Bio Platforms
ProQR Therapeutics Announces $8.1 Million in New Funding From Rett Syndrome Research Trust to Expand RNA Editing Collaboration
ProQR Therapeutics NV recently announced an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT). Building on the initial $1 million research grant…
Prelude Therapeutics Presents Preliminary Results of Phase 1 Dose-Escalation Study of PRT2527 as Monotherapy and in Combination With Zanubrutinib in Patients With Relapsed/Refractory Lymphoid Malignancies
Prelude Therapeutics Incorporated recently announced the presentation of the first interim clinical data from its ongoing open-label, dose-escalation trial of PRT2527, a potent and highly…
BriaCell Presents Unprecedented Overall Survival Data in Metastatic Breast Cancer
BriaCell Therapeutics Corp. will showcased its impressive survival and clinical benefit data in MBC patients, including those with CNS metastases, treated with the Bria-IMT plus…
Enlivex Receives Regulatory Authorization for the Initiation of a Phase 1 Trial Evaluating Allocetra in Patients With TMJ Osteoarthritis
Enlivex Therapeutics Ltd. recently announced the Israeli Ministry of Health authorized the initiation of an investigator-initiated Phase 1 trial to evaluate the safety, tolerability, and…
2025 COMPANY PROFILES & CAPABILITIES
For each participating company, this section presents a detailed summary highlighting their core technologies, capabilities, technologies, and services.
EXECUTIVE INTERVIEW - Lonza: Utilizing Analytical Tools & Predictive Models to De-Risk Drug Development
Drug Development & Delivery recently interviewed Josh Marsh, Bioavailability Enhancement and PBPK Lead Scientist at Lonza, to discuss the benefits of predictive tools and Lonza’s approach.
Arvinas & Pfizer Announce Initial Phase 1b Data From the TACTIVE-U Sub-Study of Vepdegestrant in Combination With Abemaciclib
Arvinas, Inc. and Pfizer Inc. recently announced preliminary data from the ongoing Phase 1b portion of the TACTIVE-U sub-study of vepdegestrant in combination with abemaciclib…
Ascendia Pharmaceutical Solutions Launched to Meet Drug Development & Manufacturing Needs
Jim Huang, Ph.D., founder and CEO of Ascendia Pharmaceuticals, announces that the pharmaceutical contract development and manufacturing organization (CDMO) has re-branded to Ascendia Pharmaceutical Solutions.…
uniQure Announces Alignment With FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease
uniQure N.V. recently announced the company reached agreement with the US FDA on key elements of an Accelerated Approval pathway for AMT-130. “We are very…
Zealand Pharma Announces First Participant Enrolled in Phase 2b Trial of Petrelintide in People With Overweight or Obesity
Zealand Pharma A/S recently announced the first participant has been enrolled in ZUPREME-1, a global Phase 2b trial in people with obesity or overweight with…
Immutep Announces Initiation of Phase 3 Trial in First Line Non-Small Cell Lung Cancer
Immutep Limited recently announced the initiation of the pivotal TACTI-004 Phase 3 clinical trial for the treatment of first-line metastatic non-small cell lung cancer (1L…
IMUNON Announces Continued Strong Improvement in Overall Survival Data from Randomized Phase 2 OVATION 2 Study of IMNN-001
IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage company in late-stage development with its DNA-mediated immunotherapy, today announced additional clinical data from ongoing analyses of results from the…
NurExone Announces Promising Preclinical Results in Restoring Vision After Optic Nerve Damage
NurExone Biologic Inc., a biopharmaceutical company developing exosome-based regenerative therapies, has announced significant findings from an expanded preclinical study of the potential of its portfolio…
Bionical Emas Sells EMAS Pharma to Kester Capital Following Strategic Review
Bionical Emas is selling its Clinical Development division, EMAS Pharma, to Kester Capital and an incoming executive team. The decision to sell comes after a…
Medigene AG Announces KRAS G12V as First Target for TCR-Guided T Cell Engagers
Medigene AG recently announced the selection of Kirsten rat sarcoma viral oncogene homologue (KRAS) G12V, in the context of HLA*A11, as the initial target for the…
Sonnet BioTherapeutics Announces Topline Safety Data Following Successful Completion of SON-1010 Monotherapy Dose Escalation in Phase 1 SB101 Trial
Sonnet BioTherapeutics Holdings, Inc. recently announced that the results of SON-1010 at the highest dose have been formally evaluated by the Safety Review Committee in…
Mestag Therapeutics Announces Licensing of Novel Inflammatory Disease Target From its RAFT Platform
Mestag Therapeutics recently announced that Johnson & Johnson has exclusively licensed a novel, undisclosed target identified using Mestag’s Reversing Activated Fibroblast Technology (RAFT) platform under…
CERo Therapeutics Appoints Chris Ehrlich CEO
CERo Therapeutics Holdings, Inc. recently announced its Board of Directors has appointed Chris Ehrlich as CEO. Previously he held the position of Interim CEO. Mr.…
World’s First Zero-Off-Target Base-Edited NK Cell Therapy Receives IND Approval in Both China & US
Base Therapeutics recently announced its NK510 cell injection, the world’s first zero-off-target base-edited NK cell product, has received IND approvals from both the US FDA…
Groundbreaking Cretostimogene Grenadenorepvec Monotherapy Data Demonstrates Sustained, Durable Complete Responses in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
CG Oncology, Inc. recently announced topline data from the Phase 3 BOND-003 trial in patients with high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus…
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).