Bio Platforms
Nutcracker Therapeutics Demonstrates Positive Data on Anti-Prostate Cancer Therapeutic
Nutcracker Therapeutics, Inc. recently presented two posters at the American Association for Cancer Research (AACR) Annual Meeting in San Diego: one showcasing the latest preclinical data…
Regeneration Biomedical Doses First Patient in a First-in-Human Phase 1 Clinical Trial of Stem Cell Therapy Delivered Directly Into the Brain of Patients With Alzheimer’s Disease
Regeneration Biomedical, Inc. recently announced the first patient has been dosed in an FDA-cleared, first-in-human Phase 1 clinical trial of autologous, Wnt-Activated Adipose-Derived Stem Cells…
Wave Life Sciences Announces Continued Momentum in GSK Collaboration & Advancements in siRNA & RNA Editing
Wave Life Sciences Ltd. recently provided an update on its best-in-class small interfering RNA (siRNA) and RNA editing platform capabilities. As part of Wave’s ongoing…
Palisade Bio Enters Strategic Collaboration With Strand Life Sciences to Advance Precision Medicine Approach
Partnership marks a significant value-driving milestone in Palisade Bio’s mission to redefine UC treatment through targeted interventions based on PDE4-related biomarkers….
Alvotech & Teva Announce FDA Approval of Biosimilar to Stelara
Alvotech and Teva Pharmaceuticals recently announced the US FDA has approved SELARSDI (ustekinumab-aekn) injection for subcutaneous use, as a biosimilar to Stelara, for the treatment…
Evaxion Announces Phase 2 Clinical Trial Update: First Patient Completed Dosing With Personalized Cancer Vaccine
Significant Phase 2 clinical trial progress obtained with first patient finalizing EVX-01 vaccine dosing….
Atossa Therapeutics & Quantum Leap Healthcare Announce Clinical Trial to Evaluate (Z)-Endoxifen in Combination With Abemaciclib in Women With ER+/HER2- Breast Cancer
Atossa Therapeutics, Inc. and Quantum Leap Healthcare Collaborative recently announced the initiation of a new study to evaluate Atossa’s proprietary (Z)-endoxifen in combination with abemaciclib…
Voyager Therapeutics Announces Selection of Development Candidate for GBA1 Program in Collaboration With Neurocrine Biosciences, Triggering Milestone Payment
Voyager Therapeutics, Inc. recently announced the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the GBA1 gene therapy program for the potential treatment of Parkinson’s disease….
Kincell Bio & Imugene Announce Strategic Manufacturing & Process Development Partnership
Kincell Bio to acquire Imugene’s North Carolina manufacturing facility for up to $6M USD in upfront and milestone-driven payments over 3 years….
Cullinan Therapeutics Announces Strategic Expansion Into Autoimmune Diseases & $280-Million Private Placement
CLN-978 clinical development to focus exclusively on autoimmune diseases, pursuing systemic lupus erythematosus as a first indication….
SAB Biotherapeutics Provides SAB-142 Trial Update
SAB Biotherapeutics, Inc. recently announced its Chief Medical Officer, Dr. Alexandra Kropotova, MD, MBA, disclosed SAB has completed dosing the third cohort for SAB-142 with…
GRI Bio Expands Intellectual Property Protection for Proprietary Natural Killer T Cell Modulators With Issuance of Patent
GRI Bio, Inc. recently announced the Korean Intellectual Property Office (KIPO) has issued a Notice of Patent Grant for Patent Application No. 10-2017-7018579 titled Prevention…
Organovo Announces Positive Phase 2 Results for FXR314 in Metabolic Dysfunction-Associated Steatohepatitis
Organovo Holdings, Inc. recently released the complete details of its 16-week, randomized, placebo-controlled, multi-center Phase 2 study of the non-steroidal, non-bile acid FXR agonist FXR314…
BC Platforms & NTT Group Announce Official Opening Ceremony for Exclusive Collaboration & Launch of Japanese Precision Medicine Platform
BC Platforms (BCP) recently announces an exclusive collaboration with NTT Life Science Corporation, a company in the medical field of the NTT Group, to accelerate data-driven….
Naobios & Sumagen Sign Exclusive Partnership to Develop HIV Vaccine
Naobios will manage process development and initiate launch of cGMP manufacturing for Phase 2 clinical trials….
Neurona Therapeutics Presents Positive Clinical Update From Cell Therapy Trial in Adults With Drug-Resistant Focal Epilepsy
Neurona Therapeutics presented updated preliminary data from its ongoing open-label Phase 1/2 clinical trial of NRTX-1001 in adults with drug-resistant, unilateral mesial temporal lobe epilepsy…
AviadoBio Announces First Patient Treated in Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia With GRN Mutations
AviadoBio recently announced the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal…
NovelMed Receives FDA Orphan Drug Designation for Treating Paroxysmal Nocturnal Hemoglobinuria; Seeks Partnership
NovelMed recently announced the US FDA has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment….
Asahi Kasei Bioprocess & Axolabs Announce Strategic Partnership to Accelerate Oligonucleotide Therapeutics Development
Asahi Kasei Bioprocess (AKB), part of the Asahi Kasei Group, and Axolabs have recently announced a strategic partnership in the burgeoning field of oligonucleotide….
Indaptus Therapeutics Presents Positive Mechanism of Action Data
Indaptus Therapeutics, Inc, recently unveiled its poster at the 2024 Annual Meeting of the American Association for Cancer Research (AACR) in San Diego on April…
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).