Bio Platforms
Silexion Therapeutics Unveils Innovative Expanded Development Plan for SIL204 Based on Recent Groundbreaking Preclinical Data
Silexion Therapeutics Corp. recently announced an expanded development plan for its next-generation siRNA candidate, SIL204. This strategy integrates systemic administration to target metastatic progression with…
Ocean Biomedical Announces Grant on Patent Right for Bispecific Antibodies Targeting CHI3L1 & PD1 for Enhanced Tumor Cell Cytotoxicity
Ocean Biomedical, Inc. recently announced the China National Intellectual Property Administration (CNIPA) has granted a notice of grant on patent right for its bispecific antibodies…
Regulus Therapeutics Announces Successful Completion of its Phase 1b MAD Clinical Trial for Autosomal Dominant Polycystic Kidney Disease Treatment
Regulus Therapeutics Inc. recently announced positive topline results from all patients in the fourth cohort in its Phase 1b MAD study of farabursen for the treatment…
Gyre Therapeutics Announces Publication of Protocol for Phase 3 Trial Evaluating F351 for CHB-Associated Liver Fibrosis
Gyre Therapeutics recently announced the publication of the manuscript titled Hydronidone for the Treatment of Liver Fibrosis Associated with Chronic Hepatitis B: Protocol for a…
Galatea Bio Secures $25M to Develop Global Biobank & Enable Precision Health at Scale for All
Galatea Bio has secured $25 million in funding to drive discoveries in next-generation genetic analysis, biomarker discovery, and multi-omics disease modeling. Committed to enabling precision health…
NeOnc Technologies Expands Global Clinical Trial Network With CBCC Global Research to Accelerate Study of Brain Cancer Treatments
NeOnc Technologies Holdings, Inc. recently announced a strategic partnership with CBCC Global Research (CBCC), a premier full-service clinical research organization (CRO). This collaboration will expand NeOnc’s…
CERo Therapeutics Holdings Poised to Initiate Enrollment of Phase 1 Trial of CER-1236 in AML Following Positive FDA Review of Manufacturing
CERo Therapeutics Holdings, Inc. recently announced that the company has received positive review from the US FDA on an amendment to its IND around Chemistry,…
Salarius Pharmaceuticals Merger Partner, Decoy Therapeutics, Announces its Novel Inhibitors Show Promising In Silico Activity Against Measles & Related Viruses
Decoy Therapeutics Inc. recently announced a series of antiviral drug candidates previously designed by its IMP3ACT™ platform to be broadly effective against viruses of the…
Nanoscope Announces Publication of Clinical Data on Vision Restoration in Retinitis Pigmentosa
Researchers with Nanoscope Therapeutics Inc. recently announced the publication of a paper, A synthetic opsin restores vision in patients with severe retinal degeneration, in Molecular…
Porosome Therapeutics Unveils Groundbreaking Discovery in Alzheimer’s Disease Research & Treatment
Porosome Therapeutics, Inc. recently announced its latest development in identifying the secretory dysfunction responsible for Alzheimer’s Disease along with advancements in therapeutic neuronal nanomachines. These innovations…
First Patient Dosed in Immutep’s TACTI-004 Phase 3 Trial in First Line Non-Small Cell Lung Cancer
Immutep Limited recently announced the first patient has been successfully dosed in the Company’s pivotal TACTI-004 Phase 3 trial. TACTI-004 will evaluate Immutep’s eftilagimod alfa,…
Tiziana Life Sciences Announces Yale University Commences Intranasal Foralumab Dosing in Phase 2 Multiple Sclerosis Trial
Tiziana Life Sciences, Ltd. recently announced dosing of new patients at Yale MS Center, which is participating in our multicenter Phase 2 clinical trial evaluating…
Character Biosciences Raises $93 Million to Advance Precision Medicine for Progressive Eye Diseases
Character Biosciences recently announced an oversubscribed $93 million Series B financing round to accelerate the advancement of its pipeline of precision therapies to treat degenerative…
IMUNON Finalizes Phase 3 Study Design With FDA for IMNN-001 in Newly Diagnosed Advanced Ovarian Cancer
IMUNON, Inc. recently announced the US FDA is aligned with the protocol for the Phase 3 pivotal trial, called OVATION 3, of its lead candidate…
Tenaya Therapeutics Publishes Preclinical Data Demonstrating TN-201 Enhances Cardiac Function & Survival in MYBPC3 Cardiomyopathy Models
Tenaya Therapeutics, Inc. recently announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for Myosin-Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy…
Traws Pharma’s Bird Flu Drug Candidate Presented at ICAR
Traws Pharma, Inc. recently announced that positive data supporting the potential for tivoxavir marboxil (TXM) as a bird flu treatment was presented yesterday in a…
Silexion Therapeutics Announces Completion of Innovative Expanded Development Plan for SIL204
Silexion Therapeutics Corp. recently announced it has completed an expanded development plan for its next-generation siRNA candidate, SIL204, which the company’s management will be presenting…
Wugen Announces Dosing of First Patients in Pivotal Trial of Off-the-Shelf, Allogeneic, CD7-Targeted CAR-T Cell Therapy WU-CART-007
Wugen, Inc. recently announced the dosing of the first patients in its pivotal Phase 2 study evaluating WU-CART-007, a potential first-in-class, investigational, anti-CD7 CAR-T cell…
Esperion Aligns With FDA to Initiate Phase 3 Clinical Trials of Bempedoic Acid in Pediatric Heterozygous & Homozygous Familial Hypercholesterolemia
Esperion recently announced that following meetings with the US FDA, it has gained alignment on a regulatory path forward for initiating Phase 3 studies of…
Indaptus Therapeutics Reports New Data Demonstrating Successful Broad Immune System Activation in Weekly Dosing Trial of Decoy20
Indaptus Therapeutics, Inc. recently provided an update on key pharmacodynamic findings from the weekly dosing cohort of its ongoing Phase 1 trial of Decoy20. As…
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).