Bio Platforms
Vaxart Reports Positive Clinical Data Demonstrating its Second-Generation Vaccine Technology Produces Much Stronger Antibody Responses Than its First-Generation Technology
Vaxart, Inc. recently reported positive topline results from the Phase 1 clinical trial evaluating its second-generation oral pill norovirus vaccine constructs head-to-head against its first-generation…
Adcendo ApS Announces First Patient Dosed in Phase 1/2 ADCElerate1 Trial of ADCE-D01
Adcendo recently announced the first patient has been dosed in the Phase 1/2 ADCElerate1 clinical trial evaluating ADCE-D01 in patients with metastatic and/or unresectable soft…
Avidity Biosciences Announces the Accelerated Approval Regulatory Pathway in the US is Open for Del-Brax & Initiates the Global, Confirmatory Phase 3 FORWARD Study in FSHD
Avidity Biosciences, Inc. recently announced the accelerated approval regulatory pathway in the United States is open for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral…
OBI Pharma Enters ADC Collaboration With TegMine Therapeutics Utilizing GlycOBI & TegMiner Enabling Technologies
OBI Pharma, Inc. and TegMine Therapeutics, Inc. have entered into an ADC-related Master Services Agreement (MSA). Under the terms of the MSA, OBI grants TegMine…
AstriVax Therapeutics Initiates Clinical Development of a Hepatitis B Immunotherapy Using Launch-iT Technology
AstriVax Therapeutics NV recently announced the start of clinical development with AVX70371 for chronic hepatitis B virus (HBV) infection. The phase I RUBY study (NCT06989788)…
SPECIAL FEATURE- Outsourcing Formulation Development & Manufacturing: CDMOs Are Making Their Supply Chains More Resilient & Secure
Contributor Cindy H. Dubin speaks with many CDMO professionals to get their insights and perspectives on the current state of the industry.
HCW Biologics Completes Deliverable & Solidifies License Agreement With WY Biotech
HCW Biologics Inc. and WY Biotech Co., Ltd. recently announced WY Biotech has completed its due diligence related to HCWB’s technology transfer report, including the…
Elicio Therapeutics Secures $10 Million in Financing
Elicio Therapeutics, Inc. recently announced it has entered a $10-million senior secured promissory note with existing investor, GKCC, LLC. The proceeds from the Note Financing,…
Trethera Secures New License, Strengthening TRE-515 Intellectual Property Portfolio & Extending Market Exclusivity to 2045
Trethera Corporation recently announced it entered an exclusive, worldwide licensing agreement with the University of California, Los Angeles (UCLA). The license expands Trethera’s intellectual property…
PROTEIN BARCODING - Streamlining mRNA Therapeutic & Vaccine Development
Meredith Carpenter, PhD, reviews how lipid nanoparticles have emerged as a powerful tool for delivery due to their low immunogenicity compared to viral vectors, along with their ability to encapsulate and protect nucleic acids and facilitate their entry into cells.
DRUG ADMINISTRATION - Protecting Patient Data in Cell & Gene Therapy: The Role of Tech Platforms
Matthew Lakelin, PhD, explores the unique data challenges in CGT, emphasizing the need for robust technology platforms to ensure patient privacy and security.
Kymera Therapeutics Announces Positive First-in-Human Results From Phase 1 Trial of a First-in-Class, Oral STAT6 Degrader
Kymera Therapeutics, Inc. recently announced positive clinical results from the Phase 1 healthy volunteer study of KT-621, its first-in-class, oral STAT6 degrader medicine. “Our primary objective…
Curevo Enrolls First Participants in Phase 2 Extension Trial
Curevo Vaccine recently announced the enrollment of the first patients in a Phase 2 extension trial of its investigational shingles vaccine, amezosvatein. “I’m so proud…
BioNTech & Bristol Myers Squibb Announce Global Strategic Partnership to Co-Develop & Co-Commercialize Next-Generation Bispecific Antibody Candidate Broadly for Multiple Solid Tumor Types
BioNTech SE and Bristol Myers Squibb recently announced they have entered into an agreement for the global co-development and co-commercialization of BioNTech’s investigational bispecific antibody…
CERo Therapeutics Doses First Patient With CER-1236 in Phase 1 Clinical Trial for Acute Myeloid Leukemia and is Advancing Through Protocol-Defined Evaluations
CERo Therapeutics Holdings, Inc. recently announced it has dosed the first patient in its Phase 1 clinical trial of CER-1236. The patient was dosed at…
Cellares & Mitsui Fudosan Announce the First IDMO Smart Factory for Commercial-Scale Cell Therapy Manufacturing in Japan
Cellares, in collaboration with Mitsui Fudosan, recently announced the establishment of Japan’s first next-gen commercial production site for CAR-T cell therapies. The facility is under…
Sibeprenlimab's Priority Review Highlights Potential to Differentiate in IgAN Space
Otsuka Pharmaceutical recently announced that the FDA has accepted for review the Biologics License Application (BLA) for sibeprenlimab, which acts as A Proliferation Inducing Ligand…
Cartesian Therapeutics Announces First Participant Enrolled in the Phase 3 Trial of Descartes-08 in Patients With Myasthenia Gravis
Cartesian Therapeutics, Inc. recently announced the first participant has been enrolled in its Phase 3 AURORA trial of Descartes-08 in patients with myasthenia gravis (MG).…
Alvotech & Advanz Pharma Extend Strategic Partnership to Commercialize Three Additional Biosimilars in Europe
Alvotech and Advanz Pharma recently announced they have entered an agreement to expand their commercial partnership to cover three additional biosimilar candidates. The new agreement…
Candel Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for CAN-2409 for the Treatment of Prostate Cancer
Candel Therapeutics, Inc. recently announced the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CAN-2409 (aglatimagene besadenovec), the company’s biological immunotherapy lead…
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).