Bio Platforms
CARGO Therapeutics Announces Phase 1 Clinical Study of Firi-cel CAR T-Cell Therapy
CARGO Therapeutics, Inc. recently announced The Lancet has published favorable data from a Phase 1, single-center clinical study (NCT04088890) by Stanford Medicine (Stanford), evaluating firi-cel,…
Oragenics Improves Intranasal Drug Formulation for Treating Concussed Patients
Oragenics, Inc. recently announced it has developed a new formulation for its novel neurosteroid. Oragenics’ lead drug candidate for concussion, ONP-002, is a new chemical…
EnPlusOne Biosciences Reveals Breakthrough Enzymatic Synthesis of Leqvio Antisense Strand
EnPlusOne Biosciences, Inc. recently announced it had achieved the successful synthesis of the antisense strand of the commercially approved siRNA drug, Leqvio (inclisiran), a treatment…
Intensity Therapeutics Announces First Patient Dosed in its Global Randomized, Phase 3 Study in Metastatic Soft Tissue Sarcoma
Intensity Therapeutics, Inc. recently announced the first US patient has been dosed in its Phase 3 study to treat metastatic sarcoma (NCT06263231). The trial is…
Preclinical Analyses of TGR-63 Demonstrate Blood-Brain Barrier Permeability & Safety Profile
IGC Pharma, Inc. recently announced preclinical data demonstrating TGR-63’s potential as an effective treatment for Alzheimer’s disease. Analysis of the partition coefficient and mass spectrometry…
Avalo Therapeutics Announces Active IND for AVTX-009, an anti-IL-1β mAb, to Treat Hidradenitis Suppurativa
Avalo Therapeutics, Inc. recently announced that the Investigational New Drug (IND) for AVTX-009, an anti-IL-1β monoclonal antibody (mAb), for the treatment of hidradenitis suppurativa (HS)…
Athira Pharma Announces Last Patient Completed LIFT-AD Clinical Trial of Fosgonimeton in Mild-to-Moderate Alzheimer’s Disease
Athira Pharma, Inc recently announced the completion of dosing for the last patient in the Phase 2/3 LIFT-AD clinical trial evaluating fosgonimeton in people with…
Prelude Therapeutics Announces Clinical Collaboration With Merck
Prelude Therapeutics Incorporated recently announced it has entered into a clinical trial collaboration and supply agreement with Merck (known as MSD outside of the US…
uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase 1/2 Trials for Huntington’s Disease Treatment
uniQure N.V. recently announced updated interim data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing US and…
Obsidian Therapeutics Announces FDA Fast Track Designation for Advanced Melanoma Treatment
Obsidian Therapeutics, Inc. recently announced the US FDA has granted Fast Track Designation to OBX-115, a novel engineered tumor-derived autologous T cell immunotherapy (tumor-infiltrating lymphocyte…
RS BioTherapeutics Announces Research Collaboration With University of Colorado School of Medicine
RS BioTherapeutics recently announced a collaboration agreement with the University of Colorado School of Medicine to evaluate use of its first-in-class, steroid-free therapeutic platform in…
Iktos Acquires Synsight, a Company Specializing in Protein-Protein & RNA-Protein Interactions-Targeted Drug Discovery
Iktos recently announced the acquisition of Synsight, a French biotech company applying AI and high-content cell imaging to the discovery of novel drug candidates. The…
CellProthera & BioCardia Collaborate on Successful Phase 2 Trial of ProtheraCytes for the Treatment of Acute Myocardial Infarction
CellProthera and BioCardia Inc. recently announced success from a collaborative Phase 2 trial of ProtheraCytes for the treatment of acute myocardial infarction (AMI) led by…
HilleVax Reports Topline Data From NEST-IN1 Phase 2b Clinical Study of HIL-214 in Infants
HilleVax, Inc. recently announced topline data results from NEST-IN1. NEST-IN1 is a Phase 2b, randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy, safety, and…
CERo Therapeutics Presents CER-1236 Data Supporting Use in AML
CERo Therapeutics Holdings, Inc. recently announced the presentation of a poster on its lead compound CER-1236 at the Global Cell & Gene Therapy Summit 2024.…
CureVac Initiates Strategic Restructuring to Align Resources With Focus on High-Value mRNA Pipeline Opportunities
CureVac N.V. recently announced a significant strategic restructuring to focus its resources on high-value mRNA projects in oncology and other select areas of substantial unmet…
Medigene AG Expands Patent Portfolio With the Patent Grant for its NY-ESO-1/LAGE 1a Targeted T Cell Receptor
Medigene AG recently announced it has been issued a patent by the Chinese Patent Office protecting its T cell receptor (TCR) targeting NY-ESO-1 (New York…
Apollomics Announces Updated Strategic Focus & Leadership Team Changes
Apollomics Inc. recently announced an updated strategic focus for the clinical development of vebreltinib by focusing on NSCLC patients with Met Amplification, as well as…
Sobi Initiates Rolling BLA to FDA for SEL-212 for the Potential Treatment of Chronic Refractory Gout
Sobi recently announced the initiation of a rolling Biologics License Application (BLA) to the USA FDA for SEL-212. The submission is based on the results…
Cellular Origins Acquires ACTIA Platform IP to Enhance Automated Cell Therapy Manufacturing
Cellular Origins recently announced the acquisition of the ACTIA (Autologous Cell Therapy Industrial Automation) Platform IP, developed by Geoff Hodge whilst CEO of SOTIO Biotech…
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).