Bio Platforms
Single-Cell Multiomics Enables High-Resolution Vector Copy Number Profiling in CAR-T Therapie
Mission Bio, the leader in single-cell multiomics, published research in Molecular Therapy Advances showing that its Tapestri® platform can profile vector copy number (VCN), surface…
Cellares & Cabaletta Bio Sign 10-Year Commercial Supply Agreement to Scale Rese-cel
Cellares, the first Integrated Development and Manufacturing Organization (IDMO), today announced it has entered into a 10-year commercial supply agreement with Cabaletta Bio Inc. (Nasdaq:…
Argo Biopharma Announces First Subject Dosed in Phase I Study of siRNA Therapeutic BW-50218
Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a clinical-stage biotechnology company committed to developing next-generation siRNA therapies, today announced the first subject has been dosed in…
Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing
Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced positive topline results…
SPECIAL FEATURE - Bioavailability & Solubility: Experimental Techniques Combined With Computational Decision-Making Rapidly ID the Most Viable Enhancement Pathway
Contributor Cindy H. Dubin highlights other ways formulators are relying on automation, such as Artificial Intelligence and Machine Learning, and how these tools are being combined with experimental confirmation to identify the most promising development strategies.
First Patients Dosed With Cabaletta Bio’s Rese-cel Manufactured on Cellares' Automated Cell Shuttle Platform
Cellares recently announced that Cabaletta Bio Inc.’s investigational CAR T cell therapy rese-cel (resecabtagene autoleucel) has been infused into the first two patients using drug…
POLYMACROCYCLIC PEPTIDES - Engineering Structure for Function in Next Generation Therapeutics
Karsten Eastman, PhD, and Vahe Bandarian, PhD, believe the key challenge, and opportunity, is to sculpt peptide architecture so that shape and chemistry are presented to the target in the right place, at the right time, and for long enough to matter clinically.
TECHNOLOGY TRANSFER - Streamlining Biologics Technology Transfer Through Integrated Operational Models
Lalit Saxena explains how technology transfer in biologics manufacturing is an inherently complex process that requires the simultaneous control of multiple scientific, technical, and operational variables.
RADIOLIGAND THERAPIES - Affibody Molecules: A Versatile Approach to Radiopharma
Fredrik Frejd reviews Affibody’s lead RLT candidate, ABY-271, currently being evaluated in a first-in-human clinical study in HER2 positive metastatic breast cancer.
Transgene Completes Patient Randomization in Phase 2 Part of Clinical Trial Evaluating TG4050 in the Adjuvant Treatment of Head & Neck Cancer
Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, today announced the completion of patient randomization…
PannTheraPi Announces Significant Clinical Development & Strategic Milestones
PannTheraPi recently announced significant progress in its clinical development alongside a key leadership appointment to strengthen its scientific and operational capabilities. The company has submitted…
Cyclerion Therapeutics & Korsana Biosciences Announce Merger Agreement
Cyclerion Therapeutics, Inc. and Korsana Biosciences, Inc. recently announced they have entered into a definitive merger agreement for an all-stock transaction. Upon completion of the…
ImmuneBridge Announces New Partnership Focus, Investment & CEO
ImmuneBridge recently announced it will make its platform available to partners – ranging from small biotech startups to established pharmaceutical companies. Therapeutic co-development partners will…
Nanexa Demonstrates Feasibility of Quarterly Semaglutide Dosing with PharmaShell
Nanexa AB recently announced highly promising new results from its long‑acting semaglutide program, developed using the company’s proprietary PharmaShell drug delivery platform. Building on the…
Green Light for First-in-Human Trial of KMCAR T-cell Therapy in Multiple Myeloma
HaemaLogiX Ltd and Peter MacCallum Cancer Centre (Peter Mac) are delighted to announce the achievement of a landmark regulatory milestone: receipt of Clinical Trial Approval…
Centauri Therapeutics Granted FDA QIDP Status for CTX-187 Treatment of Gram-Negative Bacterial Infections
Centauri Therapeutics Limited recently announced the its lead clinical candidate in the ABX-01 program, CTX-187, has received Qualified Infectious Disease Product (QIDP) designation from the…
NorthSea Therapeutics Announces Achievement of Key Regulatory Milestones for Orziloben
NorthSea Therapeutics B.V. recently announced the US FDA and the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) have granted Orphan Drug Designations…
Quotient Sciences Extends Commercial Manufacturing Partnership for Ultra-Rare Disease Treatment
Quotient Sciences recently announced an extended commercial supply partnership with Ipsen, a global biopharmaceutical company, to manufacture a treatment for Fibrodysplasia Ossificans Progressiva (FOP), an…
Neurizon Initiates Dosing of NUZ-001 in HEALEY ALS Platform Trial
Neurizon Therapeutics Limited recently announced the first participant has been dosed in Regimen I of the HEALEY ALS Platform Trial evaluating Neurizon’s lead candidate, NUZ-001,…
TARA-002 Demonstrates 68% Complete Response Rate at 6 Months in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Protara Therapeutics, Inc. recently announced updated interim results from its ongoing Phase 2 open-label ADVANCED-2 trial assessing intravesical TARA-002, the Company’s investigational cell-based therapy, in…
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).















