Bio Platforms
Lipella Pharmaceuticals to Advance Lead Product Candidate to Phase 2b
Lipella Pharmaceuticals Inc. recently announced the receipt of US FDA Type-C meeting guidance regarding the LP-10 (liposomal tacrolimus) clinical program. Lipella expects to initiate a…
Limula Raises $6.8M to Democratise Access to Life-Saving Cell & Gene Therapies With Swiss-Made Manufacturing Platform
Over the past decade, several breakthrough Cell and Gene Therapy products have been approved in the US and Europe to turn stem cells and immune…
Molecure Receives Approval to Initiate Phase 2 Clinical Trial for the Treatment of Pulmonary Sarcoidosis
Molecure S.A. recently announced it has received national regulatory approvals from Denmark, France, Greece, Germany, and Norway to conduct a Phase 2 clinical trial for…
Sana Biotechnology Highlights Novel In Vivo Preclinical Data Showing Potential for Pluripotent Stem Cell-Derived Human Glial Progenitor Cell Transplantation as a Treatment for Huntington’s Disease and Other Glial-Based Neurodegenerative Conditions
Healthy transplanted human glial cells replaced diseased glial cells in the brains of human glial chimeric Huntingtin mice….
TFF Pharmaceuticals & Cleveland Clinic to Advance Multivalent Universal Influenza Vaccine Candidates into Preclinical Testing
TFF Pharmaceuticals, Inc recently announced that, in collaboration with Cleveland Clinic, the company is advancing multiple multivalent universal influenza vaccines to protect against seasonal and…
Lisata Therapeutics Receives Paediatric Investigation Plan Waiver From EMA for Certepetide in Pancreatic Cancer
Lisata Therapeutics, Inc. recently announced it has reached agreement with the European Medicines Agency (EMA) on a Paediatric Investigation Plan (PIP) for Lisata’s lead investigational…
Coya Therapeutics Announces $5-Million Strategic Investment by the Alzheimer’s Drug Discovery Foundation
Coya Therapeutics, Inc. recently announces the Alzheimer’s Drug Discovery Foundation (ADDF) has purchased 603,136 shares of the company’s common stock at a purchase price of…
GRI Bio Presents Positive Preclinical Data Demonstrating Lead Program Reduces the Important Inflammatory & Fibrotic Drivers in Idiopathic Pulmonary Fibrosis
GRI Bio, Inc. recently presented positive preclinical data demonstrating its lead program GRI-0621 reduces the important inflammatory and fibrotic drivers in Idiopathic Pulmonary Fibrosis (IPF).…
Voyager Therapeutics Announces First Participants Dosed in Single Ascending Dose Trial of VY-TAU01 for the Treatment of Alzheimer’s Disease
Voyager Therapeutics, Inc. recently announced that the first participants were dosed in a Phase 1a single ascending dose (SAD) trial of VY-TAU01, an investigational anti-tau…
MoonLake Immunotherapeutics Starts Phase 3 VELA Program of the Nanobody Sonelokimab in Patients With Moderate-to-Severe Hidradenitis Suppurativa
MoonLake Immunotherapeutics recently announced the first patients have been screened at a US trial site in its global Phase 3 clinical program, VELA, evaluating sonelokimab,…
Ocean Biomedical Announces Patent Allowance for Malaria Antibodies That Have Potential Capability to Protect Against Infection & Treat Severe Disease
Ocean Biomedical recently announced that Scientific Co-founder Dr. Jonathan Kurtis, MD, PhD, has been granted a new patent for his malaria therapeutic antibody discoveries, targeted…
Avenue Therapeutics Announces Last Patient Last Visit in Phase 1b/2a Clinical Trial of AJ201 for the Treatment of Spinal & Bulbar Muscular Atrophy
Avenue Therapeutics, Inc. recently announced the completion of the last patient’s final visit in the company’s Phase 1b/2a clinical trial of AJ201 for the treatment…
Enable Injections & Serina Therapeutics Announce Agreement to Develop SER-252 in Combination With enFuse for Advanced Parkinson’s Disease
Enable Injections, Inc. and Serina Therapeutics recently announced a partnership to develop and commercialize SER-252 (POZ-apomorphine) in combination with enFuse for the treatment of Parkinson’s…
Medigene Presents Preclinical Data on Optimal Affinity TCRs Targeting the Neoantigen Mutant KRAS
Medigene AG recently presented the company’s proprietary T cell receptor (TCR) discovery process to obtain optimal affinity 3S (sensitive, specific and safe) TCRs at the…
CellProthera Announces Positive Phase 1/2b Data for ProtheraCytes Cell Therapy in Acute Myocardial Infarction
CellProthera recently announced positive results from a Phase 1/2b study of its lead ProtheraCytes in patients following a severe heart attack, positioning the stem cell…
ElevateBio Announces New US Patents for Lead Life Edit CRISPR Systems & Adenine Deaminases Enabling Advanced Gene Editing Techniques
ElevateBio is building a robust portfolio of patents protecting its novel technologies while the Company scales as the world’s first integrated genetic medicine foundry….
PTC Therapeutics Announces FDA Acceptance & Priority Review of BLA
PTC Therapeutics, Inc. recently announced the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza (eladocagene exuparvovec), a gene therapy for the…
Tavros Therapeutics Achieves Two Milestone Payments in its Collaboration With Vividion Therapeutics
Tavros Therapeutics, Inc. recently announced it has achieved two milestone payments in its collaboration with Vividion Therapeutics, Inc., a wholly owned and independently operated subsidiary…
CENTOGENE & Evotec Discover Promising New Molecule to Treat Gaucher Disease
Centogene N.V. recently announced the discovery of a new small molecule together with Evotec SE. The new molecule has the potential to treat patients with…
AC Immune & Takeda Sign Exclusive Option & License Agreement for Active Immunotherapy Targeting Amyloid Beta for Alzheimer’s Disease
Takeda to receive exclusive option to license global rights to ACI-24.060, a potential first-in-class active immunotherapy designed to delay or slow Alzheimer’s disease progression….
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).