Bio Platforms
Siren Biotechnology Launches to Pioneer Universal AAV Immuno-Gene Therapy for Cancer
Novel approach combines the promise of two transformative therapeutic technologies, AAV gene therapy and cytokine immunotherapy, into a single transformative modality….
Sania Therapeutics Launches to Advance Novel Medicines for Neural Circuit Dysfunction
Sania Therapeutics, focused on developing genetic medicines for neural circuit dysfunction, recently launched by unveiling its suite of proprietary patented platforms at….
Elevar Therapeutics Submits NDA to FDA for Combination of Rivoceranib & Camrelizumab as First-Line Treatment Option for Unresectable Hepatocellular Carcinoma
Elevar Therapeutics, Inc. recently announced it submitted an NDA to the US FDA for its investigational drug rivoceranib, an oral TKI, in combination with camrelizumab,…
Bayer Receives Fast Track Designation for Asundexian Atrial Fibrillation Program
Bayer recently announced the US FDA has granted Fast Track Designation for its investigational drug asundexian (BAY2433334) as a potential treatment to prevent stroke and…
NurExone Granted US Patent for Technology & Drug Composition of Exo-PTEN, Designed for Treating Acute Spinal Cord Injuries
NurExone Biologic Inc. recently announced it has received an issue notification from the United States Patent and Trademark Office (USPTO) for US Patent NO. 11,648,260, granted for the period of 20 years from filing date until….
APPLICATION NOTE: Process Optimization & Preclinical Production Using the ANP System
Particle Works is thrilled to announce the launch of a new Application Note that delves into the advancement of nanoparticles as carriers for targeted drug delivery. Developing nanoparticles for this purpose can be….
TILT Biotherapeutics Announces First Patient Dosed in Phase 1 Solid Tumors Trial Assessing Oncolytic Adenovirus TILT-123 in Combination With Checkpoint Inhibitor
TILT Biotherapeutics recently announced the first patient has been dosed in a Phase 1 trial of the company’s oncolytic adenovirus TILT-123. The multi-centre open-label, Phase…
First Wave BioPharma Completes Patient Screening For Phase 2 Span Adrulipase Clinical Trial in Cystic Fibrosis
First Wave BioPharma, Inc. recently announced it has completed patient screening in its ongoing Phase 2 SPAN clinical trial investigating an enhanced enteric microgranule delivery…
SERB Pharmaceuticals & SFJ Pharmaceuticals Announce US Partnership for Ticagrelor Reversal Agent
SERB Pharmaceuticals and SFJ Pharmaceuticals recently announced SERB has acquired exclusive US rights to the ticagrelor reversal agent bentracimab from SFJ. Bentracimab is a novel,…
Cellular Origins Partners With ScaleReady to Simplify, Standardize & Automate Cell Therapy Manufacturing
Cellular Origins recently announced a collaborative partnership with ScaleReady, a joint venture between Bio-Techne, Fresenius Kabi, and Wilson Wolf that brings together process development expertise, tools, and technologies for scalable CGT….
Olema Oncology Announces OP-1250 Continues to Demonstrate Attractive Combinability With CDK4/6 Inhibitor Palbociclib in Phase 1b/2 Study
Olema Pharmaceuticals, Inc. recently announced interim results from an ongoing Phase 1b/2 clinical study of OP-1250, the company’s complete estrogen receptor (ER) antagonist (CERAN) and…
Mekonos Announces Research Collaboration With bit.bio to Advance Cell Engineering
New collaboration combines Mekonos’ novel ex vivo delivery platform with bit.bio’s unique opti-ox™ precision cellular reprogramming technology to accelerate the development of human cells for research, drug discovery and….
Evonetix Delivers First Chip-Synthesized DNA to the University of Cambridge
Evonetix recently announced DNA synthesized using its unique semiconductor chip technology was delivered to the Department of Chemical Engineering and Biotechnology at the University of Cambridge. This milestone marks the start of the next stage of development of the….
Alvotech Enters Commercialization Agreement With Polifarma for Proposed Biosimilar to Eylea
Alvotech recently announced it has entered an exclusive agreement with Polifarma Ilac San. ve tic. A.S. for the commercialization in Turkey of AVT06, a proposed…
Syneos Health to be Acquired for Approximately $7.1 Billion
Syneos Health, Inc. recently announced it has entered into a definitive agreement to be acquired by a consortium of private investment firm affiliates composed of Elliott Investment Management, Patient Square Capital….
Minerva Neurosciences Announces Update on its NDA for the Treatment of Negative Symptoms in Schizophrenia
Minerva Neurosciences, Inc. recently announced that on May 8, 2023, it received confirmation from the US FDA that its New Drug Application (NDA) for roluperidone…
Salipro Biotech & DyNAbind Announce Milestone Achievement in Collaboration to Enable DEL for Membrane Proteins
The two biotech companies report that they have successfully screened and identified several small molecule compounds that bind to a challenging membrane protein drug target….
Adaptive Biotechnologies Announces FDA Acceptance of Genentech’s INDA for the First Neoantigen-Directed T-Cell Therapy Product in Oncology
Adaptive Biotechnologies Corporation recently announced the US FDA has accepted an investigational new drug (IND) application submitted by its collaborator, Genentech, a member of the…
Longeveron Announces New Long-Term Survival Data From ELPIS I Trial of Lomecel-B for Hypoplastic Left Heart Syndrome
Longeveron Inc. recently announced new long-term follow-up data from the its ELPIS I trial of Lomecel-B for patients with hypoplastic left heart syndrome (HLHS). The…
Eisai Enters Joint Development Agreement With Blissbio for Antibody Drug Conjugate With Option Rights for Strategic Collaboration
Eisai Co., Ltd. recently announced it has entered into a joint development agreement with Bliss Biopharmaceutical (Hangzhou) Co., Ltd., for BB-1701, an antibody-drug conjugate (ADC) with option rights for a….
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).