Bio Platforms
OrganaBio Launches On-Demand GMP-Compliant Hematopoietic Stem Cell Source for Advanced Therapy Manufacturing
OrganaBio recently announced the launch of HematoPAC-HSC-CB-GMP, on-demand current good manufacturing practice (cGMP)-compliant CD34+ hematopoietic stem cells (HSCs) ethically derived from fresh human cord blood.…
Ipsen & Sutro Biopharma Announce Exclusive Global Licensing Agreement for an ADC Targeting Solid Tumors
Ipsen and Sutro Biopharma recently announced an exclusive global licensing agreement for STRO-003, an antibody-drug conjugate in the final stages of pre-clinical development, which targets the….
Evaxion & Undisclosed Collaborator Announce Encouraging Results for Vaccine Antigens Against Staphylococcus Aureus Infection
Evaxion Biotech A/S recently announced the successful conclusion of a series of large non-rodent animal infection studies, testing antigens from Evaxion’s preclinical EVX-B1 vaccine candidate…
Corcept Completes Enrollment in Phase 3 Gradient Trial of Relacorilant in Patients With Adrenal Cushing’s Syndrome
Corcept Therapeutics Incorporated recently announced completion of enrollment in GRADIENT, a Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with Cushing’s…
THERAPEUTIC FOCUS - Perspectives on Sigma-2 Modulation as a Therapeutic Modality for Slowing Age-Related Degenerative Disease
Anthony Caggiano, MD, PhD, believes there is evidence the sigma-2 receptor acts as a regulator of cellular processes, such as autophagy and protein trafficking, which are impaired in age-related degenerative diseases.
VACCINE DEVELOPMENT - Therapeutic Vaccines Development: At the Edge of a New Revolution
Mario Davinelli, PhD, Narcisa Mesaros, MD, David Morland, and Judith Neville, PhD, say therapeutic vaccines are an exciting area of research that has the potential to revolutionize the way we treat diseases.
PERSONALIZED MEDICINE - Exploring the Potential of the Aryl Hydrocarbon Receptor for Personalized Medicine
Pedro Moura Alves, PhD, reviews the intricacies of AhR immune sensing and signaling, and its potential for personalized treatment strategies to optimize therapeutic outcomes and minimize adverse effects. He also touches on some of the limitations hindering the advancement of knowledge in this crucial area of research, and the direction this emerging field may take in the coming years.
THERAPEUTIC FOCUS - Sarilumab Approval for Polymyalgia Rheumatica Highlights Enduring Unmet Medical Needs
David A. Katz, PhD, and Robert Jacks, MBA, MSE, say a new, effective treatment option is now available, promotion of the drug will increase disease awareness, and the success encourages others in the industry to continue and expand their PMR research. However, the proportion of patients who can benefit from the drug is limited, and significant unmet needs remain for all persons suffering from PMR.
Intravacc & Primrose Bio Announce Partnership to Enhance Conjugate Vaccine Development
The companies will leverage their combined experience in the development of conjugate vaccines for their clients….
Mustang Bio Announces Vision for CAR T-Cell Therapy Platform Expansion Into Autoimmune Diseases
Mustang Bio, Inc. recently announced its expansion into autoimmune diseases with MB-106, a personalized CD20-targeted, 3rd-generation autologous CAR T-cell therapy. MB-106 is being developed in…
Vidac Pharma Receives Japanese Patent Office Notice of Allowance for Cancer Drug Candidate
Vidac Pharma Holdings Plc. recently announced it has received a Notice of Allowance from the Japanese Patent Office for the composition and methods of use…
Immuneering Announces First Patient Dosed in its Phase 1/2a Trial of IMM-6-415 to Treat Advanced Solid Tumors With RAF or RAS Mutations
Immuneering Corporation recently announced the first patient has been dosed in its Phase 1/2a trial of IMM-6-415 to treat advanced solid tumors with RAF or…
Vector Pharma & Oncopeptides FZCO Announce Collaboration to Provide Pepaxti to Patients in the Middle East & North Africa
Oncopeptides AB and Vector Pharma FZCO recently announced a collaboration to commercialize Oncopeptides’ flagship drug Pepaxti (melphalan flufenamide) in the Middle….
Vaccinex Announces $1.5-Million Registered Direct Offering & Concurrent Private Placement Priced At-the-Market Under Nasdaq Rules
Vaccinex, Inc. recently announced it has entered into definitive agreements with certain institutional investors for the purchase and sale of 193,000 shares of its common stock in a….
LIXTE's Lead Clinical Compound Can Force Cancer Cells to Give Up Their Cancer-Causing Properties
LIXTE Biotechnology Holdings, Inc. recently announced publication of preclinical data in the online journal, Cancer Discovery, showing its lead clinical compound, LB-100, can force cancer…
Intravacc Announces Positive Data of First in Human Intranasal OMV-Based Vaccine for SARS-CoV-2
Intravacc recently announced positive clinical data of a first in human (FIH) study of Avacc 10, an intranasal outer membrane vesicles (OMV) based booster vaccine…
Teva UK & Closed Loop Medicine Announce Strategic Partnership to Advance Development of Personalized Medicines
Teva UK Limited and Closed Loop Medicine Ltd have announced a strategic partnership to advance the development of personalized medicine. Under the agreement, Closed Loop Medicine and Teva UK will investigate….
Nanoscope Therapeutics Announces Positive Top-line Results From Randomized Controlled Trial of MCO-010 for Retinitis Pigmentosa
Nanoscope Therapeutics Inc. recently announced positive top-line results after the completion of the 2-year Phase 2b RESTORE randomized, controlled clinical trial of its lead program, MCO-010,…
Exothera Pledges to Fast-Track RNA Therapies With Expansion of World-First Continuous-Batch RNA Manufacturing Solution Into North America
Game-changing continuous-batch production of RNA service removes process development and scale-up steps through automation and significantly cuts clinical development time, while enhancing quality and yield….
Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy in Patients with Rare Inherited Retinal Disease LCA5
Opus Genetics recently announced the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an…
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).