Bio Platforms
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…
2020 COMPANY PROFILES & CAPABILITIES
For each participating company, this section presents a detailed summary highlighting their core technologies, capabilities, technologies, and services.
DRUG DEVELOPMENT EXECUTIVE - ProQR: Developing RNA Therapies for Rare Genetic Disorders
Daniel de Boer, Chief Executive Officer of ProQR, discusses the use of RNA technology to directly target the underlying cause of genetic diseases.
Excision BioTherapeutics Presents Data to Support HIV Cure Using CRISPR Gene Editing Technology
On November 20 at the 6th Annual Personalized Nanomedicine Symposium, researchers working with Excision BioTherapeutics, a gene therapy company focusing on curing viral infectious diseases, delivered two oral presentations to review the multiple years of…
Provectus Announces Presentation of Initial Results from Phase 1B Trial
Provectus recently announced that data from the company's ongoing Phase 1b/2 study of lysosomal-targeting cancer immunotherapy PV-10 (rose bengal disodium) in combination with KEYTRUDA (pembrolizumab)…
Amarantus Announces Issuance of Chinese Patent
Amarantus Bioscience Holdings, Inc. recently announced that subsidiary MANF Therapeutics has been issued a Chinese patent covering the use of mesencephalic astrocyte-derived neurotrophic factor (MANF)…
Harpoon Therapeutics & AbbVie Announce Licensing & Option Collaboration
Harpoon Therapeutics, Inc. and AbbVie Inc. recently announced an exclusive worldwide option and license transaction for HPN217, Harpoon’s B cell maturation antigen (BCMA)-targeting Tri-specific T…
Zelluna & Glycostem Announce Development, License & Supply Agreement
Zelluna Immunotherapy and Glycostem Therapeutics BV recently announce they have entered into a development, license, and supply agreement. This collaboration will focus on the development…
Biogen Announces Enrollment Completion of Global Phase 3 Gene Therapy Study
Biogen Inc. recently announced the enrollment of the last patient in the global Phase 3 STAR clinical study, which is evaluating the investigational gene therapy…
Protalix BioTherapeutics & Chiesi Farmaceutici Announce Successful Pre-BLA Meeting
Protalix BioTherapeutics, Inc. and its development and commercialization partner, Chiesi Farmaceutici S.p.A. recently announced that they have completed a successful Type B Pre-Biologics License Application (BLA) meeting with the US FDA regarding….
Atlas Nanotech Targets the World’s Largest Consumer Health Products Companies
Costas, Inc. DBA/Atlas Nanotech, (the Company) is currently in discussions to license their proprietary nanotechnology vitamin a (retinol) based crystalline eye drops known under the…
NervGen Pharma Expands Platform Into Alzheimer's Disease
NervGen Pharma Corp. recently announced a research initiative to advance its proprietary therapeutic technology platform, currently in development for spinal cord injury and multiple sclerosis,…
Flexion Announces Clearance of NDA for Gene Therapy Candidate
Flexion Therapeutics, Inc. recently announced clearance of the company’s Investigational New Drug (IND) application for FX201 in knee osteoarthritis.
Femtogenix Presents Novel Data on Antibody Drug Conjugates
Femtogenix Ltd recently announced data demonstrating the potent efficacy and favorable toxicity profile of a reduced potency analogue from its Pyridinobenzodiazepine (PDD) ADC payload platform…
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).