Bio Platforms
Longeveron Announces Positive Type C Meeting With FDA Regarding Pathway to BLA
Longeveron Inc. recently announced the positive Type C meeting with the US FDA supporting the advancement of Lomecel-B, a proprietary, scalable, allogeneic, investigational cellular therapy…
Denali Therapeutics Announces Successful Meeting With FDA
Denali Therapeutics Inc. recently announced the outcome of a recent successful meeting with the Center for Drug Evaluation and Research (CDER) division of the US…
Nuvectis Pharma Announces Orphan Drug Designation for the Treatment of ARID1a-Deficient Ovarian, Fallopian Tube & Primary Peritoneal Cancers
Nuvectis Pharma, Inc. recently announced that NXP800 was granted Orphan Drug Designation by the US FDA for the treatment of AT-rich interactive domain-containing protein 1a…
Purdue Researchers Take Inspiration From Viruses to Improve Delivery of Nucleic Acid-Based Therapies to Cancer Cells
A researcher in Purdue University’s College of Science is developing a patent-pending platform technology that mimics the dual-layer structure of viruses to deliver nucleic acid (NA)-based…
ENA Respiratory Progresses Phase 1b Study of its Dry Powder Formulation of Intranasal Innate Immunomodulator
ENA Respiratory recently announced it has advanced its Phase 1b study of its dry powder formulation of INNA-051 to enable clinical progression of an improved…
Ocean Biomedical Announces Patent Issued for PfGARP Malaria Antibodies
Ocean Biomedical recently announced its Scientific Co-founder Dr. Jonathan Kurtis, MD, PhD, has been issued a key US patent for his groundbreaking malaria therapeutic antibody…
Medicus Pharma Announces First Patient Randomized in Phase 2 Trial of SKNJCT-003 for the Treatment of Nodular Basal Cell Carcinoma in Adults
Medicus Pharma Ltd. recently announced the enrollment of the first patient in its Phase 2 clinical trial SKNJCT-003 for the treatment of nodular basal cell…
Sermonix Pharmaceuticals Completes Enrollment of Phase 2 I-SPY 2 Arm Evaluating Lasofoxifene in Neoadjuvant Breast Cancer Setting
Sermonix Pharmaceuticals Inc. and Quantum Leap Healthcare Collaborative recently announced Sermonix completed enrollment of its Phase 2 clinical trial evaluating lasofoxifene, its lead investigational drug,…
First Patient Dosed with LIXTE’s LB-100 in New Clinical Trial to Treat Colorectal Cancer
LIXTE Biotechnology Holdings, Inc. recently announced the dosing of the first patient in a new clinical trial in collaboration with the Netherlands Cancer Institute (NKI)…
Galapagos Announces FDA Clearance of IND Application for Phase 1/2 ATALANTA-1 Study of CD19 CAR-T, GLPG5101, in Relapsed/Refractory Non-Hodgkin Lymphoma
Galapagos NV recently announced the US FDA has cleared its Investigational New Drug (IND) application for ATALANTA-1, a Phase 1/2 multicenter study evaluating the feasibility,…
Abata Therapeutics Receives FDA Fast Track Designation for Progressive Multiple Sclerosis Treatment
Abata Therapeutics recently announced the US FDA has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The…
Emergex Receives Patent Protection for First-in-Class Influenza A Vaccines With Potential to Provide Long-Term T Cell Immunity
Emergex Vaccines Holding Limited recently announced it has received patent protection from the United States Patent and Trademark Office (USPTO) for its novel class of…
ST Pharm & Quantoom Biosciences Announce First Supply Agreement of SmartCap Under Extended Collaboration to Advance RNA Manufacturing
ST Pharm and Quantoom Biosciences recently announced an extended collaboration to accelerate the development and manufacturing of RNA-based vaccines and therapeutics. This collaboration brings together…
Opus Genetics Receives Rare Pediatric Disease Designation for Ocular Gene Therapy
Opus Genetics recently announced the US FDA has granted Rare Pediatric Disease designation (RPD) for its ocular gene therapy OPGx-LCA5 to treat patients with the…
ImmuneSensor Therapeutics Receives Clearance to Initiate First-in-Human Phase 1 Clinical Trial of cGAS inhibitor Drug Candidate
ImmuneSensor Therapeutics recently announced it has received Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration…
Processa Pharmaceuticals Announces Positive Preclinical Data for NGC-Iri
Processa Pharmaceuticals, Inc. recently announced positive data from preclinical studies that support the potential for NGC-Iri to have improved efficacy and a better side effect…
Abata Therapeutics Announces Strategic Investment From Bristol Myers Squibb
Abata Therapeutics recently announced it has received an equity investment from Bristol Myers Squibb to support the development of its Treg cell therapy products. Their…
TransCode Therapeutics Announces Phase 1 Clinical Trial Initiation
TransCode Therapeutics, Inc. recently announced initiation of its Phase 1 multicenter, open-label, clinical study of its lead therapeutic candidate, TTX-MC138. Two clinical trial sites have…
Denali Therapeutics Announces Publication Demonstrating the Potential of the Oligonucleotide Transport Vehicle Platform to Achieve Broad Biodistribution of Antisense Oligonucleotides in the CNS & Muscle Following Intravenous Administration
Denali Therapeutics Inc. recently announced publication of nonclinical data in the August 14, 2024, issue of Science Translational Medicine (link) demonstrating the ability of the…
Nuntius Therapeutics Announces Collaboration With Taiho Pharmaceutical to Develop Next-Generation mRNA Therapies Using Nuntius’ Proprietary Cell-Specific Delivery Technology
Nuntius Therapeutics recently announced it has entered into a collaboration agreement with Taiho Pharmaceutical Co., Ltd., a leading company in Japan for developing innovative medicines…
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).