Bio Platforms
Lantheus Grants Rights for the Use of its Microbubbles to SonoThera for its Ultrasound-Guided Nonviral Gene Therapy Platform
Lantheus Holdings, Inc. recently announced a strategic collaboration with SonoThera, Inc. in which Lantheus’ microbubbles will be used in combination with SonoThera’s ultrasound-guided, nonviral, gene…
Caribou Biosciences Selects ROR1 as the Target for CB-020, an iPSC-Derived Allogeneic CAR-NK Cell Therapy
Caribou Biosciences, Inc. recently announced target selection for CB-020, an induced pluripotent stem cell (iPSC)-derived allogeneic anti-ROR 1 (receptor tyrosine kinase like orphan receptor 1)…
UM171 Cell Therapy Demonstrates Improved Outcomes Compared to Cord & Matched Unrelated Donor Peripheral Blood Transplants in Real-World Setting
ExCellThera Inc. recently announced findings from a safety and efficacy retrospective analysis comparing outcomes of patients treated with ECT-001 (UM171-expanded) Cell Therapy in a Phase 1/2 trial to those treated with….
FDA Accepts Biogen Biologics License Application for Biosimilar Candidate Referencing ACTEMRA (tocilizumab)
Biogen Inc. recently announced the US FDA has accepted for review the abbreviated Biologics License Application (aBLA) for BIIB800, a biosimilar candidate referencing ACTEMRA (tocilizumab),…
Catalent Opens One of the World’s Largest Commercial-Scale Cell Therapy Manufacturing Facilities at its European Center of Excellence for Cell Therapies in Belgium
Catalent recently announced the completion and opening of a new commercial-scale cell therapy manufacturing facility at its European center of excellence for cell therapies in….
VYNE Therapeutics Announces First Patent Granted for Novel BET Inhibitor Platform
VYNE Therapeutics Inc. recently announced GB Patent No. 2597228, titled Compounds Comprising N-Methyl-2-Pyridone, And Pharmaceutically Acceptable Salts, has been granted by the United Kingdom’s Intellectual Property…
Summit Therapeutics Partners With Akeso Inc. in Deal for Up to $5 Billion to In-License Breakthrough Innovative Bispecific Antibody
Summit Therapeutics Inc. recently announced a definitive agreement of its partnership with Akeso Inc. to in-license its breakthrough bispecific antibody, ivonescimab…..
Evonik Launches Plant-Based Squalene to Boost Vaccine Efficacy
Evonik has recently launched a non-animal-derived squalene suitable for vaccines and other pharmaceutical applications. PhytoSquene is the first known amaranth oil-derived squalene on the market…
Enzyvant Announces Merger With Altavant
Combined company has full range of capabilities required to advance bold science for rare diseases in immunology and cardiopulmonology….
Ocuphire Pharma Announces Submission of NDA for Eye Drops for Reversal of Mydriasis
Ocuphire Pharma, Inc. recently announced the submission of a New Drug Application (NDA) to the US FDA for Phentolamine Ophthalmic Solution 0.75% (Nyxol) for the…
Athira Pharma Presents Preclinical Data Supporting Therapeutic Potential of ATH-1105 in ALS
ATH-1105 demonstrated consistent improvements across measures of motor function, nerve function, and neurodegeneration in a TDP-43 mouse model of ALS….
Wheeler Bio Licenses ATUM’s Proprietary Leap-In Transposase Technology in Support of its Disruptive CDMO Service Offering
Wheeler Bio, Inc. recently announced it has been granted a license to ATUM’s proprietary Leap-In Transposase and miCHO cell line development technology…..
Bluejay Therapeutics Received Regulatory Clearance to Initiate Clinical Studies of BJT-778 for Treatment of Chronic Hepatitis B & Chronic Hepatitis D
Bluejay Therapeutics recently announced it received approval from the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) and from the local Ethics Committee to…
Synlogic Announces SYNB1353 Achieves Proof of Mechanism for Treatment of Homocystinuria & Provides Business Update
Synlogic, Inc. recently announced SYNB1353 has achieved proof of mechanism and positive results in a Phase 1 study in healthy volunteers treated with multiple ascending…
Roivant & Pfizer Form New Vant Company Focused on Developing TL1A Drug Candidate for Inflammatory & Fibrotic Diseases
Roivant to develop PF-06480605 (now RVT-3101), a potential first in class, fully human monoclonal antibody that blocks tumor necrosis factor-like ligand 1A (TL1A), a cytokine believed to play a key role in inflammation and fibrosis ….
2023 COMPANY PROFILES & CAPABILITIES
For each participating company, this section presents a detailed summary highlighting their core technologies, capabilities, technologies, and services.
ARMED ONCOLYTIC VIRUSES - Instant Messaging Cancer With a TGF-Beta Trap Carrying Oncolytic Adenovirus
Bryan Oronsky, MD, PhD, says cancer is a systemic disease, the eradication of which heavily depends on immune responses. In his opinion, to overcome impaired immune cell function and, hence, to increase the responsiveness to CIs likely requires a 3-in-1 therapy.
Ryvu Therapeutics & BioNTech Enter Global Collaboration to Develop & Commercialize Immuno-Modulatory Small Molecule Candidates
The companies enter a multi-target research collaboration to develop multiple small molecule programs targeting immune modulation in cancer and potentially other disease areas based on targets selected by BioNTech….
First Wave BioPharma Files INDA for Enhanced Microgranule Delivery Formulation of Adrulipase
Upon IND clearance, Phase 2 clinical trial investigating adrulipase as a treatment for exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis is expected to initiate in early 2023 with topline clinical data anticipated in the first half of 2023….
Aridis Announces a Key Milestone of Closing of Patient Enrollment in the Phase 2a Study of AR-501 in Cystic Fibrosis Patients
Aridis Pharmaceuticals, Inc. recently announced the closing of patient enrollment in the multiple ascending dose (MAD) and dose-ranging cohorts in the Phase 2a clinical trial of…
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).