Bio Platforms
OLIGONUCLEOTIDE DELIVERY - Dectin-1 Receptor-Mediated Delivery of Oligonucleotide Drugs Complexed With Schizophyllan Dendritic Cells & Macrophages
Kenji Arima, PhD, and Akihiko Watanabe, PhD, introduce NapaJen Pharma’s technology with a specific focus on solving some of the current challenges of oligonucleotide therapeutics.
MARKET BRIEF - Tumor-Infiltrating Lymphocytes: A New Frontier in Cancer Immunotherapy
Cheryl L. Barton, PhD, and Bianca Piachaud-Moustakis, PhD, believe the role of TIL therapy as an anticancer therapy in melanoma and possibly other solid tumors holds great promise and could become a viable course of treatment in the future.
Nurix Therapeutics & Sanofi Establish Strategic Collaboration
Nurix Therapeutics, Inc. recently announced a global strategic collaboration with Sanofi to discover, develop, and commercialize a pipeline of innovative targeted protein….
Sebia Enters Development Agreement With Sanofi
Sebia recently announced it is entering into an agreement with Sanofi to develop an in vitro diagnostic (IVD) test that mitigates the potential interference caused by……
WPD Pharmaceuticals’ New Discovery for Cancer Targeting Drugs is Granted Patent
WPD Pharmaceuticals recently announced Wake Forest University received a patent from the United States Patent and Trademark Office (USPTO) for patent 105019210 (issued under application…
Oxford BioTherapeutics Initiates Dose-Escalation Portion of Phase I Clinical Trial
Oxford BioTherapeutics Ltd. recently announced the initiation of the dose-escalation portion of its US Phase 1 program for OBT076, a CD205 targeting antibody-drug conjugate (ADC),…
Zai Lab & Novocure Announce First Patient Enrolled in a Phase 2 Pilot Trial
Zai Lab Limited and Novocure recently announced the first patient has been enrolled in a Phase 2 pilot clinical trial evaluating the safety and efficacy…
TYME Announces First Patient Dosed in Pivotal Trial
Tyme Technologies, Inc. recently announced the first pancreatic cancer patient has been dosed in Part 2 of the TYME-88-Panc pivotal trial designed to support approval…
Cytovia Therapeutics & the University of California, San Francisco Enter Partnership to Develop Precision Gene-Edited CAR-NK Cell Therapy
Cytovia Therapeutics and the University of California, San Francisco (UCSF) recently announced they have entered a 3-year research partnership to develop novel precision gene editing…
Acceleron & Fulcrum Therapeutics Announce Pulmonary Research & Discovery Collaboration Agreement
Acceleron Pharma Inc. and Fulcrum Therapeutics, Inc. recently announced they have entered into a collaboration and license agreement to identify small molecules designed to modulate…
Pfenex Earns $15-Million Development Milestone Under its Development & License Agreement
Pfenex Inc. recently announced it has earned a $15-million development milestone under its development and license agreement with Jazz Pharmaceuticals plc. The milestone is associated…
FDA Approves Intra-Cellular Therapies’ Novel Treatment of Schizophrenia in Adults
Intra-Cellular Therapies, Inc. recently announced that CAPLYTA (lumateperone) has been approved by the US FDA for the treatment of schizophrenia in adults. The company expects…
Inceptua Medicines Access & Onconova Therapeutics Announce Pre-Approval Access Collaboration
Onconova Therapeutics, Inc. and Inceptua Medicines Access (a business unit of the Inceptua Group) recently announced they have entered into a collaboration to make available…
Fate Therapeutics Presents its First Off-the-Shelf, iPSC-Derived CAR T-Cell Immunotherapy Program
Fate Therapeutics, Inc. recently announced new in vivo preclinical data for FT819, its first off-the-shelf, iPSC-derived chimeric antigen receptor (CAR) T-cell product candidate, at the…
Retrogenix & Resonant Therapeutics Announce Strategic Alliance
Retrogenix and Resonant Therapeutics, Inc. recently announced they have entered into a non-exclusive strategic partnership to identify the targets of Resonant antibodies directed against the…
Navigen Announces FDA Clearance of its IND Application
Navigen, Inc. recently announced the US FDA has cleared its Investigational New Drug application (IND) for the initiation of a Phase 1 study in healthy…
Bioncotech Therapeutics Announces Oncology Clinical Trial Collaboration With MSD
Bioncotech Therapeutics recently announced it has entered into a Phase 2 clinical trial collaboration with a subsidiary of Merck & Co., Inc., Kenilworth, NJ, known…
Soligenix Completes Enrollment in its Pivotal Phase 3 Clinical Trial
Soligenix, Inc. recently announced it has completed patient enrollment in its Phase 3 "Fluorescent Light Activated Synthetic Hypericin" (FLASH) study for SGX301 (synthetic hypericin) in…
IONTAS & Adaptate Biotherapeutics Enter Collaboration Agreement
IONTAS Limited recently announced it has entered into a collaboration agreement with Adaptate Biotherapeutics to generate and optimize antibodies for novel immuno-oncology targets, including access…
Akcea & Ionis Announce Initiation of NEURO-TTRansform Phase 3 Clinical Trial
Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. recently announced initiation of the NEURO-TTRansform Phase 3 clinical trial for…
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).