Bio Platforms
Marinomed Biotech AG Develops First Medicinal Product for Autoimmune Gastritis
Marinomed Biotech AG, a Vienna-based biopharmaceutical company, develops groundbreaking treatment for autoimmune gastritis together with the Medical University of….
Cytovia Therapeutics Partners Enters Research & Licensing Agreement With Inserm
Cytovia Therapeutics recently announced it has entered a research and licensing agreement with Inserm to develop NK engager bi-specific antibodies and iPSC CAR NK cell therapy targeting….
Takeda & Arrowhead Collaborate to Co-Develop & Co-Commercialize ARO-AAT for Alpha-1 Antitrypsin-Associated Liver Disease
Takeda Pharmaceutical Company Limited and Arrowhead Pharmaceuticals Inc. recently announced a collaboration and licensing agreement to develop ARO-AAT, a Phase 2 investigational RNA interference (RNAi)…
Vaxart Announces Dosing First Subject in Phase 1 Clinical Trial of Its Oral Tablet COVID-19 Vaccine
Vaxart, Inc. recently announced the first subject has been dosed in its Phase 1 study of VXA-CoV2-1, an oral tablet COVID-19 vaccine candidate. “We are…
uniQure Announces Enrollment of Next Two Patients in Phase 1/2 Clinical Trial
uniQure N.V. recently announced that two additional patient procedures have been completed in the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s…
Mogrify Introduces Platform to Systematically Identify Epigenetic Switches Driving Cell Identity & Cell Maintenance
Mogrify Limited recently announced the introduction of EpiMOGRIFY. The platform models the epigenetic state of the cell to predict the switches important for cell identity,…
NapaJen Pharma Reports Positive Results From Phase 1 Clinical Trial of Novel RNAi Therapeutic
NapaJen Pharma, Inc. recently announced positive results from the company’s Phase 1 first-in-human clinical trial of NJA-730. NJA-730 is a novel immunosuppressive therapeutic that combines…
Mustang Bio Licenses LentiBOOST Technology From SIRION Biotech
Mustang Bio, Inc. and SIRION Biotech GmbH recently announced a licensing agreement under which Mustang has acquired rights to SIRION’s LentiBOOST technology for the development…
Evaxion Biotech Launches New AI Powered Platform to Enable Faster Response to Emerging Viral Pandemics
Evaxion Biotech recently announced it has received approximately $800,000 in funding from Innovation Fund Denmark for the development of its new RAVEN platform, an integral…
Recipharm Signs Agreement With Arcturus Therapeutics to Support the Manufacture of LUNAR-COV19 (ARCT-021) Vaccine Candidate
Recipharm has recently entered into an agreement with Arcturus Therapeutics, a leading US-based clinical-stage messenger RNA medicines company focused on the development of infectious disease vaccines and….
Bristol Myers Squibb to Acquire MyoKardia for $13.1 Billion
Bristol Myers Squibb and MyoKardia, Inc. recently announced a definitive merger agreement under which Bristol Myers Squibb will acquire MyoKardia for $13.1 billion, or….
PLATFORM TECHNOLOGY - Morphomer(TM) & SupraAntigen(TM) Platforms: Targeting Misfolded Proteins in Neurodegenerative Disorders
Andrea Pfeifer, PhD, details the efforts to develop the therapeutic and diagnostic tools necessary to enable precision medicine approaches targeting the right protein, at the right time, in the right patient.
EXECUTIVE INTERVIEW - Immunic Therapeutics: Developing Next-Generation Oral Drugs in Chronic Inflammatory, Autoimmune & Infectious Diseases
Daniel Vitt, PhD, Immunic’s Chief Executive Officer and President, discusses his company’s pipeline of selective, small molecule therapies aimed at treating chronic inflammatory and autoimmune diseases, and its recent progress.
EXECUTIVE INTERVIEW - Enteris BioPharma: Pioneers in Oral Formulation Development
Dr. Rajiv Khosla, CEO of Enteris BioPharma, discusses how his company’s innovative oral formulation technology is helping the pharmaceutical industry overcome the hurdle of low bioavailability to reshape treatment categories and expand market opportunities with minimal financial and regulatory risks.
Orgenesis Announces Agreement to Acquire Koligo Therapeutics
Orgenesis Inc. and Koligo Therapeutics, Inc. recently announced the two companies have entered into a definitive merger agreement, subject to final closing conditions, with expected completion….
Mereo BioPharma Receives FDA Rare Pediatric Disease Designation
Mereo BioPharma Group plc recently announced that the US FDA has granted Rare Pediatric Disease designation to setrusumab for the treatment of osteogenesis imperfecta (OI).…
Celyad Oncology Announces Clinical Trial Collaboration With MSD
Celyad Oncology SA recently announced it has entered into a clinical trial collaboration with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, through…
Aqualung Therapeutics Awarded NIH Fast-Track Award
Aqualung Therapeutics, an early stage biotech company developing an immune-focused, anti-inflammatory therapeutic platform for unchecked inflammation in patients with serious acute and chronic diseases, has been awarded….
OXGENE Introduces TESSA Technology for Robust & Reproducible AAV Manufacture at Scale
OXGENE recently announced the launch of its scalable, plasmid-free manufacturing system for AAV. OXGENE’s new TESSA technology addresses….
T-knife & Catalent Sign Technology Transfer & Manufacturing Agreement
T-knife GmbH and Catalent recently announced they have signed an agreement to provide technology transfer and CGMP clinical manufacturing of T-knife’s T1367 T-cell receptor (TCR) program…..
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).