Bio Platforms
Wave Life Sciences to Develop Programs in Rare, Genetic Eye Diseases
Wave Life Sciences Ltd. recently announced plans to design and advance stereopure oligonucleotide therapeutics for the potential treatment of rare, inherited eye diseases. Wave’s research…
Ziopharm & Precigen Redefine Relationships, Announce New License Agreement
Ziopharm Oncology, Inc. and Precigen, Inc. recently announced a new definitive license agreement to replace all existing agreements between the companies that will provide Ziopharm with certain exclusive and non-exclusive rights to technology controlled by Precigen, Inc.
Dragonfly Therapeutics Announces New Multi-Target Collaboration With Merck
Dragonfly Therapeutics, Inc. recently announced a strategic collaboration with Merck, known as MSD outside the US and Canada, through a subsidiary, to discover, develop, and…
ANTIBODY DRUG CONJUGATES - Expansion of Approved Indications Backs 25% Increase in Global Market
Laurie L. Sullivan and Shalini Shahani Dewan, MS, believe technological advancements, the rising incidence of cancer, and an increasing demand for biologic therapies are all factors driving growth in the global ADC market.
EXECUTIVE INTERVIEW - BriaCell Therapeutics: Recognizing the Power of Targeted Immunotherapies
Dr. William “Bill” Williams, MD, President and CEO of BriaCell Therapeutics, discusses the value of targeted immunotherapies in the biopharmaceutical industry.
VisionGate Presents Breakthrough Research on Cell-CT Platform
VisionGate recently presented two peer-reviewed poster presentations at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer (WCLC)…
Cobra Biologics, GE Healthcare & Centre for Process Innovation Collaborate to Advance Gene Therapy
Cobra Biologics recently announced a collaboration with the Centre for Process Innovation (CPI), a UK-based technology innovation centre and GE Healthcare Life Sciences. The three-way…
Brammer Bio Announces Progress on $200-Million Program to Advance Gene Therapies
Brammer Bio recently announced that a 3-year, $200-million investment program is on track to establish over 30 suites for clinical and commercial viral vector supply for a broad pipeline of advanced gene therapies and gene-modified cell therapies.
Inovio Develops Novel H3N2 Influenza DNA Vaccine; Generates Cross-Reactive Responses & Provides Complete Protection Against Lethal Preclinical Challenges
Inovio Pharmaceuticals, Inc. recently announced its SynCon vaccine approach using a collection of DNA antigens generated broadly protective antibody responses against the most deadly strains…
ContraVir Pharmaceuticals Announces Completion of Phase 1 With CRV431
ContraVir Pharmaceuticals, Inc. recently announced that the primary endpoints of safety and tolerability were met in a single ascending dose (SAD) study of CRV431 conducted…
Neurimmune Achieves Milestone in Collaboration With Ono Pharmaceutical
Neurimmune recently announced the achievement of a key preclinical milestone in the ongoing collaboration with Ono Pharmaceutical Co., Ltd. In November 2017, the parties entered…
Molecular Templates Announces Agreement With Takeda for Joint Development of Protein-Based Oncology Therapy
Molecular Templates, Inc. recently announced an agreement with Takeda Pharmaceutical Company Limited for the joint development of CD38-targeted engineered toxin bodies (ETBs) for the treatment of patients with diseases such as multiple myeloma.
IMV Announces Phase 2 Basket Trial in Collaboration With Merck
IMV Inc. recently announced it has expanded its clinical program with a Phase 2 basket trial evaluating its lead candidate, DPX-Survivac, in combination with low-…
Pieris Pharmaceuticals Announces Dosing of First Patient in Phase I Combination Trial
Pieris Pharmaceuticals, Inc. recently announced it has dosed the first patient in the company’s Phase 1 combination clinical trial of PRS-343, its lead proprietary immuno-oncology…
Biothera Pharmaceuticals Announces Immuno-Oncology Clinical Trial Collaboration With AstraZeneca
Biothera Pharmaceuticals, Inc. recently announced a clinical collaboration with AstraZeneca to evaluate whether the combination of Biothera’s Imprime PGG and AstraZeneca’s durvalumab (IMFINZI) can decrease tumor volume in patients with primary untreated locally advanced head and neck cancer prior to surgical resection.
Reata Announces Receipt of $30-Million Milestone Payment
Reata Pharmaceuticals, Inc. recently announced it has received a $30-million milestone payment from its licensee, Kyowa Hakko Kirin Co., Ltd. following the initiation of AYAME, a Phase 3 clinical trial to assess the efficacy and safety of bardoxolone methyl (bardoxolone) for the treatment of diabetic kidney disease in Japan.
Adverum Biotechnologies Announces IND Active for ADVM-022, a Novel Gene Therapy
Adverum Biotechnologies, Inc. recently announced its Investigational New Drug (IND) application is active for the planned multi-center, open-label, Phase 1, dose-escalation study of ADVM-022, a…
Cynata’s Stem Cell Therapy Meets All Safety & Efficacy Endpoints in Trial
Australian stem cell and regenerative medicine company Cynata Therapeutics Limited recently announced that CYP-001, its lead Cymerus mesenchymal stem cell (MSC) product candidate, met all clinical endpoints in a Phase 1 trial for the treatment of steroid-resistant acute graft-versus-host disease (GvHD).
DRUG DEVELOPMENT - Cell & Gene Therapies Calling for Innovation in Drug Development
Lev Gerlovin and Pascale Diesel, PharmD, believe there is significant evidence to indicate that we are entering a golden age of gene and cell therapy development, and while industry works to advance these programs, the debate over new approaches in both structuring and financing clinical research is likely to continue.
DRUG DELIVERY - Targeted Delivery of Submicron Particle Cancer Chemotherapy: Helping Shift the Immunotherapy Paradigm
Marc A. Iacobucci says that with much attention and research investment into IOT, NanOlogy is shaping a new paradigm for cancer treatment by attempting to improve the safety and effectiveness of tried and true CT through local delivery.
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).