Bio Platforms
WHITE PAPER - Nanoparticles in Drug Delivery & Photothermal Therapies
Download this white paper to learn more about the applications of silica and metal-based nanoparticles in drug delivery and photothermal therapy…..
GeoVax Receives Notice of Allowance for Cancer Vaccine Patent in China
GeoVax Labs, Inc. recently announced the Chinese Patent Office has issued a Notice of Allowance for GeoVax’s patent application titled Compositions and Methods for Generating…
Synlogic Receives Positive Opinion on Orphan Designation From the EMA for SYNB1618 for the Treatment of Phenylketonuria
Synlogic, Inc. recently announced the European Medicines Agency (EMA) issued a positive opinion on the company’s application for orphan designation for SYNB1618 for the treatment…
Day One Announces First Patients Dosed in Phase 1b/2 Combination Study With Tovorafenib & Pimasertib in RAF-Altered Solid Tumors
Day One Biopharmaceuticals recently announced the first patients have been dosed in sub-study 2 of FIRELIGHT-1, a Phase 1b/2 clinical trial evaluating tovorafenib (DAY101) in…
N4 Pharma Highlights Potential of Nuvec as Gene Therapy Delivery Platform
A recent preclinical in-vivo study found that Nuvec – N4 Pharma’s novel silica-based nanoparticle – formulated with TNF-α pDNA oncotherapy, suppressed tumor growth and improved survival of….
Aileron Therapeutics Announces First Patients Treated in Phase 1b Trial of ALRN-6924 in Patients With p53-Mutated Neoadjuvant Breast Cancer
Aileron Therapeutics recently announced it has treated the first patients in its Phase 1b randomized, controlled clinical trial evaluating ALRN-6924 to protect against chemotherapy-induced neutropenia…
Biomea Fusion Announces IND Candidate Selection: BMF-500, a Potential Best-in-Class Oral Covalent Inhibitor of FLT3
Biomea Fusion, Inc. recently announced the nomination of its second product candidate, BMF-500, a highly selective and potent covalent investigational third-generation….
Caladrius Biosciences Announces IDMC Recommendation to Advance CLBS201 Trial for the Treatment of Diabetic Kidney Disease
Caladrius Biosciences, Inc. recently announced the Independent Data Monitoring Committee (IDMC) overseeing the Phase 1b, open-label, proof-of-concept study of CLBS201 for the treatment of diabetic kidney disease (DKD) has reviewed the initial safety….
4D pharma Presents Late-Breaking Abstract From Phase 1/2 Trial of MRx-4DP0004 for the Treatment of Asthma
4D pharma plc recently announced the presentation of a late-breaking poster on data from Part A of the Phase 1/2 trial of MRx-4DP0004 for the…
IN8bio Announces Data Emerging From its Recently Unveiled Gamma-Delta T cell “Off-the-Shelf” Induced Pluripotent Stem Cell Platform
IN8bio, Inc. recently announced data emerging from the company’s recently unveiled iPSC-based gamma-delta T cell platform. iPSCs represent a significant step toward next generation approaches…
Codiak Presents New Preclinical Data on Exosome-Based Strategy to Enhance AAV Gene Therapy
Codiak BioSciences, Inc. recently announced new preclinical data on the company’s engEx-AAV discovery program, a novel strategy that aims to leverage exosomes to improve adeno-associated virus (AAV) vector gene….
Nurix Therapeutics Reports Dosing of First Patient in Phase 1 Clinical Trial of a Selective BTK Degrader in Development for B-cell Leukemias & Lymphomas
Nurix Therapeutics, Inc. recently announced the first patient has been dosed in its Phase 1a/1b study to evaluate orally available small molecule NX-5948, a potent…
Catalent Launches UpTempo Virtuoso AAV Platform to Reduce Gene Therapy Development Time & Accelerate Path to Clinic
Catalent recently introduced its new, UpTempo Virtuoso platform process for the development and CGMP manufacturing of adeno-associated viral (AAV) vectors. The UpTempo Virtuoso platform standardizes…
Prota Therapeutics Achieves 51% Peanut Allergy Remission Rate, First Company to Validate Significant Quality of Life Improvements Compared With Standard Care
Prota Therapeutics Pty Ltd is the first company to validate significant Health-Related Quality of Life improvements (HRQL), when compared to placebo, in a Phase 2b…
Travere Therapeutics Announces FDA Acceptance & Priority Review of NDA for Sparsentan for the Treatment of IgA Nephropathy
Travere Therapeutics, Inc. recently announced the US FDA has accepted and granted Priority Review of its New Drug Application (NDA) under Subpart H for accelerated…
Akston Biosciences Doses First Participants in Phase 2 Clinical Trial for Shelf-Stable COVID-19 Booster
Akston Biosciences Corporation recently announced it dosed the first set of volunteers in an open-label study of AKS-452, its protein subunit COVID-19 vaccine, as a…
Pneumagen Announces Positive Top Line Results From Phase 1 Study Evaluating a Product Designed for the Prevention & Treatment of Viral Respiratory Tract Infections
Pneumagen recently reported positive topline data from a Phase 1 study with Neumifil designed to evaluate safety and tolerability in healthy volunteers. The Phase 1, single…
NGM Bio Announces Initiation of Phase 1/1b Clinical Study for the Treatment of Patients With Advanced Solid Tumors
NGM Biopharmaceuticals, Inc. recently announced it has initiated a Phase 1/1b clinical study of NGM438 for the treatment of patients with advanced solid tumors. With…
Update on Positive Pre-Clinical Results From AIM Biologicals Development Program for the Potential Treatment of Parkinson’s Disease
Aeterna Zentaris Inc. recently provided an update on the positive results from pre-clinical studies of Aeterna’s Autoimmunity Modifying Biologicals (AIM Biologicals), for the potential treatment of Parkinson’s Disease (PD)…..
Artax Biopharma Closes Financing to Advance Clinical-Stage, First-in-Class Oral Immunomodulator to Treat T Cell-Mediated Diseases
Artax Biopharma, Inc. recently announced the close of a $26-million financing round to develop AX-158, the company’s first oral small molecule immunomodulating agent…..
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).