Bio Platforms
Cartesian Therapeutics Receives FDA Rare Pediatric Disease Designation for Descartes-08 for the Treatment of Juvenile Dermatomyositis
Cartesian Therapeutics, Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). Descartes-08, Cartesian’s…
Hongene Biotech Corporation & ReciBioPharm Establish a Gene Editing Development Partnership
Hongene Biotech Corporation and ReciBioPharm have announced a strategic collaboration that will enhance gene editing drug manufacturing capabilities. Hongene, renowned for its expertise in the…
GRIN Therapeutics Announces Positive Topline Data from Honeycomb Trial of Radiprodil in GRIN-Related Neurodevelopmental Disorder
GRIN Therapeutics, Inc. recently reported topline results from the its ongoing two-part global Phase 1b open-label trial evaluating the safety, tolerability, pharmacokinetics, and efficacy of…
Protara Therapeutics Announces Completion of First Cohort in Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients With Lymphatic Malformations
Protara Therapeutics, Inc. recently announced completion of the first cohort of the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment…
ENA Respiratory Announces Extension of US Department of Defense Funding for Novel Prophylactic Antiviral
ENA Respiratory has been awarded an additional $3.13 million contract from the U.S. Department of Defense (DOD). The new funding extends the $8.18 million already…
DRUG DELIVERY - Viewing Lipid Nanoparticle Delivery Technology Through the Lens of a CRDMO
Lu Tian, PhD, says the complexity of LNP formulations demands diverse and integrated capabilities for development – incorporating lipids, oligonucleotides, small molecules, and sometimes antibodies or ligands, and thus it requires a cohesive approach.
SILICON-STABILIZED HYBRID LNPS - Next-Generation Delivery of RNA Therapeutics
Suzanne Saffie-Siebert, PhD, Michael Welsh, PhD, Nissim Torabi-Pour, PhD, and Flavia M. Sutera, PhD, review current shortcomings, as well as some accompanying manufacturing challenges, and will show how they can be addressed by SiSaf’s silicon-stabilized hybrid LNPs.
EXECUTIVE INTERVIEW - Coya Therapeutics: Unlocking the Power of Tregs to Combat Inflammation & Fight Neurodegenerative Diseases
Dr. Howard Berman, Founder and CEO of Coya Therapeutics, discusses how the company is leveraging Tregs, its investigational products and the conditions targeted, Coya’s significant partnership with Dr. Reddy’s Laboratories, and his goals throughout the next 5 years.
Voyager Enters License for Next-Generation Capsid, Bringing Partnered Portfolio of TRACER-Enabled Gene Therapies to 14
Voyager Therapeutics, Inc. recently announced Novartis AG has agreed to license a novel capsid generated from Voyager’s TRACER capsid discovery platform for use in a…
Rigel Announces First Patient Enrolled in Phase 1b/2 Trial of REZLIDHIA (olutasidenib) in mIDH1 AML
Rigel Pharmaceuticals, Inc.recently announced the first patient has been enrolled in a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with REZLIDHIA…
Sonoma Biotherapeutics Receives $45-Million Milestone Payment From Regeneron
Sonoma Biotherapeutics, Inc. recently announced it has received a $45-million milestone payment from Regeneron Pharmaceuticals, Inc., under the terms of its active collaboration to discover,…
Invivyd Doses First Participants in Phase 1 Clinical Trial of Next Generation Monoclonal Antibody Candidate for COVID-19
Invivyd, Inc. recently announced dosing of the first participants in the Phase 1 healthy volunteer clinical trial of VYD2311, a next-generation monoclonal antibody (mAb) candidate…
Novartis Begins Construction of Two New Radioligand Therapy Facilities in the US
Novartis recently announced the construction of two new radioligand therapy (RLT) manufacturing facilities in the US that will extend its world-class manufacturing and supply chain…
Allyx Therapeutics Announces First Parkinson’s Disease Patient Treated
Allyx Therapeutics recently announced the first patient has been dosed with its lead compound, ALX-001, in a new clinical study assessing safety, pharmacokinetics and potential…
LIXTE Receives US Patent Issue Notification for Immune Oncology
LIXTE Biotechnology Holdings, Inc. recently announced it has received a Notice of Allowance from the United States Patent and Trademark Office for US Patent application…
Valneva & Pfizer Report Further Positive Phase 2 Booster Results for Lyme Disease Vaccine Candidate
Valneva SE and Pfizer Inc. recently announced positive immunogenicity and safety data from their VLA15-221 Phase 2 study following a second booster vaccination of their Lyme disease…
Caribou Biosciences Announces Fast Track Designations to CB-010 in Refractory SLE & to CB-012 in Relapsed or Refractory AML
Caribou Biosciences, Inc. recently announced the US FDA granted Fast Track designations to CB-010 for refractory systemic lupus erythematosus (SLE) and to CB-012 for relapsed…
Cartesian Therapeutics Announces First Patient Dosed in First-In-Human Phase 1 Trial of Next-Generation mRNA CAR-T Cell Therapy
Cartesian Therapeutics, Inc. recently announced the first patient has been dosed in its first-in-human Phase 1 trial of Descartes-15, the Company’s next-generation autologous anti-B cell…
Longeveron Announces Positive Type C Meeting With FDA Regarding Pathway to BLA
Longeveron Inc. recently announced the positive Type C meeting with the US FDA supporting the advancement of Lomecel-B, a proprietary, scalable, allogeneic, investigational cellular therapy…
Denali Therapeutics Announces Successful Meeting With FDA
Denali Therapeutics Inc. recently announced the outcome of a recent successful meeting with the Center for Drug Evaluation and Research (CDER) division of the US…
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).