Bio Platforms
OBI Pharma Announces Phase 1/2 Study Initiation for a TROP2-Targeted ADC for Cancer Therapy
OBI Pharma recently announced the initiation of a Phase 1/2 clinical trial of OBI-992, an antibody-drug conjugate (ADC) targeting TROP2 (Trophoblast cell-surface antigen 2), an…
NodThera’s NLRP3 Inhibitor Meets Primary Endpoint of Inflammation Reversal in Phase 1b/2a Trial in Obese Subjects With Cardiovascular Risk
NodThera recently announced positive data from its Phase 1b/2a cardiovascular risk study in inflamed obese subjects, evaluating the effects of its oral, brain-penetrant NLRP3 inflammasome…
KSQ Therapeutics Announces First Patient Dosed in Clinical Development Program for a CRISPR/Cas9 Engineered Tumor Infiltrating Lymphocyte Therapy
KSQ Therapeutics, Inc. recently announced the first patient dosed in the Phase 1/2 clinical study of KSQ-001EX, a novel edited TIL therapy. KSQ-001EX consists of…
Wave Life Sciences Announces Presentations Highlighting Novel siRNA Program in Obesity & Leadership in RNA Editing
Wave Life Sciences Ltd. recently announced presentations at the 2nd Annual Obesity & Weight Loss Drug Development Summit and the 5th Annual RNA Editing Summit,…
MD Anderson & Sibylla Biotech Announce Strategic Collaboration to Discover & Develop Small Molecule Protein Degraders
The University of Texas MD Anderson Cancer Center and Sibylla Biotech recently announced a strategic collaboration agreement to discover and develop novel small molecule cancer…
SiSaf’s Bio-Courier Technology Enables Clinical Trial Success for Topical Treatment of Mild-to-Moderate Alopecia Areata
SiSaf Ltd recently announced that a licensee of the ProSilic variant of its technology has reported positive topline results from a randomized, placebo-controlled, multi-dose Phase 2…
Assembly Biosciences Doses First Participant in Phase 1a/b Clinical Trial of Herpes Simplex Virus Helicase-Primase Inhibitor Candidate
Assembly Biosciences, Inc. recently announced the first participant has been dosed in the Phase 1a/b trial of its long-acting herpes simplex virus (HSV) helicase-primase inhibitor…
MoonLake Immunotherapeutics Announces Positive Regulatory Feedback From Both FDA & EMA
MoonLake Immunotherapeutics recently announced the successful outcome of its end-of-Phase 2 interactions with the US FDA as well as positive feedback from its interactions with…
Atriva Therapeutics Secures US Patent for First-in-Class Broad Spectrum Therapy for Severe RNA Virus Infections Including Bird Flu
Atriva Therapeutics recently announced it has been granted a US patent for its MEK inhibitor zapnometinib (ATR-002). The patent includes claims for the treatment of…
CERo Therapeutics Successfully Completes IND-Enabling Toxicology Studies for Lead Compound
CERo Therapeutics Holdings, Inc. recently announced the successful completion of toxicology studies for its lead compound, CER-1236. The toxicology studies will be included in the…
ArriVent Announces Multi-Target ADC Collaboration With Alphamab
ArriVent BioPharma, Inc. recently announced it has entered into a collaboration agreement with Jiangsu Alphamab Biopharmaceuticals Co., Ltd. to discover, develop and commercialize novel antibody…
Ocean Biomedical Congratulates Joint Venture Partner, Virion Therapeutics, on Positive Immunogenicity Results
Ocean Biomedical, Inc. congratulated its JV partner Virion Therapeutics, LLC, a clinical-stage biotechnology company developing novel T cell-based immunotherapies, on its late breaker presentation highlighting the…
Assembly Biosciences Presents New Data Highlighting Hepatitis D Virus Entry Inhibitor
Assembly Biosciences, Inc. recently announced new data for ABI-6250, the company’s orally bioavailable, small molecule hepatitis D virus (HDV) entry inhibitor candidate, featured in a…
First Participants Vaccinated in IMUNON’s Phase 1 Clinical Trial
IMUNON, Inc. recently announced the first participants have been treated in the IMNN-101 Phase 1 clinical trial. This proof-of-concept study of IMUNON’s proprietary PlaCCine platform…
Wistar Scientists Develop Novel Antibody Treatment for Kidney Cancer
Advanced clear cell renal cell carcinoma (ccRCC) is a deadly form of kidney cancer with few treatment options; even with new immunotherapies, only around one…
ExeVir Bio Announces Exceptional Virus Neutralization Potency of its Variant-Proof Antibody Against the SARS-CoV-2 Variant JN.1
ExeVir Bio recently announced new data demonstrating its antibodies are exceptionally potent in neutralizing the SARS-CoV-2 variant JN.1, the parental strain of the currently most…
PLATFORM TECHNOLOGY - Antibody Oligonucleotide Conjugates (AOCs™) - Revolutionizing a New Class of Targeted RNA Therapeutics
Arthur A. Levin, PhD, highlights the first-ever successful targeted delivery of RNA to muscle in humans, a revolutionary advancement for the field of RNA therapeutics that may help transform the opportunities to advance research targeting many previously untreatable diseases in the years ahead.
SPECIAL FEATURE - Outsourcing Formulation Development & Manufacturing: Going Beyond the Science to Become True Partners
Contributor Cindy H. Dubin speaks with leading CDMOs about how they are adapting to bio/pharma client needs, their capabilities in handling complex molecules, and how they are transforming from specialist contractors to true partners.
FORMULATION FORUM - Advances in Drug Delivery by Antibody Drug Conjugates (ADCs)
Shaukat Ali, PhD, and Jim Huang, PhD, focus on two aspects of drug delivery through ADCs. One, where an antibody is conjugated via a ligand with functionalized LNPs carrying cytotoxic drugs; and two, where an antibody is conjugated directly with drug through a linker at the specific site.
AMORPHOUS SOLID DISPERSION SPECIATION - Impact of Polymer Chemistry & Drug Properties
Wesley K. Tatum, PhD, focuses on the use of in vitro techniques for characterizing ASD performance and on how these techniques can be used to help better understand the role of polymer chemistry in ASD performance.
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).