitive” approaches to develop the next generation. A group of companies might develop a platform capability they then share. The principle goal of such initiatives is to access even grander and more expensive tools than individual companies could afford, rather than to dramatically cut costs (although sharing platforms rather than developing the same thing in parallel in each silo should at least keep a lid on rising costs).