Bio Platforms
Bluejay Therapeutics Secures $182 Million in Series C Financing to Propel Clinical Pipeline
Bluejay Therapeutics recently announced the successful closure of a $182 million Series C financing round. This capital infusion will accelerate the clinical development of BJT-778,…
Attovia Therapeutics Raises $105 Million in Financing
Attovia Therapeutics recently announced the closing of a $105 million oversubscribed Series B financing, bringing the total capital raised by the company since its launch…
Akoya Biosciences & NeraCare Enter Exclusive Agreement to Enable Personalized Therapy Selection for Early Stage Melanoma Patients
Leveraging Akoya’s market-leading PhenoImager HT platform and clinical development expertise with NeraCare’s Immunoprint test and best-in-class clinical data, the partnership will…
Curia & Carterra Partner on Biologics Symposium to Further Biotechnology Research in the Pacific Northwest
Curia and Carterra Inc. recently announced their partnership in hosting a high-throughput biologics symposium slated for May 31 at the Residence Inn at Marriott….
Siren Biotechnology & Catalent Enter Partnership for Manufacturing of AAV Gene Therapies for Cancer
Siren Biotechnology and Catalent Inc. recently entered a strategic partnership to support the development and manufacturing of Siren Biotechnology’s AAV immuno-gene….
ENA Respiratory Receives USPTO Notice of Allowance for Key Patent Covering First-in-Class Antiviral Innate Immunomodulator
ENA Respiratory recently announced it has received a Notice of Allowance from the US Patent and Trademark Office (USPTO) for patent application US 18/112091 titled “Novel molecules” covering claims for….
ProQR Announces Preclinical Proof of Concept Data for AX-0810 Axiomer RNA Editing Program Targeting NTCP for Cholestatic Diseases
ProQR Therapeutics NV recently announced new preclinical data for its proprietary Axiomer RNA editing technology platform, including the first preclinical proof of concept data for…
Therini Bio Announces Positive Preclinical Data Supporting the Development of THN391 in Neurodegenerative Ocular Diseases
Therini Bio, Inc. recently announced positive preclinical data supporting the development of its lead candidate, THN391, for the treatment of neurodegenerative ocular diseases. The data…
FDA Grants Orphan Drug Designation to Biompharma’s Microbiome-Modifying Biologic
BIOM Pharmaceutical Corporation recently announced Bi104, its innovative drug product, has been granted orphan-drug designation by the US FDA for the treatment of Angelman syndrome…
Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran for Treatment of Myotonic Dystrophy Type 1
Avidity Biosciences, Inc. recently announced the US FDA has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for…
Eledon Announces Clinical Progress With Tegoprubart in the Prevention of Transplant Rejection
Eledon Pharmaceuticals, Inc. recently announced the first participant in an investigator-led clinical trial has received an islet cell transplant and is being treated with a…
Oregon Therapeutics & Lantern Pharma Launch Strategic AI Collaboration to Optimize Development of First-In-Class Drug Candidate XCE853 – A Potent Inhibitor of Cancer Metabolism
Lantern Pharma Inc. recently announced a strategic AI-driven collaboration with French biotechnology company, Oregon Therapeutics, to optimize the development of its first-in-class protein disulfide isomerase (PDI) inhibitor drug…
EyeDNA Therapeutics Announces Positive 24-month Data From Ongoing Phase 1/2 Trial of HORA-PDE6b Gene Therapy in Patients With Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b
eyeDNA Therapeutics recently announced positive 24-month follow-up results from its Phase 1/2 study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b, its investigational gene therapy…
Creyon Bio to Present Clinical Data on Rapid AI-Enabled Engineering of Oligonucleotide-Based Medicines
Creyon Bio, Inc. recently announced encouraging clinical data highlighting the use of its custom designed data set paired with artificial intelligence (AI) to rapidly engineer…
Inmagene Announces Completion of Enrollment in Phase 2a Trial of a Nondepleting & Half-Life Extended Anti-OX40 Monoclonal Antibody
Inmagene Biopharmaceuticals recently announced it completed target enrollment in its Phase 2a trial evaluating IMG-007 in adult patients with AA. IMG-007 is a nondepleting anti-OX40…
Medigene AG Expands Patent Portfolio With Patent Grant for its Targeted T Cell Receptor
Medigene AG recently announced it has been issued a patent by the European Patent Office protecting its T cell receptor (TCR) targeting NY-ESO-1….
Guangzhou Bio-gene Technologies Announces Initiation of Phase 1 Clinical Trial for CAR-T Targeting CLL-1
Guangzhou Bio-gene Technologies recently announced the first patient was treated with BG1805, an investigational autologous CAR-T cell therapy targeting CLL-1, a member of the C-type…
Allarity Therapeutics’ Stenoparib Shows Clear Clinical Benefit & Achieves Significant Milestone With Early Conclusion of Phase 2 Trial in Advanced Ovarian Cancer
Allarity Therapeutics, Inc. recently announced the early discontinuation of its Phase 2 clinical trial of stenoparib, a novel PARP inhibitor, for the treatment of advanced…
Pluri Launches Novel Immunotherapy Platform for Solid Tumor Treatment
Pluri Inc. recently launched its placental allogeneic MAIT cell platform for immunotherapy treatment for solid tumors – a significant medical need which currently lacks effective…
ASLAN Pharmaceuticals Announces Expansion of its Collaboration With Zenyaku to Investigate the Biology Underlying Differential Effects of Eblasakimab Compared to Other Biologics
ASLAN Pharmaceuticals recently announced it has signed a new research collaboration agreement with its partner, Zenyaku Kogyo Co., Ltd., to establish a framework for a…
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).