Bio Platforms
ViGeneron Signs Global Development & Commercialization Agreement With Biogen
ViGeneron GmbH recently announced a global collaboration and licensing agreement with Biogen Inc. to develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors to treat….
University of Calgary Joins the Phase 2 Trial of LSALT Peptide for the Treatment of Complications in Hospitalized COVID-19 Patients
Arch Biopartners Inc. RECENTLY announced the University of Calgary Cumming School of Medicine has joined the Phase 2 trial of its lead drug LSALT peptide (Metablok), targeting the prevention….
Akston Biosciences & LakePharma Announce Strategic Partnership to Manufacture Second-Generation COVID-19 Vaccine
Akston Biosciences and LakePharma recently announced they have established a strategic partnership in which LakePharma will manufacture commercial quantities of Akston’s adjuvated COVID-19 vaccine candidate (AKS-452),…
Oncorus to Build GMP Viral Immunotherapy Clinical Manufacturing Facility
Oncorus, Inc. recently announced it has signed a 15-year lease to build a state-of-the-art, 88,000-square-foot Good Manufacturing Practice (GMP) viral immunotherapy clinical manufacturing facility in…
Inozyme Pharma Announces Authorization to Proceed in US & UK With Phase 1/2 Clinical Trial
Inozyme Pharma, Inc. recently announced the US FDA has cleared the company’s Investigational New Drug (IND) application and that the United Kingdom Medicines and Healthcare…
Passage Bio Teams With Catalent to Start cGMP Manufacturing for Lead Gene Therapy Products
Passage Bio, Inc. and Catalent recently announced that manufacturing operations have commenced to support adeno-associated virus (AAV) production for Passage Bio’s lead….
Theratechnologies Announces New Findings for its Lead Investigational Compound for the Treatment of Several Additional Cancers
Theratechnologies Inc. recently announced new preclinical in vivo findings regarding the efficacy and tolerability of its novel investigational proprietary peptide-drug conjugate (PDC), TH1902, for the….
Ionis Announces Initiation of the Global Phase 3 BALANCE Study
Ionis Pharmaceuticals, Inc. recently announced the initiation of the Phase 3 BALANCE study for AKCEA-APOCIII-LRx in adult patients with familial chylomicronemia syndrome (FCS). FCS is…
BioAge Raises $90 Million to Treat Aging & Age-Related Diseases
BioAge Labs, Inc. recently announced it has raised $90 million in an oversubscribed Series C financing. The raise was co-led by Andreessen Horowitz and serial…
Galecto Publishes GB0139 Phase 2a Idiopathic Pulmonary Fibrosis (IPF) Results
Galecto, Inc. recently announced the publication of a paper detailing full results from a Phase 2a study of GB0139 in Idiopathic Pulmonary Fibrosis (IPF) in…
Biogen & Sage Therapeutics Announce Global Development & Commercialization Collaboration
Biogen Inc. and Sage Therapeutics, Inc. recently announced they have executed a global collaboration and license agreement to jointly develop and commercialize zuranolone (SAGE-217) for…
Samsung Biologics Breaks Ground on Super Plant, the World’s Largest & Most Innovative Bio-Manufacturing Facility
Samsung Biologics officially began construction of its Plant 4 in Incheon, South Korea. Dubbed the "Super Plant,” the new, multi-story 238,000-square-meter construction will be the…
Gene Therapy Market to Surpass $6.21 Billion Globally, by 2027 at 13.4% CAGR
Allied Market Research published a report, titled Gene Therapy Market by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide,…
BioNTech & InstaDeep Announce Strategic Collaboration & Form AI Innovation Lab to Develop Novel Immunotherapies
BioNTech SE and InstaDeep Ltd recently announced a multi-year strategic collaboration aimed at applying the latest advances in artificial intelligence (AI) and machine learning (ML) technology to develop….
Aravive Announces Phase 3 Trial Design for AVB-500 in Platinum Resistant Ovarian Cancer
Aravive, Inc. recently announced the company has received guidance from the US FDA on a Phase 3 trial design for AVB-500 in platinum-resistant ovarian cancer…
uniQure Announces Positive Top-Line Data From the HOPE-B Pivotal Trial of Gene Therapy
uniQure N.V. recently announced positive top-line data from its pivotal, Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based…
AB201 Development as a Potential Treatment for COVID-19 Receives US FDA Fast-Track Designation
ARCA biopharma, Inc. recently announced the US FDA has designated as a Fast-Track development program the investigation of AB201 as a potential treatment for COVID-19.…
2021 COMPANY PROFILES & CAPABILITIES
For each participating company, this section presents a detailed summary highlighting their core technologies, capabilities, technologies, and services.
PLATFORM TECHNOLOGY - Overcoming the Challenges of Drug Brain Delivery With a Novel Brain Delivery Vector
Mei Mei Tian, PhD, explains how the xB3 platform has the potential to support development of treatments for CNS diseases that can treat thousands of patients in the years ahead.
VACCINE DEVELOPMENT - COVID-19 Vaccine Focusing on T Cells to Protect the Most Vulnerable
Jeffrey Wolf, MBA, JD, says while most vaccines in development are targeted to protect healthy people, few are focusing on those who are most at risk – seniors and those with co-morbidities like heart failure, obesity, or type 2 diabetes.
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).