Bio Platforms
DRUG & DEVICE DEVELOPMENT - Integration of siRNA, Nanoparticles & Capsule Endoscopy for Treatment of Inflammatory Bowel Disease
Nila Murali, Leia Jiang, and Ravali Bhavaraju highlight a promising technology that can change the way IBD is treated. Current imaging technology can successfully identify inflammation, and current treatments can address active inflammation and manage symptoms.
CELL & GENE THERAPY - It’s Time to Build Infrastructure to Handle the Coming Surge
Fran Gregory says the complexities and innovations associated with the production of cell and gene therapies also necessitates a shift in infrastructure, which will affect manufacturers, distributors, and providers. From development, manufacturing, storage, and delivery to patients; each step in the process requires forging a new path.
Wave Life Sciences Announces Approval of First Clinical Trial Application for RestorAATion-2 Trial of WVE-006 in Individuals With Alpha-1 Antitrypsin Deficiency
Wave Life Sciences Ltd. recently announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of WVE-006, the company’s first-in-class…
ENA Respiratory Announces FDA IND Clearance for First-in-Class Antiviral Innate Immunomodulator
ENA Respiratory recently announced the US FDA has issued a "safe to proceed" notice for its investigational new drug (IND) application for a Phase 1b…
Coave Therapeutics Showcases its ALIGATER Platform for Generating Conjugated AAV Vectors Exhibiting Superior Performance in Delivering Ocular Gene Therapy Through the Suprachoroidal Route
Coave Therapeutics recently announced preclinical studies on its novel Conjugated AAV (coAAV) gene therapy vectors demonstrate promising results for ocular gene therapy. Engineered coAAV vectors,…
Deciphera Pharmaceuticals to be Acquired by ONO Pharmaceutical for $2.4 Billion
Deciphera’s kinase inhibitor expertise and established commercialization platform in key markets will reinforce ONO Pharmaceutical’s pipeline and accelerate global reach….
Corcept Completes Enrollment in Phase 4 Trial
Corcept Therapeutics Incorporated recently announced completion of enrollment in CATALYST, a Phase 4 trial examining the prevalence of hypercortisolism (Cushing’s syndrome) in patients with difficult-to-control…
Oligonucleotides & mRNA Vaccines Dominate in Strategic Alliances for RNA Therapies in Major APAC Countries
RNA-based therapies have already proven and will continue to play an integral role in the development of novel therapeutic agents for diseases in the future.…
CanSinoBIO CSO Shares Latest Results of Innovative Pneumococcal Vaccine
On April 20, the 2024 National Vaccines and Health Conference organized by the Chinese Preventive Medicine Association (CPMA) and the Chinese Center for Disease Control…
Biolojic Design Announces Nektar Therapeutics Has Exercised its License Option to Develop an AI-Designed Agonistic Antibody Targeting TNFR2 for the Treatment of Autoimmune Diseases
Program is the Product of a Research Collaboration between Biolojic Design and Nektar entered in 2021….
Immutep Announces Positive Preliminary Topline Results From TACTI-003 Cohort B
Immutep Limited recently announced preliminary topline results from Cohort B of the TACTI-003 (KEYNOTE-PNC-34) Phase 2b trial evaluating eftilagimod alpha (efti) in combination with MSD’s…
PEP-Therapy & Institut Curie Announce First Patients Dosed in Phase 1b Clinical Trial Evaluating PEP-010 in Ovarian & Pancreatic Cancers
PEP-Therapy and Institut Curie recently announced the dosing of the first patients in a Phase 1b clinical trial evaluating PEP-Therapy’s lead candidate, PEP-010, based on…
XOMA Earns $9-Million Milestone as FDA Grants Accelerated Approval to Day One’s OJEMDA for Relapsed or Refractory BRAF-Altered Pediatric Low-Grade Glioma
XOMA Corporation recently announced it has earned a $9-million milestone related to the US FDA’s approval of Day One Biopharmaceuticals’ New Drug Application (NDA) for…
Barinthus Bio Announces Topline Data From Phase 1b/2 of VTP-200 in Persistent High-Risk Human Papillomavirus Infections
Barinthus Biotherapeutics plc recently announced topline final data from the APOLLO trial (also known as HPV001), a completed randomized, placebo-controlled Phase 1b/2 dose-ranging trial of…
Nykode Therapeutics Initiates Phase 2 Trial of VB10.16 in Second Line HPV16-Positive Cervical Cancer
Nykode Therapeutics ASA recently announced the initiation of the Phase 2 clinical trial VB-C-04. The trial evaluates VB10.16, the company’s off-the-shelf therapeutic cancer vaccine candidate…
Tiziana Life Sciences Announces Study Results From Intranasal Anti-CD3 Foralumab in Multiple Sclerosis Patients
Tiziana Life Sciences, Ltd. recently announced a study related to its lead candidate, foralumab, was highlighted in Neurology Today, the official news source of the…
Kynos Therapeutics Announces Positive Top-Line Results From First-in-Human Phase I Study of its KMO Inhibitor
Kynos Therapeutics Ltd recently announced the key findings from the first-in-human Phase 1 trial of its lead drug candidate, KNS366. KMO is an enzyme that…
Salubris Biotherapeutics Announces $35 Million in Financing & Provides Pipeline Progress Update
Salubris Biotherapeutics, Inc. recently announced a new capital infusion of $35 million to fund continued research and development of clinical and preclinical programs. SalubrisBio also…
Cellares & Bristol Myers Squibb Announce $380-M Worldwide Capacity Reservation & Supply Agreement
Cellares, the world’s first Integrated Development and Manufacturing Organization (IDMO), will allocate multiple Cell Shuttles and its fully automated, high-throughput Cell Q systems across its IDMO Smart Factories in the US, EU, and Japan for Bristol Myers Squibb’s use ….
Neurolentech Signs Technology Access Partnership With Kaerus Bioscience
Kaerus Bioscience will access Neurolentech’s NDD Drug Discovery Platform to support development of its preclinical asset pipeline for numerous genetic syndromes….
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).