Bio Platforms
Tiziana Life Sciences Announces University of Massachusetts Commences Dosing Intranasal Foralumab in Phase 2 Multiple Sclerosis Trial
Tiziana Life Sciences, Ltd. recently announced dosing has commenced at the fourth clinical site in its ongoing Phase 2 trial evaluating intranasal foralumab in patients…
Bluejay Therapeutics Announces Upcoming Presentation of Preclinical Data in Metabolic Dysfunction-Associated Steatohepatitis
Bluejay Therapeutics recently announced it will present new preclinical data on the characterization of BJT-188, a liver-targeted fatty acid synthase (FASN) inhibitor being investigated for the…
Kymera Therapeutics Announces First Patient Dosed in BROADEN Phase 1b Atopic Dermatitis Clinical Trial of First-in-Class Oral STAT6 Degrader
Kymera Therapeutics, Inc. recently announced it recently initiated dosing in its BroADen Phase 1b clinical trial evaluating KT-621, an oral, highly selective, potent degrader of…
March Biosciences Announces First Patient Dosed in Phase 2 Clinical Trial of First-in-Class CD5 CAR-T Cell Therapy for T-Cell Lymphoma
March Biosciences recently announced the first patient has been dosed in its Phase 2 clinical trial evaluating MB-105, a first-in-class CD5-targeted CAR-T cell therapy for…
Vaccinex to Report Promising New Clinical Data Revealing Pepinemab’s Unique Mechanism to Enhance Immunotherapy
Vaccinex, Inc. recently announced it will present exciting new data characterizing the unique mechanism of pepinemab to enhance immune responses to checkpoint therapies, corresponding with…
Cullinan Therapeutics Receives Approval From EMA to Initiate Phase 1 Trial of a Bispecific CD19 T Cell Engager Administered Subcutaneously in Patients With RA
Cullinan Therapeutics, Inc. recently announced the European Medicines Agency (EMA) approved the company’s Clinical Trial Application (CTA) for CLN-978. The Phase 1 clinical trial will…
Q32 Bio Doses First Patients in Both Part A Open-Label Extension & Part B of SIGNAL-AA Phase 2a Trial Evaluating Bempikibart in Alopecia Areata
Q32 Bio Inc. recently announced it has dosed the first patients in both the Part A open-label extension (OLE) and Part B of the SIGNAL-AA…
Salarius Pharmaceuticals Merger Partner, Decoy Therapeutics, Appoints Renowned MIT Professor Robert S. Langer to its Scientific Advisory Board
Decoy Therapeutics, Inc. recently announced Massachusetts Institute of Technology (MIT) Professor and Moderna Co-founder Robert S. Langer, ScD will join the company’s Scientific Advisory Board…
Addgene Adds Science & Tech Leaders to its Management Team & Board of Directors
Addgene recently announced the expansion of its management team and Board of Directors with numerous industry leaders. These key appointments support Addgene’s growth strategy and illustrate…
Researchers Recruit Killers to a Tumor Fight: Antibody Immunotherapies Developed to Treat Glioblastoma
Purdue University researchers are developing innovative antibody-based immunotherapies that recruit and improve the function of the body’s innate immune system to treat glioblastoma, an incurable…
Solu Therapeutics Closes $41M Financing & Announces First Patient Dosed in Phase 1 Clinical Trial of STX-0712 in Patients With CMML & Other Advanced Hematologic Malignancies
Solu Therapeutics recently announced the successful completion of a $41 million Series A financing that included participation from five new investors – Eli Lilly and…
CERo Therapeutics Announces Initial Clinical Trial Site for its Phase 1 Clinical Trial of CER-1236 in Acute Myeloid Leukemia
CERo Therapeutics Holdings, Inc. recently announced its first clinical trial site for the its Phase 1 clinical trial of CER-1236. The trial is focused on…
Palisade Bio Completes Dosing in Phase 1a Portion of Ongoing Phase 1a/b Study of PALI-2108
Palisade Bio, Inc. recently announced the completion of enrollment and dosing in all five Phase 1a SAD cohorts, all four MAD cohorts, and the food…
Comanche Biopharma Announces Orphan Drug Designation for CBP-4888 in sFlt1-Mediated Preterm Preeclampsia
Comanche Biopharma Corp. recently announced the European Medicines Agency (EMA) has granted orphan drug designation for CBP-4888 for the treatment of sFlt1-mediated preterm preeclampsia. A…
Recursion Announces First Patient Dosed in Phase 1 Clinical Study of a Selective MALT1 Inhibitor for Relapsed or Refractory B-cell Lymphomas
Recursion recently announced that the first patient has been dosed in the Phase 1 EXCELERIZE clinical study evaluating REC-3565 for the treatment of relapsed or…
Opus Genetics Announces 1-Month Clinical Data From Pediatric Patient in Phase 1/2 Trial of Gene Therapy in Inherited Retinal Diseases
Opus Genetics, Inc. recently announced 1-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label…
ALX Oncology Receives IND Clearance From FDA for a Novel EGFR-Targeted ADC
ALX Oncology Holdings Inc. recently announced receipt of US FDA clearance for the Investigational New Drug (IND) application for ALX2004, the company’s potential best- and…
ADC PAYLOADS - Progress in Development of Camptothecin-Based ADC Therapeutics
Paul Moore, PhD, Raffaele Colombo, PhD, and Jamie Rich, PhD, say the recent progress in research on novel camptothecin payloads highlights the potential for developing a new generation of ADCs that can overcome some of the limitations of first-generation therapies.
CELL-FREE GENE SYNTHESIS - Eliminating Critical Bottlenecks in Genetic Medicine Development
Marc Unger explores the market trends, challenges, and opportunities new DNA production technologies provide to accelerate genetic advances in healthcare.
MoonLake Secures up to $500 Million in Non-Dilutive Financing From Hercules Capital
MoonLake Immunotherapeutics recently announced that it has entered into an agreement with Hercules Capital, Inc. (NYSE:HTGC), a leader in customized debt financing for companies in…
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).