Bio Platforms
Adverum Biotechnologies Announces IND Active for ADVM-022, a Novel Gene Therapy
Adverum Biotechnologies, Inc. recently announced its Investigational New Drug (IND) application is active for the planned multi-center, open-label, Phase 1, dose-escalation study of ADVM-022, a…
Cynata’s Stem Cell Therapy Meets All Safety & Efficacy Endpoints in Trial
Australian stem cell and regenerative medicine company Cynata Therapeutics Limited recently announced that CYP-001, its lead Cymerus mesenchymal stem cell (MSC) product candidate, met all clinical endpoints in a Phase 1 trial for the treatment of steroid-resistant acute graft-versus-host disease (GvHD).
DRUG DEVELOPMENT - Cell & Gene Therapies Calling for Innovation in Drug Development
Lev Gerlovin and Pascale Diesel, PharmD, believe there is significant evidence to indicate that we are entering a golden age of gene and cell therapy development, and while industry works to advance these programs, the debate over new approaches in both structuring and financing clinical research is likely to continue.
DRUG DELIVERY - Targeted Delivery of Submicron Particle Cancer Chemotherapy: Helping Shift the Immunotherapy Paradigm
Marc A. Iacobucci says that with much attention and research investment into IOT, NanOlogy is shaping a new paradigm for cancer treatment by attempting to improve the safety and effectiveness of tried and true CT through local delivery.
Novan Announces Promising Clinical Results With SB414
Novan, Inc. recently announced an update on the SB414 nitric oxide-releasing cream product candidate and its application to two therapeutic indications: atopic dermatitis and psoriasis.…
Immunocore Announces Start of Phase I ImmTAC Study in Patients with Solid Tumors
Immunocore Limited recently announced it has dosed the first patient in a Phase I study, part of an ongoing collaboration with GlaxoSmithKline (GSK). The study,…
Evotec & Novo Nordisk Form Strategic Research Alliance
Evotec AG recently announced a strategic alliance with Novo Nordisk to discover and develop novel small molecule therapies to treat patients suffering from diabetes and…
CCAB Partners With ImmunoBiochem as it Launches New Co-Development Strategy to Advance Early-Stage Biotech Companies
The Centre for the Commercialization of Antibodies and Biologics (CCAB) and ImmunoBiochem Corporation have announced an agreement that will see CCAB provide a new investment to help advance ImmunoBiochem’s novel breast cancer therapeutic candidate one step closer to the clinic.
Orchard Therapeutics Announces $150-Million Financing to Advance Pipeline
Orchard Therapeutics recently announced the completion of an oversubscribed $150 million Series C financing. Deerfield Management led the financing with significant new investments from RA…
Stemline Therapeutics Announces FDA Accepts ELZONRIS BLA & Grants Priority Review
Stemline Therapeutics, Inc. recently announced US FDA has accepted for filing the company’s Biologics License Application (BLA) for ELZONRIS (tagraxofusp; SL-401) for the treatment of…
MeiraGTx Announces AAV-CNGA3 Has Received Orphan Drug Designation
MeiraGTx Holdings Plc recently announced that the US FDA has granted orphan drug designation (ODD) for its AAV-CNGA3 gene therapy product candidate for the treatment…
Mustang Bio & St. Jude Children’s Research Hospital Enter Exclusive Worldwide License Agreement
Mustang Bio, Inc. and St. Jude Children’s Research Hospital recently announced they have partnered and entered into an exclusive worldwide license agreement for the development…
CytoDyn Announces Strong Preclinical Results Using PRO 140 in Human Colon Carcinoma
CytoDyn Inc. recently announced that PRO 140 (leronlimab) has been shown effective at inhibiting the growth of a human colon carcinoma cell line (SW480) in…
International Stem Cell Corporation Developed 3D Liver Structures
International Stem Cell Corporation recently announced that ISCO’s Research and Development team has developed novel methods that efficiently generate human 3D liver-like tissue. According to…
Fortress Biotech Announces Aevitas Therapeutics Enters Sponsored Research Agreement to Advance AAV Gene Therapy Technology
Fortress Biotech recently announced that its subsidiary, Aevitas Therapeutics, Inc. has entered into a sponsored research agreement (SRA) with the laboratory of Wenchao Song, PhD,…
Avalon GloboCare Forms Strategic Partnership With Weill Cornell Medical College to Co-develop Technologies & Bio-production of CAR-T Therapy
This strategic partnership aims to co-develop bio-production and standardization procedures in procurement, storage, processing, clinical study protocols, and bio-banking for Chimeric Antigen Receptor (CAR)-T therapy, in accordance with the Foundation of Accreditation for Cellular Therapy (FACT) and American Association of Blood Banks (AABB) standards.
ARCA Biopharma Announces Positive Outcome of End-of-Phase 2 Meeting With FDA
ARCA Biopharma, Inc. recently announced it has received guidance from the US FDA following an End-of-Phase 2 meeting regarding the Phase 3 program for ARCA’s…
BioPharmX Receives IRB Approval to Initiate Phase 2 Trial
BioPharmX Corporation recently announced it has received Institutional Review Board approval for its Phase 2 study of BPX-04 for the treatment of papulopustular rosacea. The…
Translate Bio Announces Closing of Collaboration & Licensing Agreement with Sanofi Pasteur
Translate Bio recently announced the closing of a previously announced research collaboration and licensing agreement with Sanofi Pasteur to develop mRNA vaccines for up to…
WuXi Biologics & Immune Pharmaceuticals Announce Late-Phase Development & Manufacturing Partnership
WuXi Biologics and Immune Pharmaceuticals recently announced a development and manufacturing agreement for the production of bertilimumab, Immune’s first-in-class anti-eotaxin-1 monoclonal antibody. Bertilimumab is designed…
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).