Bio Platforms
RNA EDITING - New Editing Technology Enables Body to Repair its Own RNA
Daniel de Boer explains how the Axiomer technology, a powerful RNA-editing technique that enables the body to repair itself, is being developed as a next-generation therapeutic option for genetic disorders.
NANOPARTICLE DELIVERY - Ultra-Small Particles Offer Big Hope in Cancer Theranostics
Kai Ma, PhD, Ulrich Wiesner, and Michelle Bradbury, MD, PhD, believe ultra-small sub-10 nm particles hold unique properties and represent an emerging area of investigation for biomedical applications.
Sarepta Therapeutics Announces Positive & Robust Expression & Biomarker Data
Sarepta Therapeutics, Inc. recently announced positive results from three Limb-girdle muscular dystrophy (LGMD) Type 2E clinical trial participants who received MYO-101. MYO-101 is a novel…
AKCEA-APO(a)-LRx Advances as Novartis Exercises Option to License
Akcea Therapeutics, Inc. recently announced that Novartis has exercised its option to license AKCEA-APO(a)-LRx, a drug to treat patients with elevated levels of lipoprotein(a), or Lp(a), and established cardiovascular disease (CVD).
BrainStorm Announces First Contracted US Clinical Site for Phase 2 Progressive MS Study
BrainStorm Cell Therapeutics Inc. recently announced Cleveland Clinic as the first US clinical site contracted for a planned Phase 2 open-label, multicenter study of repeated…
Mogrify Raises $3.7 Million to Accelerate Mission to Transform Cell Therapy Via Direct Cellular Conversion
Cell Mogrify Ltd (Mogrify) recently announced a second close on its seed funding, bringing the total raised to $3.7 million, and the appointment of Darrin…
ADC Therapeutics Announces First Patient Dosed in Phase I Clinical Trial
ADC Therapeutics recently announced the first patient has been dosed in a Phase I clinical trial evaluating the safety, tolerability, pharmacokinetics, and anti-tumor activity of…
CytoDyn Files for Orphan Drug Designation for Triple-Negative Breast Cancer
CytoDyn Inc. recently announced it was able to reduce by more than 98% the incidence of human breast cancer metastasis in a mouse xenograft model…
Sapreme Technologies to Develop an Oligonucleotide Delivery Platform
Sapreme Technologies has been awarded a $7.71 million grant together with a multidisciplinary consortium including 11 other academic and industrial parties.
Avalon GloboCare Announces Breakthrough in Identifying Human Angiogenic Exosomes/Extracellular Vesicles (EV) Derived From Endothelial Cells
Avalon GloboCare Corp. recently announced its ongoing co-development program with Weill Cornell Medicine, led by Yen-Michael Hsu, MD, PhD, Director of cGMP Cellular Therapy Facility…
Aduro Announces First Patient Dosed in Phase 1 Study
Aduro Biotech, Inc. recently announced the first patient has been dosed in a Phase 1 trial of ADU-S100 (MIW815), a novel stimulator of interferon genes…
Genocea Announces Private Placement Financing of Up to $39 Million
Genocea Biosciences, Inc. recently announced it has entered into a private placement with certain existing and new investors providing for the purchase of up to…
Active Biotech's Partner NeoTX Enters Clinical Collaboration With AstraZeneca
Active Biotech recently announced its partner NeoTX entered a clinical collaboration with AstraZeneca Group Plc global biologics research and development arm, MedImmune, to support Phase…
Passage Bio Launches With $115.5 Million Financing to Develop AAV-Delivered Therapeutics
Passage Bio recently debuted with a $115.-million Series A financing led by OrbiMed Advisors and joined by Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures.
Codexis Announces Nestlé Health Science Exercises Option for Exclusive Global License: Triggers Milestone
Codexis, Inc. recently announced that Nestlé Health Science has exercised its option to obtain an exclusive license for the global development and commercialization of Codexis’ novel, orally delivered enzyme CDX-6114 for the management of phenylketonuria (PKU), an orphan metabolic disorder.
ProteoNic Biotechnology Licenses Protein Production Technology to Eli Lilly
ProteoNic Biotechnology BV recently announced that it licensed its 2G UNic technology for boosting recombinant protein production to Eli Lilly and Company. Under the agreement,…
argenx & Halozyme Enter Global Collaboration & License Agreement
argenx and Halozyme Therapeutics, Inc. recently announced a global collaboration and license agreement that enables use by argenx of Halozyme’s ENHANZE drug delivery technology to…
uniQure Announces First Patient Treated in Pivotal Trial
uniQure N.V. recently announced it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.
MeiraGTx Enters Strategic Collaboration With Janssen
MeiraGTx Holdings plc recently announced it has entered into a broad strategic collaboration with Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson…
Menlo Therapeutics Granted Breakthrough Therapy Designation
Menlo Therapeutics Inc. recently announced the US FDA has granted Breakthrough Therapy Designation for serlopitant for the treatment of pruritus associated with prurigo nodularis (PN).…
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).