Bio Platforms
Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation
Orchard Therapeutics recently announced the presentation of new clinical data at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting to be held virtually…
Cerevel Therapeutics Announces First Patients Dosed in all Phase 3 Trials for the Treatment of Parkinson’s Disease
Cerevel Therapeutics recently announced that the first participants have been dosed in all three of the clinical trials in their Phase 3 program evaluating tavapadon…
Novartis Acquires Vedere Bio, a Novel Optogenetics AAV Gene Therapy Company
Vedere Bio, Inc. recently announced it has been acquired by Novartis. Shareholders in Vedere Bio received $150 million upfront and….
Apellis & Sobi Enter Collaboration for Global Co-Development & Ex-US Commercialization of Systemic Pegcetacoplan
Apellis Pharmaceuticals, Inc. and Swedish Orphan Biovitrum AB recently announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan, a targeted….
Catalent to Acquire Cell Therapy Manufacturing Facility From Bone Therapeutics
Catalent recently announced it had signed an agreement with Bone Therapeutics to acquire its cell therapy manufacturing subsidiary, Skeletal Cell Therapy Support SA (SCTS), including…
XOMA Earns $25-Million Milestone Payment as Anti-TGFβ Antibody Enters Phase 2 Clinical Study
XOMA Corporation recently announced NIS793, an anti-TGFβ monoclonal antibody licensed from XOMA, has advanced to the Phase 2 development stage, triggering a $25-million milestone payment…
Novavax Provides Phase 3 COVID-19 Vaccine Clinical Development Update
Novavax, Inc. recently announced updates on its Phase 3 clinical development program of NVX-CoV2373, its COVID-19 vaccine candidate. NVX‑CoV2373 is a stable, prefusion protein made…
miRagen Therapeutics Announces Acquisition of Viridian Therapeutics
miRagen Therapeutics, Inc. recently announced it has completed the acquisition of Viridian Therapeutics, Inc., a privately held biotechnology company focused on advancing new treatments for…
Bayer Acquires Company to Broaden Innovation Base in Cell & Gene Therapy
Bayer AG recently announced the acquisition of Asklepios BioPharmaceutical, Inc., a US-headquartered biopharmaceutical company specialized in the research, development, and manufacturing of gene therapies across different therapeutic areas…..
Quantum Genomics Enters Exclusive Licensing & Collaboration Agreement With Qilu Pharmaceutical
Quantum Genomics recently announced it has entered into an exclusive licensing and collaboration agreement with Qilu Pharmaceutical to develop and commercialize….
NGM Expands Oncology Portfolio With First Immuno-Oncology Development Candidate
NGM Biopharmaceuticals, Inc. recently announced the expansion of its oncology portfolio with its first immuno-oncology development candidate….
BrainStorm Cell Therapeutics & Catalent Announce Manufacturing Partnership
Catalent and BrainStorm Cell Therapeutics Inc. recently announced an agreement for the manufacture of NurOwn, BrainStorm’s autologous cellular therapy being investigated for the treatment of….
VBL Therapeutics Announces Additional New European Patent
VBL Therapeutics recently announced the European Patent Office (EPO) has granted Patent No. 3328401, which covers VBL’s proprietary investigational anti-MOSPD2 monoclonal antibodies to treat oncology…
PepTC Vaccines Publishes Positive Preclinical Results for COVID-19 Peptide Vaccine
PepTC Vaccines Limited, a subsidiary of Treos Bio Limited, recently announced the publication of positive preclinical results of its investigational PolyPEPI-SCoV-2 peptide vaccine. The study…
Castle Creek Biosciences Treats First Patient in Phase 1/2 Gene Therapy Trial
Castle Creek Biosciences, Inc. recently announced that the first adult patient has been dosed in a Phase 1/2 clinical trial evaluating FCX-013, the company’s investigational…
Aptose Initiates Dosing of CG-806 in Patients With Acute Myeloid Leukemia
Aptose Biosciences Inc. recently announced dosing of the first patient with acute myeloid leukemia (AML) in a Phase 1 a/b clinical study with CG-806, the…
MilliporeSigma’s New Platform Speeds Development of Cell & Gene Therapies
MilliporeSigma has recently bolstered its viral vector manufacturing capabilities with the launch of its VirusExpress Lentiviral Production Platform…..
Marinomed Biotech AG Develops First Medicinal Product for Autoimmune Gastritis
Marinomed Biotech AG, a Vienna-based biopharmaceutical company, develops groundbreaking treatment for autoimmune gastritis together with the Medical University of….
Cytovia Therapeutics Partners Enters Research & Licensing Agreement With Inserm
Cytovia Therapeutics recently announced it has entered a research and licensing agreement with Inserm to develop NK engager bi-specific antibodies and iPSC CAR NK cell therapy targeting….
Takeda & Arrowhead Collaborate to Co-Develop & Co-Commercialize ARO-AAT for Alpha-1 Antitrypsin-Associated Liver Disease
Takeda Pharmaceutical Company Limited and Arrowhead Pharmaceuticals Inc. recently announced a collaboration and licensing agreement to develop ARO-AAT, a Phase 2 investigational RNA interference (RNAi)…
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).