Bio Platforms
Ocular Therapeutix Achieves Target Randomization of 555 Subjects in SOL-R
Ocular Therapeutix, Inc. recently announced that its SOL-R registrational trial of AXPAXLI™ (also known as OTX-TKI) in wet age-related macular degeneration (wet AMD) has achieved…
Enara Bio’s DARKFOX Discovery Propels ENA101, the First Ever Bispecific T Cell Engager Targeting a Cancer-Specific Dark Antigen, Into IND-Enabling Studies
Enara Bio recently will unveil its newly discovered cancer-specific target DARKFOX as the first in a new category of validated Dark Antigens in an oral…
Eder Therapeutics & XCubeBio Partner to Establish Joint NewCo for Global Development & Future Market Entry Activities
Eder Therapeutics, a Canadian biopharmaceutical company focused on rare genetic disorders and XCubeBio AG, a biotechnology platform company focused on accelerating successful international market entry…
Rein Therapeutics Receives FDA Clearance to Resume Phase 2 Trial of LTI-03 in Idiopathic Pulmonary Fibrosis
Rein Therapeutics recently announced the US FDA has lifted the full clinical hold on the Company’s Phase 2 “RENEW” trial evaluating LTI-03 in patients with…
Piramal Pharma Solutions Formalizes Partnership With IntoCell, Expanding its Payload-Linker Platform & Bioconjugate Capabilities
Piramal Pharma Solutions has signed a Memorandum of Understanding (MOU) to solidify its strategic partnership and explore potential collaboration opportunities in ADC development with IntoCell Inc.…
Zag Bio Launches With $80 Million Financing to Advance Thymus-Targeted Medicines for Autoimmune Diseases
Zag Bio, Inc. recently announced the company’s launch with $80 million in financing, including a recently closed Series A financing. Polaris Partners founded and incubated Zag…
Slingshot Biosciences & Cellares Combine TruCytes Biomarker Controls With Cell Q Platform to Enhance Quality & Automation in Cell Therapy Manufacturing
Slingshot Biosciences recently announced a strategic collaboration with Cellares that integrates Slingshot’s TruCytes Biomarker controls into Cellares’ Cell Q, the industry’s first fully automated quality-control…
Zenas BioPharma Announces Positive Results From Phase 2 MoonStone Trial of Obexelimab in Relapsing Multiple Sclerosis
Zenas BioPharma, Inc. recently announced positive results from the Phase 2 MoonStone trial of obexelimab in Relapsing Multiple Sclerosis (RMS). Obexelimab met the primary endpoint,…
IN8bio Presents T cell Engager Data Demonstrating Deep B Cell Depletion for Autoimmune Indications
IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, today presented new preclinical data…
Enterome Receives FDA Fast Track Designation in Follicular Lymphoma for Lead OncoMimics Immunotherapy
Enterome recently announced the US FDA has granted Fast Track designation for follicular lymphoma in the low tumor burden “watch-and-wait” setting for its lead OncoMimics…
Gyre Therapeutics Announces Completion of Patient Enrollment in Phase 3 Clinical Trial of Pirfenidone Capsules for the Treatment of Pneumoconiosis
Gyre Therapeutics recently announced its indirect, majority-owned subsidiary, Gyre Pharmaceuticals Co., Ltd. (Gyre Pharmaceuticals), has completed patient enrollment in the 52-week Phase 3 clinical trial…
Amphista Therapeutics Nominates Orally Available Targeted Glue Degrader as its First Clinical Development Candidate
Amphista Therapeutics recently announced the nomination of AMX-883; a potent, selective and orally bioavailable degrader of BRD9, as its first clinical development candidate. Amphista is…
Tagworks Pharmaceuticals Announces CTA Authorization & Initiation of Phase 0/1 Clinical Trial for TGW211, a Radiopharmaceutical for the Treatment of HER2+ Tumors
Tagworks Pharmaceuticals BV recently announced the Dutch regulatory authorities approved the Clinical Trial Application (CTA) for the CleavHER Phase 0/1 clinical trial, sponsored by the…
Denali Therapeutics Announces FDA Review Extension of BLA for Tividenofusp Alfa for the Treatment of MPS II (Hunter Syndrome)
Denali Therapeutics Inc. recently announced the US FDA has extended its review timeline of the Biologics License Application (BLA) seeking accelerated approval of tividenofusp alfa…
Bicara Therapeutics Announces Ficerafusp Alfa Granted Breakthrough Therapy Designation
Bicara Therapeutics Inc. recently announced the US FDA has granted Breakthrough Therapy Designation (BTD) to ficerafusp alfa in combination with pembrolizumab for the first line…
Shape Therapeutics Shares Breakthrough Preclinical Data for Non-Invasive Treatment of Parkinson’s Disease Using a Brain Targeted AAV Gene Therapy
Shape Therapeutics recently announced it presented new preclinical data highlighting the therapeutic potential of its lead product (SHP-201), a disease-modifying gene therapy for the treatment…
AviadoBio & UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients With Retinal Disease
AviadoBio Ltd. and UgeneX Therapeutics, today announced an exclusive option and license agreement for the development and commercialization of UGX-202, an investigational, AAV-based gene therapy…
Ligand Partner SQ Innovation Receives FDA Approval for Lasix ONYU, an At-Home Treatment for Edema in Heart Failure Patients
Ligand Pharmaceuticals Incorporated recently announced that its partner SQ Innovation Inc. has received approval from the US FDA for Lasix ONYU (furosemide injection), a novel…
New Preclinical Data From Encoded Therapeutics Demonstrate Therapeutic Potential of its One-time, Non-opioid Gene Therapy Candidate for Chronic Pain
Encoded Therapeutics Inc. recently announced preclinical data demonstrating that its AAV9 microRNA (miRNA) gene therapy candidate achieved robust knockdown of SCN9A (NaV1.7), a key mediator of…
Rein Therapeutics Receives European Regulatory Approval to Initiate Phase 2 Trial of LTI-03 in Idiopathic Pulmonary Fibrosis
Rein Therapeutics recently announced it has received authorization from the European Medicines Agency (EMA) to initiate the Company’s Phase 2 RENEW clinical trial of its…
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).
















