Bio Platforms
Kinnate Biopharma Inc. Presents Preclinical Data on its Lead FGFR Inhibitor Candidate
Kinnate Biopharma Inc. recently announced results from preclinical studies evaluating its lead Fibroblast Growth Factor Receptor (FGFR) inhibitor candidate, KIN-3248. These findings were presented during…
Evelo Biosciences Announces Issuance of US Patent for Pharmaceutical Compositions of Single Strains of Naturally Occurring Bacteria as Medicines
Evelo Biosciences, Inc. recently announced the US Patent and Trademark Office has issued a new composition of matter patent (No. 11,090,341) for medicines comprising pharmaceutical…
Decibel Therapeutics Receives Orphan Drug & Rare Pediatric Disease Designations for DB-OTO for the Treatment of Otoferlin-Related Congenital Hearing Loss
Decibel Therapeutics recently announced the US FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s lead gene therapy product…
PolyPid Announces Positive Preclinical Data of its Intra-Tumoral OncoPLEX in Brain Cancer
PolyPid Ltd. recently announced positive preclinical data in two key Glioblastoma Multiform (GBM) animal models of its OncoPLEX intra-tumoral cancer therapy program. OncoPLEX utilizes the…
MacroGenics Announces Final Overall Survival Results From SOPHIA Study of MARGENZA in Patients with HER2-Positive Metastatic Breast Cancer
MacroGenics, Inc. recently announced the final overall survival (OS) results of the SOPHIA Phase 3 study in adult patients with metastatic HER2-positive breast cancer…..
AIM ImmunoTech Submits Pre-IND Application to the FDA for Phase 2 Clinical Study of Ampligen as a Potential Infusion Therapy for Post-COVID-19 Cognitive Dysfunction
AIM ImmunoTech Inc. recently announced it has submitted a Pre-Investigational New Drug application (Pre-IND) to the US FDA for a Phase 2 clinical study of…
Celsion Reports T-cell & B-cell Response From In Vivo Studies With its PLACCINE DNA Vaccine Platform
Celsion Corporation recently announced results from preclinical in vivo studies showing production of antibodies and cytotoxic T-cell response specific to the spike antigen of SARS-CoV-2…
TCR² Therapeutics Receives FDA Orphan Drug Designation for Gavo-cel for the Treatment of Cholangiocarcinoma
TCR2 Therapeutics Inc. recently announced the US FDA has granted the company Orphan Drug Designation (ODD) to gavo-cel for the treatment of cholangiocarcinoma. New clinical…
Genezen Appoints New CEO as GMP Expansion Begins
Genezen Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has appointed Ray Kaczmarek as CEO. Mr. Kaczmarek has over 28 years…
Fusion Antibodies Partners With Eurofins Discovery to Support Innovative Preclinical Drug Discovery Research
Fusion Antibodies plc recently announced it has entered into a commercial collaboration with Eurofins, a Eurofins Scientific SE (EUFI.PA) group company; a leading provider of products and services to the….
Poseida Therapeutics Announces FDA Clearance of IND Application for Allogeneic CAR-T Candidate for Relapsed/Refractory Multiple Myeloma
Poseida Therapeutics, Inc. recently announced the US FDA has cleared its Investigational New Drug (IND) application for P-BCMA-ALLO1, the company's first fully allogeneic CAR-T product…
Quantum Genomics Presents Results From Phase 2b QUORUM Study of Firibastat in Heart Failure
Quantum Genomics recently announced results of the Phase 2b QUORUM study were presented by Prof. Gilles Montalescot (Paris) on August 27, 2021 during the 2021…
AC Immune Announces First Positive Cognitive Results for a Tau-Targeting Monoclonal Antibody in Alzheimer’s Disease
AC Immune SA recently announced Genentech, a member of the Roche Group, has informed the company that Lauriet, a placebo-controlled Phase 2 study evaluating the…
GENE THERAPY - Solving the Puzzle: Aligning the Pieces of Gene Therapy & Creating Success for Patients
Sue Washer, MBA, reviews the various elements that must come together in developing safe, effective, and commercially viable viral-based gene therapies.
DRUG DEVELOPMENT EXECUTIVE - Poseida Therapeutics: Creating the Next Wave of Cell & Gene Therapies With the Capacity to Cure
Eric M. Ostertag, MD, PhD, CEO of Poseida Therapeutics, discusses the company’s innovative approach to develop safer, more effective, accessible and affordable cell and gene therapies for patients.
Aptamer Group & Bio-Works Technologies Partner to Enable Improved Solutions for Gene Therapy Manufacturing
Aptamer Group Ltd and Bio-Works Technologies AB recently announced they have entered into an agreement to co-develop a new affinity resin for improved purification and scale up of….
Shape Therapeutics Enters Strategic Research Collaboration With Roche to Advance Breakthrough AAV-Based RNA Editing Technology for Neuroscience & Rare Disease Indications
Through this partnership Shape will apply its proprietary RNA editing platform RNAfix and potentially leverage its AAVid technology platform for next-generation tissue-specific adeno-associated viruses (AAVs) for the development of gene therapy for….
Vifor Pharma & Cara Therapeutics Announce US FDA Approval of Injection for Treating Moderate-to-Severe Pruritus in Hemodialysis Patients
Cara Therapeutics and Vifor Pharma recently announced the US FDA has approved KORSUVA (difelikefalin) for injection for the treatment of moderate-to-severe pruritus associated with….
SomaLogic & Twist Bioscience Corporation Announce Partnership to Discover Novel Therapeutic Targets & Antibodies
CM Life Sciences II and SomaLogic, Inc., and Twist Bioscience Corporation recently announced a collaboration aimed at creating novel antibodies and enabling innovative drug discovery based on….
Junshi & Coherus Biosciences Announce Positive Interim Results of CHOICE-01 Phase 3 Clinical Trial
Shanghai Junshi Biosciences Co., Ltd. and Coherus BioSciences, Inc. recently announced positive interim results from the pivotal study CHOICE-01 (NCT03856411), a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating….
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).