Bio Platforms
CELL & GENE THERAPY - Cell & Gene Therapy’s Everest – The Challenges & Opportunities That Will Shape Success
Samir Acharya, PhD, Rajiv Vaidya, PhD, Laura Kerepesi, PhD, and Cyrill Kellerhals, MBA, provide their unique insights as they explore the challenges cell and gene therapy developers and manufacturers are currently facing, those they can expect to see in the future, and more critically, how to overcome them.
THERAPEUTIC FOCUS - Effect of NE3107 on the Pharmacokinetics Profile of Carbidopa/Levodopa in Patients With Parkinson’s Disease
Joseph M. Palumbo, MD, says addressing Parkinson’s via the inflammatory pathway offers a unique perspective that was virtually unheard of only 10 years ago.
Nykode Therapeutics Announces FDA Approval of IND for VB-C-04, a Trial of VB10.16 in HPV16-Positive Cervical Cancer
Nykode Therapeutics ASA recently announced FDA approval of its investigational new drug (IND) application for the Phase 2 clinical trial. The trial is designed to…
Zenas BioPharma Announces Strategic License & Collaboration Agreement With Bristol Myers Squibb
Collaboration furthers Zenas’ vision to bring innovative immunology-based medicines to patients around the world by leveraging Bristol Myers Squibb’s long-standing expertise in immune-mediated diseases….
SomaLogic Expands Agreement With Novo Nordisk to 2025
Novo Nordisk will extend its use of SomaLogic’s innovative proteomics technologies for research in cardiometabolic and other diseases….
Zealand Pharma Announces Designation of Priority Review by the US FDA for Dasiglucagon in Congenital Hyperinsulinism
Zealand Pharma A/S recently announced that the US FDA has granted priority review designation for dasiglucagon for the prevention and treatment of hypoglycemia in pediatric…
OKYO Pharma Achieves 90% Enrollment in 240-Patient Phase 2 Clinical Trial of OK-101 to treat Dry Eye Disease
OKYO Pharma Limited recently announced it has enrolled and randomized 90% of the patients in its 240-patient Phase 2 multi-center, double-masked, placebo-controlled clinical trial of…
Denali Therapeutics Announces New Interim Data From Phase 1/2 Study of DNL310 in MPS II (Hunter Syndrome)
Denali Therapeutics Inc. recently announced new interim data from the ongoing open-label, single-arm Phase 1/2 study of DNL310 (ETV:IDS) in children with MPS II (Hunter…
Biostax Signs Collaboration Agreement With Immgenuity to Pursue Remission in HIV
Immune Therapeutics, Inc. d/b/a Biostax Corp. recently announced the signing of a research collaboration agreement with Immgenuity, Inc. The joint research will focus on using…
Eagle Pharmaceuticals Announces Positive Type C Meeting With FDA for an Estrogen Receptor Antagonist Used in the Treatment of Metastatic Breast Cancer in Post-Menopausal Women
Eagle Pharmaceuticals, Inc. recently announced a positive Type C meeting with the US FDA. Eagle and the FDA agreed on a path forward to advance…
Ocean Biomedical Announces New Patent for Anti-Fibrosis Discovery With Allowance in Alcoholic Liver Disease & Multiple Fibrotic Conditions
Ocean Biomedical, Inc. recently announced the United States Patent and Trademark Office has issued a patent covering Ocean’s anti-Chitinase 1 small molecule candidate. Dr. Jack…
Cellares Announces Bristol Myers Squibb has Joined Technology Adoption Partnership Program to Evaluate Automated Manufacturing of CAR-T Cell Therapy on the Cell Shuttle Platform
Cellares, the first Integrated Development and Manufacturing Organization (IDMO) dedicated to clinical and industrial-scale cell therapy manufacturing, recently announced global biopharmaceutical company and cell therapy….
GeoVax Receives Notice of Allowance for Malaria Vaccine Patent
GeoVax Labs, Inc. recently announced the US Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/726,254 titled Compositions and…
Vaxxas Opens World-Class Manufacturing Facility to Produce Proprietary Needle-Free Vaccine Patch For Late-Stage Clinical Trials & First Commercial Products
Vaxxas recently announced the opening of its first, state-of-the-art manufacturing facility in Brisbane, Queensland. The custom-built 60,000-sq-ft Vaxxas Biomedical Facility will serve as….
NKGen Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of Allogeneic NK Cell Therapy Product Candidate for the Treatment of Solid Tumors
NKGen Biotech, Inc. recently announced the first patient has been dosed in a Phase 1, multi-center, open-label, dose-escalation study evaluating its cryopreserved investigational allogeneic blood-derived…
US FDA Approves Orphan Drug Designation for NXC-201 as a Treatment for Multiple Myeloma
Nexcella, Inc. recently announced the US FDA has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening form of blood…
Foundery Launches Inaugural Biotech Venture Creation Fund to Accelerate the Development of Novel Immunotherapies
Foundery’s co-development venture studio model aligns first-in-class drug development opportunities with financing and experimental execution to catalyze university discoveries….
BriaCell Awarded National Cancer Institute Grant to Advance its Bria-OTS Immunotherapy for Cancer
BriaCell Therapeutics Corp. recently announced the National Cancer Institute (NCI), the US federal government's principal agency for cancer research and training, has awarded the company…
Avidity Biosciences Receives FDA Orphan Drug Designation for for Treatment of Duchenne Muscular Dystrophy in People With Mutations Amenable to Exon 44 Skipping
Avidity Biosciences, Inc. recently announced the US FDA has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment…
Tiziana Life Sciences Announces FDA IND Clearance of Intranasal Foralumab for the Treatment of Alzheimer’s Disease
Tiziana Life Sciences Ltd. recently announced the US FDA has cleared the IND application for intranasal foralumab to be studied in Alzheimer’s disease. Foralumab could be a potentially….
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).