Bio Platforms
Catalent Adds New Cryogenic Capabilities at Shiga, Japan, Facility to Support Clinical Supply Demand for Cell & Gene Therapy Development
Catalent recently announced it has expanded the services and capabilities at its facility in Shiga, Japan, to include the storage, kitting, and distribution of advanced…
Transgene & BioInvent Report Positive Phase 1a Data on Oncolytic Virus BT-001 in Solid Tumors
Transgene and BioInvent International AB recently announce positive Phase 1a data on the oncolytic virus BT-001 for the treatment of solid tumors. Treatment with single agent BT-001 in 18 patients has now been….
Oxford BioTherapeutics & Boehringer Ingelheim Agree on 2-Year Extension to their Second Multi-Year Collaboration in Cancer Immunology
Oxford BioTherapeutics Ltd. recently announced it has extended its second multi-year collaboration with Boehringer Ingelheim for an additional 2 years. With this extension, further programs will….
Apellis Reports Top-Line Results from Phase 2 MERIDIAN Study in ALS
Apellis Pharmaceuticals, Inc. recently announced the Phase 2 MERIDIAN study investigating systemic pegcetacoplan for the treatment of amyotrophic lateral sclerosis (ALS) did not meet its…
Antabio Announces First Subjects Dosed in Phase 1 Clinical Trial of Highly Differentiated Antibiotic for Severe Hospital Infections
Antabio SAS recently announces dosing of the first subjects in its Phase 1 clinical trial of MEM-ANT3310, a next- generation antibacterial combination that has been…
BriaCell Enters Definitive Arrangement Agreement for the Spin Out of BriaPro Therapeutics Corp.
BriaCell Therapeutics Corp. recently announce it has entered into an arrangement agreement dated May 24, 2023, with BriaPro Therapeutics Corp. (SpinCo) pursuant to which certain…
Revvity Announces New License Agreement for Next-Generation Base Editing Technology
Revvity, Inc. recently announced a new license agreement with AstraZeneca for the technology underlying its Pin-point base editing system, a next-generation modular gene editing platform with a…
ImmunOs Therapeutics Announces Initiation of Dosing in Phase 1 Trial of Lead Compound IOS-1002
ImmunOs Therapeutics AG recently announced the initiation of dosing of patients in a Phase 1 trial of its lead program IOS-1002 in Australia. IOS-1002 is a…
Alvotech & Advanz Pharma Extend Strategic Partnership to Commercialize Five Proposed Biosimilars in Europe
Alvotech and Advanz Pharma recently announced the companies have entered into an exclusive partnership agreement regarding the supply and commercialization of five biosimilar candidates in…
Pyxis Oncology to Acquire Apexigen
Pyxis Oncology, Inc. and Apexigen, Inc. recently announced a definitive agreement by which Pyxis Oncology will acquire Apexigen in an all-stock transaction for an implied value of….
Eloxx Pharmaceuticals to Advance ELX-02 Into Pivotal Trial for the Treatment of Alport Syndrome With Nonsense Mutations Following Achievement of Remission in Patient in Phase 2 Study
Eloxx Pharmaceuticals, Inc. recently announced the company intends to advance ELX-02 into a pivotal trial for the treatment of Alport syndrome with nonsense mutations, following…
Valo Therapeutics Announces First Patient Dosed With PeptiCRAd-1 - Innovative Immuno-Oncology
Valo Therapeutics Oy (ValoTx) recently announced the first patient has been treated in its Phase 1 trial of PeptiCRAd-1 (Peptide-coated Conditionally Replicating Adenovirus) in three tumor….
AnaMar Announces Positive Phase 1 Data for its Phase 2 Ready Compound for Treating Fibrosis
AnaMar, a clinical-stage biotech company developing a first-in-class, peripheral 5-HT2B receptor antagonist, AM1476, as a pan anti-fibrotic medicine, recently announced positive results from its Phase…
Ocean Biomedical, Inc. Announces Patent Award for Novel Cancer Immunotherapy Treatment Granted for Prostate Cancer, Colon Cancer, Ovarian Cancer, Kidney Cancer, Breast Cancer, Glioblastoma, Melanoma & Lung Cancer
Ocean Biomedical recently announced its Scientific Co-founder, Dr. Jack A. Elias, MD, has been issued a broad patent for his cancer immunotherapy discovery related to…
Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease
Vega Therapeutics, Inc. recently announced the US FDA has granted the company orphan drug designation for VGA039 for the treatment of the rare bleeding disorder,…
AiViva Biopharma Initiates Phase 1 Clinical Trial of AIV007 for Age-Related Macular Degeneration & Diabetic Macular Edema
AiViva Biopharma Inc. recently announced it has begun a Phase 1 trial and completed dosing the first cohort of patients diagnosed with wet age-related macular…
NeuBase Announces Positive Preclinical Data Supporting the Capabilities of Stealth Editors
NeuBase Therapeutics, Inc. recently announced a broad set of preclinical safety and efficiency data for its Stealth Editors development program that demonstrate the ability to achieve…
Sangamo Receives FDA Fast Track Designation for Fabry Disease Gene Therapy Product Candidate
Sangamo Therapeutics, Inc. recently announced the US FDA has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate…
Cell & Gene Therapy CDMO Targets North American Growth With Major US Acquisition
uBriGene is expanding into the US market with the acquisition of a state-of-the-art GMP manufacturing facility from NASDAQ-listed company Mustang Bio, Inc. The cell & gene therapy (CGT) CDMO has an….
Biodexa Pharmaceuticals Enters Non-binding Letter of Intent for Proposed Acquisition of Varian Biopharmaceuticals
Biodexa Pharmaceuticals recently announced it has signed non-binding letter of intent to potentially acquire Varian Biopharmaceuticals, Inc., a private US precision oncology company developing….
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).