Bio Platforms
Genezen Breaks Ground on cGMP Lentiviral Vector Production Facility
Genezen Laboratories, Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO) focused on early-phase process development, vector production, and analytical testing services,…
uniQure to Acquire Corlieve Therapeutics & Advance its Gene Therapy Program to Treat Temporal Lobe Epilepsy (TLE)
uniQure N.V. recently announced it has entered into a definitive agreement to acquire Corlieve Therapeutics and its lead program, which will be known as….
vTv Therapeutics & Cantex Pharmaceuticals Announce Strategic Licensing Agreement for Development & Commercialization of Cancer Treatment
vTv Therapeutics Inc. and Cantex Pharmaceuticals, Inc. recently announced they have entered into a licensing agreement under which Cantex has obtained exclusive worldwide rights to….
Processa Pharmaceuticals Enters Licensing & Development Agreement With Ocuphire Pharma
Processa Pharmaceuticals, Inc. recently announced it has entered into a licensing agreement with Ocuphire Pharma, Inc. to license in RX-3117. RX-3117 is an oral, anticancer…
Onconova Therapeutics Announces Preclinical Data Demonstrating the Synergistic Anti-Cancer Activity of Rigosertib Combined With Immune Checkpoint Blockade
Onconova Therapeutics, Inc. recently announced the publication of a preclinical study in the journal Molecular Cancer. The study, titled Novel Induction of CD40 Expression by…
BioNTech Announces First Patient Dosed in Phase 2 Clinical Trial of mRNA-Based BNT111 in Patients With Advanced Melanoma
BioNTech SE recently announced the first patient has been treated in its BNT111 Phase 2 cancer vaccine trial (2020-002195-12; NCT04526899). The study is evaluating the…
Biogen Announces First Patient Dosed in Phase 3 Systemic Lupus Erythematosus Study
Biogen Inc. recently announced the first patient has been dosed in the global clinical study, TOPAZ-1. The Phase 3 study will evaluate the clinical efficacy…
Evonik & Stanford University Sign Research Collaboration: Evonik to Market & Develop New Drug Delivery Platform for mRNA & Gene Therapy
Evonik is working with Stanford University on a technology to deliver mRNA to tissues and organs that goes beyond the capabilities of lipid nanoparticles (LNPs)…..
Jounce Therapeutics Achieves First Milestone in Exclusive License Agreement With Gilead Sciences
Jounce Therapeutics, Inc. recently announced the US FDA clearance of its Investigational New Drug (IND) application for JTX-1811, an anti-CCR8 antibody, for which Gilead Sciences, Inc. has exclusive rights….
iTeos Therapeutics & GSK Announce Development & Commercialization Collaboration
iTeos Therapeutics and GlaxoSmithKline plc recently announced an agreement to co-develop and co-commercialize EOS-448, an anti-TIGIT monoclonal antibody currently in Phase 1 development as a….
VERAXA Biotech & Quadira Biosciences Enter Partnership on ADC Development
VERAXA Biotech GmbH recently announced it has entered into a partnership agreement with Quadira Biosciences AG, Zug, Switzerland, to jointly develop a suite of novel ADCs for the….
Inceptor Bio Launches Next-Generation CAR-T, CAR-M, & NK/NKT Platform Focused on Advancing Cell & Gene Therapies to Cure Difficult-to-Treat Cancers
Inceptor Bio recently announced it is developing multiple next-generation cell and gene therapy platforms to cure difficult-to-treat cancers. Inceptor Bio will create a portfolio of…
Cue Biopharma Announces First Patient Dosed in Part B Patient Expansion of CUE-101 Phase 1 Monotherapy Trial in HPV+ Second Line & Beyond HNSCC
Cue Biopharma, Inc. recently announced it has dosed the first patient in the Part B expansion phase of its Phase 1 monotherapy clinical trial of…
Cocrystal’s Lead COVID-19 Antiviral CDI-45205 Shown to be Active Against SARS-CoV-2 & Two Prominent SARS-CoV-2 Variants
Cocrystal Pharma, Inc. recently announced its lead preclinical SARS-CoV-2 3CL protease inhibitor CDI-45205 is active against SARS-CoV-2 and two prominent SARS-CoV-2 variants. A third-party laboratory…
Liminatus Pharma R&D Consortium Companion Diagnostic YN 1203 Demonstrates ~70% Sensitivity & Specificity in Clinical Testing
Liminatus Pharma recently announced its companion diagnostic to support checkpoint inhibitor therapies, YN1203 CDx liquid biopsy test (YN1203 ELISA Kit), has demonstrated….
Cyclerion Therapeutics Announces Global Licensing Agreement With Akebia Therapeutics
Cyclerion Therapeutics, Inc. recently announced it has entered into an exclusive, global license agreement with Akebia Therapeutics, Inc., a leading biopharmaceutical company focused on kidney disease, for the….
Bayer to Advance Two First-of-its-Kind Cell & Gene Therapies for Parkinson’s Disease
Bayer AG recently announced that BlueRock Therapeutics (BlueRock) successfully administered the first dose of its pluripotent stem cell-derived dopaminergic neurons, named DA01, to a….
Akoya & AstraZeneca to Collaborate on Spatial Biology Approach to Profiling Tumor-Immune Biology
Akoya Biosciences, Inc. recently announced a collaboration with AstraZeneca to advance new multiplex immunofluorescence (mIF) workflows and spatial biomarker signatures….
Harpoon Therapeutics Presents Updated Interim Clinical Data for the PSMA-Targeting TriTAC HPN424
Harpoon Therapeutics, Inc. recently presented interim data from the ongoing dose-escalation portion of a Phase 1/2a trial for HPN424 in patients with metastatic castration-resistant prostate…
iTeos Therapeutics Announces New Phase 1/2a Data Indicating Antitumor Activity of its Adenosine A2A Receptor Antagonist
iTeos Therapeutics, Inc. recently announced new clinical data from its ongoing Phase 1/2a clinical trial of inupadenant (EOS-850), a next-generation adenosine receptor (A2AR) antagonist, at…
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).