RecipharmCobra Biologics, Keele (previously Cobra Biomanufacturing Plc) has been producing plasmid DNA for clinical trials for more than 10 years and has an approved site under the EU clinical trials directive. During this period, the company has produced more than 40 plasmids (ranging from 500 mg to 5 g) for 25 customers in Europe and the United States. These plasmids have been used for gene therapy and vaccines as well as to produce viral vectors. RecipharmCobra has developed…
Pre-Clinical and Clinical Trials
Fluorescence-Activated Cell Sorting for CGMP Processing of Therapeutic Cells
Cell therapy using embryonic or adult stem cells for regenerative medicine is generating high interest in the global medical community and in the general population.Physicians and patients are looking to cell therapies as potentially curative treatments for diseases such as diabetes, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Graft versus Host disease (GvHD), and cancer. Cell-based therapeutic products have been administered in clinics for nearly 90 years in the form of blood transfusions and for 50 years in the…
Quantitative Crowdsourcing
This past fall, we collaborated with researchers from MIT and Harvard Business School to develop and launch the Pharmer’s Market, a prediction market that aggregates the opinions, insight, and experience of a wide range of pharmaceutical professionals to forecast outcomes of drug clinical trials. The market is designed to be a showcase of how pharmaceutical development could run in the future. This concept was born when we agreed on a critical premise: Discovery should be a collective process. That isn’t…
A Formulation Strategy for Quickly Reaching Clinical Trials
The aim of any company making protein-based therapeutics is to get to the clinic quickly with a product formulation that has the best chance of success. Any number of specific formulation development and manufacturing issues can keep such drugs from advancing expeditiously to the clinic. To be successful organizations must balance the strengths and weaknesses of each individual molecule against timelines, budgets, and priorities. Ultimately, it’s not just about deploying the best methodologies and processes, but of applying them appropriately…
A Few Small Steps for Biotech Will Yield Giant Leaps for Humankind
Biotechnology’s mission has never been more critical. In a call to action reminiscent of President Kennedy’s challenge to place a man on the moon in the 1960s, President Obama has challenged the scientific community to seek “a cure for cancer in our time.” The challenge is tremendous, but the place to look for such a momentous and meaningful achievement is among the great minds of scientists and inside the research laboratories of biotech companies. And that is but the tip…
Overcoming Barriers to Success
Eli Lilly’s 2001 annual report stated that “Xigris… will prove to be one of our industry’s genuine breakthroughs” (1). The company believed that its first-in-class drug “could help save one in five people who otherwise would die” from severe sepsis. One of the world’s oldest and most virulent killers, sepsis is the third most common cause of death for hospitalized patients in the developed world after heart disease and cancer. In the United States alone, 750,000 patients are hospitalized with…
A Vital Link
Clinical studies serve as a bridge between biopharmaceutical laboratories and the patients who need therapies. Drugs need to be tested in small populations before they are made available to the world at large. In a 2006 interview with BioProcess International, editorial advisor Michiel Ultee (vice president of Process Sciences for Laureate Pharma) said, “Until your product is tested in humans — and shown to be safe and to have some efficacy — then you really don’t have…
Manufacturing Patient-Specific Cell Therapy Products
Several cellular therapies are currently progressing through clinical development with the potential to address unmet medical needs affecting millions of patients. As cell-based therapeutics receive regulatory approval and reach the market, the primary challenge will quickly become manufacturing such products in sufficient volume to meet demand. Aastrom Biosciences has developed tissue-repair cell (TRC) technology for use in autologous, patient-specific cellular therapy (PSCT) and is conducting late-stage clinical trials both in the United States and Europe. TRCs are derived from a…
In the Drug Delivery Zone
A rule of thumb in drug development states that the larger a therapeutic molecule is, the more trouble it will be to make, ship/store, and administer to patients. Biotherapeutics include proteins (such as antibodies), vaccines, some smaller peptides (such as hormones), DNA for gene-transfer therapies, cells and tissues, and to a lesser extent blood-fractionation products, allergenics, and RNA/oligonucleotides. Biomolecules are big and unwieldy, they’re produced in complex mixtures by biological processes, and they face numerous challenges in storage and within…
Cell Cultivation Process Transfer and Scale-Up
The introduction of therapeutic monoclonal antibodies (MAbs) has greatly revolutionized therapies for several cancer immune disorders (1,2,3,4,5). Benefits to patients have been substantial, translating into both increased life expectancy and improved quality of life. Currently, twenty-one therapeutic MAbs are registered for marketing in the United States, with the introduction of several more expected in the coming years (6,7,8,9,10,11). PRODUCT FOCUS: MONOCLONAL ANTIBODIESPROCESS FOCUS: Technology transfer (production) and analytical methods developmentWHO SHOULD READ: PRODUCTION AND PROCESS DEVELOPMENT, MANUFACTURINGKEYWORDS: IGF-1R, EXPRESSION, TECH…