Bio Platforms
Investigational Treatment for Fragile X Syndrome Receives Rare Pediatric Disease Designation From FDA
Shionogi & Co., Ltd. recently announced the US FDA has granted Tetra Therapeutics Inc. Rare Pediatric Disease Designation for zatolmilast (BPN14770), an investigational treatment being…
Omega Therapeutics Announces Promising Preliminary Clinical Data for OTX-2002 From Ongoing MYCHELANGELO I Trial
Omega Therapeutics, Inc. recently announced encouraging preliminary safety, tolerability, pharmacokinetic, and translational data from the initial two dose level cohorts (n=8) from Part 1 of its ongoing Phase 1/2 MYCHELANGELO I study evaluating….
bYoRNA & Dyadic Announce a Collaboration Targeting the Production of Abundant, Low-Cost mRNA From C1-Cells
bYoRNA SAS and Dyadic International, Inc. recently announced they entered into a development and commercialization agreement to work on disruptive bioproduction technologies that will help…
SOHM Announces Acquisition of ABBIE, a World-Class Gene-Editing Platform That Can Deliver Genetic Payloads Using Non-Viral Vectors
With this acquisition, SOHM is well-positioned as a competitive player in the $5.3 billion gene-editing market in 2023. The gene-editing market is expected to grow….
AcuraStem Enters Into License Agreement With Takeda to Advance PIKFYVE Therapeutics
AcuraStem recently announced it has entered into a license agreement with Takeda to develop and commercialize AcuraStem’s PIKFYVE targeted therapeutics including AS-202, an innovative antisense oligonucleotide…
Coya Therapeutics Licenses Exclusive Worldwide Rights to Exosome Engineering Technology From Carnegie Mellon University
Coya Therapeutics, Inc. recently announced licensing of the exclusive, worldwide rights of a proprietary Exosome Engineering Technology from CMU with potential applications across multiple indications, including…
Clene Reports Significant Long-Term Survival Improvement From CNM-Au8 Treatment in HEALEY ALS Platform Trial Compared to PRO-ACT Historical Controls
Clene Inc. recently announced long-term follow-up data for patients treated with CNM-Au8 30 mg for up to 133 weeks in the HEALEY ALS Platform Trial.…
Replay Announces Partnership Between Syena & JURA Bio to Advance T Cell Receptor NK Therapies in Cancer
Replay recently announced a research collaboration between JURA Bio and Syena to develop T cell receptor (TCR) based therapies. “JURA Bio’s highly differentiated TCR discovery…
Revolo Biotherapeutics Announces Completion of Phase 1 Multiple Ascending Dose Clinical Trial of Immune-Resetting Drug Candidate
Revolo Biotherapeutics recently announced the completion of a Phase 1 multiple ascending dose (MAD) clinical trial evaluating the safety and tolerability of the company’s immune-resetting…
Oxford Biomedica & Institut Mérieux Enter Exclusive Negotiations With Respect to Proposed Acquisition by Oxford Biomedica of ABL Europe From Institut Mérieux as Part of Pure-Play CDMO Transformation
Oxford Biomedica plc and Institut Mérieux SA recently announced they have entered into exclusive negotiations with respect to the proposed acquisition by Oxford Biomedica of…
Vir Biotechnology Announces First Participant Dosed in New Phase 1 Trial Evaluating Investigational T Cell Vaccine for the Prevention of HIV
Vir Biotechnology, Inc. recently announced the first participant has been dosed in a Phase 1 trial evaluating the safety, reactogenicity, and immunogenicity of VIR-1388, an investigational novel T cell vaccine for….
LIXTE Biotechnology Announces a Supported Collaborative Trial to Study LIXTE’s First-in-Class PP2A Inhibitor, Plus GSK’s Immunotherapy in Clear-Cell Ovarian Cancer
The Phase 1b Clinical Trial Focuses on Assessing the Safety and Efficacy of the Two-Drug Combination in a Cancer Associated with Longer Survival to Immunotherapy When Genetically Deficient in PP2A….
ALSA Ventures Launches Novel Gene Therapy Portfolio Company Axovia Therapeutics
ALSA Ventures recently announced the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd. Axovia is developing the…
BioSenic Publishes New Evidence of Beneficial Effects of Arsenic Trioxide Treatment Supporting Clinical Trial for Systemic Sclerosis
BioSenic recently announced publication of data providing additional key indications of its lead API (Active Pharmaceutical Ingredient) arsenic trioxide (ATO) to treat systemic sclerosis (SSc)…
Samsung Biologics Announces Expanded Strategic Agreement With Bristol Myers Squibb
Samsung Biologics recently announced a new agreement with Bristol Myers Squibb for large-scale manufacturing of a Bristol Myers Squibb commercial antibody cancer drug….
BioNTech & CEPI Announce Partnership to Advance mRNA Mpox Vaccine Development & Support CEPI’s 100 Days Mission
BioNTech SE and the Coalition for Epidemic Preparedness Innovations (CEPI) recently announced a strategic partnership to advance mRNA-based vaccine candidates with the development of….
Orchard Therapeutics Announces Acceptance of BLA for OTL-200 in MLD; Receives Priority Review
Orchard Therapeutics recently announced the US FDA has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority…
Rani Therapeutics Initiates Phase 1 Study of RT-111 (RaniPill Containing Ustekinumab Biosimilar, CT-P43)
Rani Therapeutics Holdings, Inc. recently announced the initiation of a Phase 1 clinical trial to evaluate the safety and tolerability of RT-111, an orally administered…
POINT Biopharma & Athebio Announce Partnership to Develop Designed Ankyrin Repeat Protein Targeted Radioligands
POINT Biopharma Global Inc. and Athebio AG recently announced a collaboration and license agreement to develop and commercialize DARPin-targeted radioligands (Radio-DARPins). DARPins are an attractive…
New Collaboration With Stevanato Group to Elevate mRNA Production With Nfinity Platform
Quantoom Biosciences recently announced a new collaboration with Stevanato Group, a leading global provider of drug containment and delivery solutions to the pharmaceutical, biotechnology, and life science industries, with the goal 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).