Bio Platforms
Elevar Therapeutics & Jiangsu Hengrui Pharma Announce Global Commercialization Licensing Agreement
Elevar Therapeutics, Inc. and Jiangsu Hengrui Pharmaceuticals Co., Ltd. recently announced a global licensing agreement that grants Elevar rights to commercialize and develop Hengrui Pharma’s anti-PD-1 antibody camrelizumab in combination with….
ABVC BioPharma Announces Completion of Subject Recruitment for Interim Analysis on ADHD Phase 2b Clinical Study
ABVC BioPharma, Inc. recently announced an update on the company's 8-week long ADHD Phase 2b clinical study taking place at the University of California San…
GRI Bio Announces Partnership With the Respiratory Translational Research Collaboration to Advance Leading NKT Regulation Technology Targeting Earlier in the Inflammatory Cascade to Modulate Disease Progression
GRI Bio, Inc. recently announced it has entered into a collaboration with the UK consortia, National Institute for Health and Care Research Respiratory Translational Research…
Terns Pharmaceuticals Announces Global Phase 1 Clinical Trial Design of TERN-701 for the Treatment of Chronic Myeloid Leukemia
Terns Pharmaceuticals, Inc. recently announced the US FDA’s clearance of its Investigational New Drug application and the design of the CARDINAL Trial, Terns’ global Phase…
Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy Designation for Severe Sickle Cell Disease Treatment
Editas Medicine, Inc. recently announced the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene-editing medicine for the treatment of…
Silo Pharma’s Peptide Shows Positive Results Against Rheumatoid Arthritis
Silo Pharma, Inc. recently announced positive data from a preclinical study investigating the binding affinity and optimization of SPU-21 liposomal joint homing peptide in human…
eXmoor Completes Expansion Into Full-Service Cell & Gene Therapy CDMO Partner With Opening of GMP Facilities
eXmoor pharma recently launched its new Cell and Gene Therapy Centre GMP facility, to support the simultaneous production of multiple therapies for clinical trials. The…
Olema Oncology Announces Expansion of Collaboration Agreement With Novartis
Olema Pharmaceuticals, Inc. recently announced an amendment to its existing clinical collaboration and supply agreement with Novartis Institutes for BioMedical Research, Inc. to increase the size of….
BioXcel Therapeutics Reports Positive Overall Survival Results From Single-Arm, Open-Label Phase 2 Trial of BXCL701 in Patients With Small Cell Neuroendocrine Prostate Cancer
BioXcel Therapeutics, Inc. recently announced positive overall survival (OS) data from its Phase 2 trial of BXCL701, the company's investigational oral innate immune activator, in…
AviadoBio Announces Initiation of Phase 1/2 Clinical Trial Evaluating AVB-101 in People With Frontotemporal Dementia With GRN Mutations
AviadoBio recently announced it has initiated the Phase 1/2 ASPIRE-FTD clinical study of its investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with…
MBX Biosciences Doses First Participant in Phase 1/2 Clinical Trial for the Treatment of Post-Bariatric Hypoglycemia
MBX Biosciences, Inc. recently announced the first healthy adult participant has been dosed in its Phase 1/2 clinical trial of MBX 1416, the company’s long-acting…
Salubris Biotherapeutics Presents Positive 6-Month Data From Phase 1b Clinical Trial of JK07
Salubris Biotherapeutics, Inc. recently announced positive updated results from the completed Phase 1b study of JK07 in patients with HFrEF. The data were presented in…
Tenaya Therapeutics Presents Encouraging New Clinical & Preclinical Data From HDAC6 Inhibitor Program for the Potential Treatment of Heart Failure With Preserved Ejection Fraction
Tenaya Therapeutics, Inc. recently released new data for TN-301 at the 2023 Heart Failure Society of America (HFSA) Annual Scientific Meeting. TN-301 is Tenaya’s highly…
BioXcel Therapeutics Provides Update on Recent Developments for Late-Stage Clinical Programs & Expansion of IP Portfolio for Sublingual Film
BioXcel Therapeutics, Inc. recently provided an update on recent developments with its late-stage clinical programs as well as its patent portfolio for IGALMI (dexmedetomidine) sublingual…
Oxford BioTherapeutics Announces Partner Boehringer Ingelheim Received US FDA Fast Track Designation for the Treatment of Extensive Stage Small Cell Lung Cancer & Extrapulmonary Neuroendocrine Cancers
Oxford BioTherapeutics (OBT) recently announced the US FDA has granted Fast Track designation to BI 764532 for the treatment of extensive stage small cell lung…
Ocean Biomedical Announces Newly Published Findings Demonstrating Ability to Restore Treatment Sensitivity to AstraZeneca’s Leading Lung Cancer Drug After Resistance Has Formed
Ocean Biomedical, Inc. recently announced its cancer-targeting immunotherapy antibody candidate has demonstrated effective tumor reduction against an aggressive subset of Non-Small Cell Lung Cancer (NSCLC)…
FORMULATION FORUM - CUBOSOMES – The Next Generation of Lipid Nanoparticles for Drug Delivery
Jim Huang, PhD, and Shaukat Ali, PhD, shed light on the design and formation of cubosomes with special focus on their applications for delivery of hydrophobic and hydrophilic small and large molecules, including oncology drugs and polynucleotides (DNA, mRNA, and siRNA).
PLATFORM TECHNOLOGY - The PTXΔLNP® Platform: On the Promise of Developing New LNPs for Tomorrow’s mRNA Therapies
Charlotte Dunne, PhD, Katrin Radloff, PhD, and Leonidas Gkionis, PhD, review the PTXΔLNP platform and how it offers a synergistic sister technology to the mRNA platform to obtain potent mRNA-LNPs for therapeutic applications.
BIOLOGICS DEVELOPMENT - Five Steps to a Robust Cell Line Development Process
Robert Gustines reviews the critical steps for designing and implementing a robust cell line development process to help overcome the obstacles that often hinder biologic production.
Assembly Biosciences Nominates Development Candidate ABI-6250, an Orally Bioavailable HDV Entry Inhibitor
Assembly Biosciences, Inc. recently announced the selection of development candidate ABI-6250 to progress to IND-enabling studies. ABI-6250, an orally bioavailable small molecule entry inhibitor, is…
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).