Bio Platforms
Cullinan Oncology Announces Phase 1/2a Interim Data for Cullinan Pearl’s CLN-081 in NSCLC EGFR Exon 20 Patients
Cullinan Oncology, Inc. recently announced additional details pertaining to Cullinan Pearl’s ongoing Phase 1/2a trial of CLN-081 in Non-Small Cell Lung Cancer (NSCLC) patients whose…
Mirati Therapeutics & Zai Lab Enter Development & Commercialization Collaboration
Mirati Therapeutics, Inc. and Zai Lab Limited recently announced the companies have entered into a collaboration and license agreement for adagrasib, a small-molecule….
Immutep Enters Into New Collaboration With Merck KGaA
Immutep Limited recently announced a new collaboration and supply agreement with Merck KGaA , Darmstadt, Germany, for a Phase 1/2a clinical trial in patients with….
Corbus Pharmaceuticals Licenses Two Integrin Targeting mAbs Further Expanding Pipeline Into Cancer & Fibrotic Diseases
Corbus Pharmaceuticals Holdings, Inc. recently announced licensing deals for two new monoclonal antibodies (mAbs), CRB-601 and CRB-602, that target integrins to inhibit activation of….
PDS Biotechnology Establishes Partnership With Head & Neck Cancer Alliance
PDS Biotechnology Corporation and the Head and Neck Cancer Alliance (HNCA) recently announced a partnership that seeks to raise awareness of new and developing treatment…
uniQure Announces Positive Recommendation to Advance Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
uniQure N.V. recently announced the independent Data Safety Monitoring Board (DSMB) overseeing the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease…
Ocugen On Track to Submit EUA Application to US FDA for its COVID-19 Vaccine Candidate
Ocugen, Inc. recently confirmed its plan to submit its Emergency Use Authorization (EUA) application for COVAXIN to the US FDA in June. “Since we have…
GENE-EDITING TECHNOLOGY - Expanding the CRISPR Toolbox for Genome Editing
Ashley Jacobi says while there are many ever-improving tools available to scientists performing ground-breaking research, and the potential of CRISPR genome editing appears limitless, there remain challenges that need to be overcome to realize the technology’s full potential.
EXECUTIVE INTERVIEW - Poseida Therapeutics: Creating the Next Wave of Cell & Gene Therapies With the Capacity to Cure
Eric M. Ostertag, MD, PhD, Chief Executive Officer of Poseida Therapeutics, discusses the company’s innovative approach to develop safer, more effective, accessible and affordable cell and gene therapies for patients.
Ginkgo Bioworks & Biogen Announce Collaboration & License Agreement to Develop Novel Gene Therapy Manufacturing Platform
Ginkgo Bioworks and Biogen recently announced a gene therapy collaboration in which the companies aim to redefine the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors…..
Xalud Therapeutics’ Lead Candidate Achieves Multiple Clinical Milestones in Pathologic Inflammation & Neuroimmunology Therapeutic Areas
Xalud Therapeutics recently announced that the Phase 2b study of XT-150 in patients with moderate-to-severe pain due to osteoarthritis (OA) of the knee has completed…
VBL Therapeutics to Provide Update on OVAL Phase 3 Registration Enabling Study of VB-111 in Ovarian Cancer
VBL Therapeutics recently announced that a poster providing an update on the OVAL Phase 3 registration enabling clinical trial of VB-111 in ovarian cancer will…
Onconova Therapeutics Announces Initial Dosing of First Patient in US Phase 1 Clinical Trial
Onconova Therapeutics, Inc. recently announced the first patient has been dosed in the US Phase 1 clinical trial of ON 123300, the company’s proprietary, novel…
The Discovery Labs Signs Foundational Lease With the University of Pennsylvania Gene Therapy Program as Anchor Tenant
The Discovery Labs has recently signed a foundational lease with the University of Pennsylvania Gene Therapy Program (GTP), which will use Discovery Labs’ suburban campus…
CatalYm to Present Initial Data of First-in-Human Trial of GDF-15 Neutralizing Antibody CTL-002
CatalYm GmbH recently announced that their abstract with first interim data from the Phase 1 clinical trial investigation CTL-002 as monotherapy and in combination with…
Biogen & Envisagenics Announce Collaboration to Advance RNA Splicing Research
Biogen Inc. and Envisagenics recently announced a new collaboration to advance ribonucleic acid (RNA) splicing research within central nervous system (CNS) diseases….
Mustang Bio & City of Hope Announce First Patient Dosed in Phase 1 Clinical Trial
Mustang Bio, Inc. and City of Hope recently announced the first patient has been dosed in a clinical trial to establish the safety and feasibility…
Calithera Biosciences & Antengene Enter Worldwide License Agreement for Development & Commercialization of CB-708
Calithera Biosciences, Inc. and Antengene Corporation, Ltd. recently announced an exclusive, worldwide license agreement for the development and commercialization of CB-708, Calithera’s small….
Agenus & Bristol Myers Squibb Announce Exclusive Global License for Anti-TIGIT Bispecific Antibody Program
Bristol-Myers Squibb Company and Agenus Inc. recently announced they have entered into a definitive agreement under which Bristol Myers Squibb will be granted a global exclusive license to….
uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients With Pre-existing Antibodies to AAV5 Vector
uniQure N.V. recently presented 26-week clinical data from the pivotal, Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients…
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).