Bio Platforms
MARKET LANDSCAPE - The Future of Cancer Care Post-Pandemic: What Doctors, Patients, and the Healthcare Industry at Large Should Expect
Angelos Stergiou, PhD, explains how the COVID-19 pandemic saw failures in the healthcare delivery system not only in general medicine, but also specifically in oncology. At the same time, several opportunities came to light during such a difficult, frightening moment, bringing a number of large- and small-scale innovations forth.
GENE EDITING TECHNOLOGY - Harnessing a Cell’s Natural DNA Repair Process to Develop Medicines With Higher Levels of Precision & Durability
Mariana Nacht, PhD, reviews a new gene editing approach that harnesses a cell’s natural DNA repair process, known as homologous recombination, to insert a corrective copy of the gene (or transgene) at a precise spot in a patient’s genome.
Editas Medicine Announces Favorable Decision From US PTO in CRISPR Patent Interference
Editas Medicine, Inc. recently announced the USPTO issued another favorable decision to the Broad Institute, Inc. (Broad) involving specific patents for CRISPR/Cas9 editing in human…
Santhera Signs Gene Therapy Agreement With SEAL Therapeutics
Santhera recently entered into an agreement with SEAL Therapeutics, a spin-off company from the Biozentrum of the University of Basel, which will further develop a….
Genevant Sciences & Arbutus Biopharma File Patent Infringement Lawsuit Against Moderna
Genevant Sciences and Arbutus Biopharma Corporation recently filed a lawsuit in the US District Court for the District of Delaware against Moderna, Inc. and an…
TFF Pharmaceuticals Announces Inhaled Niclosamide Significantly Inhibits Viral Replication of the Omicron Variant of SARS-CoV-2
TFF Pharmaceuticals, Inc. recently announced that results from its recently completed in vitro neutralization and viral replication assays indicate that the company’s inhaled niclosamide product candidate completely….
Vaxart’s S-Only COVID-19 Vaccine Candidate Produces Strong-Cross Reactive Mucosal & Systemic Immune Responses in Non-Human Primates
A new study published on BioRxiv.org demonstrates that Vaxart’s S-only COVID-19 clinical vaccine candidate, now being studied by Vaxart in Phase 2 trials, generated antibodies…
HiberCell Initiates its Phase 1a/b Clinical Trial in Patients With Advanced Solid Tumors
HiberCell has recently initiated a Phase 1a/b clinical trial of HC-7366, a first-in-class selective, orally bioavailable modulator of General Control Nonderepressible 2 (GNC2) for the…
Cyclerion Therapeutics & Ariana Pharma Announce Artificial Intelligence-Driven Precision Medicine Collaboration
Cyclerion Therapeutics, Inc. and Ariana Pharma recently announced an artificial intelligence-driven, precision medicine collaboration. This collaboration is expected to identify biomarkers of response to refine…
Moderna Initiates Phase 3 Portion of Pivotal Trial for mRNA Respiratory Syncytial Virus Vaccine Candidate
Moderna, Inc. recently announced the Data and Safety Monitoring Board (DSMB) for the RSV program has endorsed the start of the Phase 3 portion of…
Poseida Therapeutics Appoints New President, Gene Therapy
Poseida Therapeutics, Inc. recently announced Brent Warner has joined the company as President, Gene Therapy, effective February 21, 2022. “Brent brings deep pharmaceutical expertise and…
Medigene & BioNTech Announce Global Collaboration to Advance T Cell Receptor Immunotherapies Against Cancer
Medigene AG and BioNTech SE recently announced they have entered a multi-target research collaboration to develop T cell receptor (TCR) based….
Adaptive Biotechnologies Announces New Data Demonstrating ImmunoSEQ Technology Can Identify T-Cell Receptors Associated With Crohn’s Disease
Adaptive Biotechnologies Corporation recently presented data on T-cell receptor (TCR) sequences associated with Crohn’s disease (CD) during an oral presentation today at the 17th Congress…
Recipharm Announces Acquisition of Vibalogics & Arranta Bio
Recipharm is continuing to build its service offerings in new biologic modalities through the acquisition of Vibalogics and Arranta Bio….
Gracell Doses First Patients in First-in-Human Clinical Trial of Dual-Targeting CAR-T Therapy
Gracell Biotechnologies Inc. recently announced it has dosed multiple patients in a clinical trial evaluating GC012F, the company's autologous CAR-T therapeutic candidate dual-targeting B cell…
Zai Lab Announces Breakthrough Therapy Designation
Zai Lab Limited recently announced the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for investigational….
Celsion Reports Data Safety Monitoring Board Unanimous Recommendation to Continue Dosing Patients in the Phase 2 Portion of the OVATION 2 Study With GEN-1 in Advanced Ovarian Cancer
Celsion Corporation recently announced that following a pre-planned interim safety review of 81 as treated patients randomized in the Phase 1/2 OVATION 2 Study with….
Hillstream BioPharma Receives Orphan Drug Designation for Uveal Melanoma Treatment
Hillstream BioPharma Inc. recently announced the FDA granted Orphan Drug Designation to HSB-1216 for Uveal Melanoma (UM). Hillstream continues to….
BioXcel Therapeutics Presents Positive Data From Phase 2 Trial of BXCL701 in Aggressive Forms of Prostate Cancer
BioXcel Therapeutics, Inc. recently announced results from its ongoing Phase 2 trial of BXCL701, the company's investigational, oral innate immunity activator, in metastatic castration-resistant prostate…
Zealand Pharma Completes Enrollment in Phase 3 Trial of Dasiglucagon in Children With Congenital Hyperinsulinism
Zealand Pharma A/S recently announced completion of patient enrollment in the second Phase 3-trial, 17103, of dasiglucagon for the treatment of Congenital hyperinsulinism (CHI) 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).