Bio Platforms
Mogrify & Astellas Announce Collaboration to Conduct Research on In Vivo Regenerative Medicine Approaches to Address Sensorineural Hearing Loss
Mogrify Limited and Astellas Pharma Inc. recently announced they have executed a collaborative research agreement on in vivo regenerative medicine approaches to….
Codiak BioSciences & CEPI Partner to Develop Broadly Protective Betacoronavirus Vaccine
CEPI, the Coalition for Epidemic Preparedness Innovations, and Codiak BioSciences recently announced the latest funding award under CEPI’s US $200 million program to advance the development of vaccines that provide broad protection against….
Fulcrum Therapeutics Enrolls First Patient in Pivotal Global Phase 3 Clinical Trial
Fulcrum Therapeutics, Inc. recently announced the first patient has been dosed in REACH, a Phase 3 clinical trial designed to evaluate the safety and efficacy…
Evonik Launches Non-Animal-Derived Collagen Platform at Commercial Scale for Medical Device Applications
Evonik’s recombinant, non-animal-derived collagen platform is now available at commercial scale. Vecollan is a highly pure and soluble collagen that is produced via fermentation. It…
BriaCell Partners With Waisman Biomanufacturing to Manufacture & Supply Prostate Cancer Immunotherapy
BriaCell Therapeutics Corp. has recently entered a manufacturing service agreement with Waisman Biomanufacturing at the University of Wisconsin–Madison (Waisman), to manufacture Bria-Pros, BriaCell’s off-the-shelf personalized….
Chinook Therapeutics Receives Orphan Drug Designation for BION-1301 for Treatment of Primary IgA Nephropathy
Chinook Therapeutics, Inc. recently announced the European Commission has granted orphan drug designation for BION-1301 for the treatment of primary IgA nephropathy (IgAN). The decision…
Neurona Therapeutics Announces Initial Subject Dosed in First Clinical Trial of Regenerative Human Cell Therapy in Adults with Drug-Resistant Focal Epilepsy
Neurona Therapeutics recently announced the first patient dosed in a Phase 1/2 clinical trial of its lead program, NRTX-1001, in a first-in-human epilepsy study…..
AN2 Therapeutics Initiates Pivotal Phase 2/3 Trial Evaluating Epetraborole for Treatment-Refractory MAC Lung Disease
AN2 Therapeutics, Inc. recently announced it has screened its first patient in its pivotal Phase 2/3 clinical trial evaluating once-daily, oral epetraborole for treatment-refractory Mycobacterium avium complex…..
Ventyx Biosciences Announces Positive Topline Phase 1 Data for its Peripheral NLRP3 Inhibitor
Ventyx Biosciences, Inc. recently announced positive data from the company’s Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) trial of VTX2735, a…
BriaCell Enters Research Agreement to Identify Novel Targets for Cancer Treatment
BriaCell Therapeutics Corp. recently announced a research collaboration agreement with Harvard Medical School in support of a project led by Joan S. Brugge, PhD, a faculty…
Roivant and Pfizer Unveil Priovant Therapeutics & Ongoing Registrational Studies for Oral Brepocitinib in Dermatomyositis and Lupus
Roivant Sciences and Pfizer recently announced the unveiling of Priovant Therapeutics, dedicated to developing and commercializing novel therapies for autoimmune diseases with the greatest morbidity…
Terumo Blood and Cell Technologies & GenCure Collaborate to Advance Cell & Gene Therapy Manufacturing Solutions
Terumo Blood and Cell Technologies recently announced it has signed a new collaborative agreement with GenCure, a subsidiary of BioBridge Global, to extend and unify cell and gene therapy manufacturing solutions…..
Fate Therapeutics Announces Expansion of Solid Tumor Collaboration With ONO Pharmaceutical
Fate Therapeutics, Inc. recently announced it has expanded its off-the-shelf, iPSC-derived, cell-based cancer immunotherapy collaboration with ONO Pharmaceutical Co., Ltd. to include the development of….
Akoya Biosciences to Partner With Acrivon Therapeutics for the Clinical Development of Acrivon’s Proprietary OncoSignature Test Into a Companion Diagnostic
Akoya Biosciences, Inc. and Acrivon Therapeutics, Inc. recently announced an agreement to co-develop, validate, and commercialize Acrivon’s OncoSignature test, a first-of-its-kind companion diagnostic. The test will…
Soligenix Receives FDA IND Clearance for Phase 2 Clinical Trial of Synthetic Hypericin in the Treatment of Psoriasis
Soligenix, Inc. recently announced the US FDA has cleared the Investigational New Drug (IND) application for a Phase 2a clinical trial titled Phase 2 Study…
Evaxion Biotech Expands Its EVX-03 DNA Vaccine Program Into Non-Small Cell Lung Cancer
Evaxion Biotech A/S recently announced it has selected EVX-03 as the product candidate within its DNA technology platform to target a new indication with planned…
Cullinan Oncology & Taiho Pharmaceutical Complete Agreement for Strategic Collaboration to Jointly Develop & Commercialize CLN-081/TAS6417
Cullinan Oncology, Inc. recently announced the completion of its agreement with Taiho Pharmaceutical Co., Ltd. (Taiho) signed in May 2022. Per the terms of the agreement….
Apellis Expands R&D Collaboration With Affilogic to Develop Targeted Complement Therapies for Delivery into the Brain
Apellis Pharmaceuticals, Inc. and Affilogic recently announced the companies have expanded their research and development (R&D) collaboration, which was initially formed in 2018, to include the development of Nanofitins….
Precision BioSciences Announces In Vivo Gene Editing Collaboration With Novartis
Precision BioSciences, Inc. recently announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma….
Pasithea Therapeutics Acquires Alpha-5 Integrin, LLC
Pasithea Therapeutics Corp. recently announced its acquisition of Alpha-5 integrin, LLC, a privately-held preclinical-stage company developing a monoclonal antibody (mAbs) for the treatment of amyotrophic lateral sclerosis (ALS) and….
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).