Nonantibody Biologics

Peptide Therapies: Designing a Science-Led Strategic Quality Control Program

Peptide therapeutics are a unique class of pharmaceuticals. They can fall into one or more regulatory categories: as conventional chemical molecules, biological entities, or biosimilars. Developing a well–thought-out quality control (QC) strategy is key to meeting development milestones and complying with evolving regulatory requirements. Safety assessment is critical because slight changes in the structure, physicochemical properties, stability, and impurity profile of a peptide can provoke an adverse immune response. A robust analytical QC program should be conducted in laboratories that…

challenges with exosome therapy

eBook: Challenges in Industrial Process Development of Exosome-Based Therapies: Characterizing and Managing Diversity

The traditional classification of extracellular vesicles (EVs) includes three types: exosomes, microvesicles, and apoptotic vesicles. Each type arises from a distinct origin and exhibits distinct characteristics. The problem is that their size ranges overlap and that the major surface proteins presented by exosomes also are present on the surfaces of microvesicles and apoptotic bodies. This makes it a challenge for process developers to identify the vesicle fraction that best serves a particular exosome therapy. Anion-exchange chromatography (AEC) can fractionate EVs…

Fighting Alzheimer’s Disease with a Breakthrough Peptide

Even more so than cancer, Alzheimer’s disease is one disease that many people fear greatly. The thought of falling prey to the inevitable desecration of our own minds is something that makes even the bravest among us shudder. If we’re robbed of our sense of who we really are, we imagine, then we are doomed to live our last days without the dignity that defines us and that we hold most dear. The ultimate horror of the condition is that…

eBook: Peptide Therapies — Small Biomolecules Address Big Health Problems

This crash course in the development of peptide therapies brings together two articles: one on product research and the other on manufacturing and regulatory concerns. In “Fighting Alzheimer’s Disease from Within: A Breakthrough Peptide,” freelance journalist Marc Davis highlights current developments in Alzheimer’s disease treatment, focusing specifically on a peptide approach from Canadian company NervGen. In “Making Peptides Work,” BPI’s senior technical editor adds perspective on developing and manufacturing peptide therapies from the Informa Connect community, with commentary from experts…

Long RNA Synthesis Report

Long single-stranded RNA oligonucleotides are increasingly necessary for a variety of genomic pathway research and drug discovery studies. However synthesizing and purifying long RNAs can pose unique challenges due to their sequence composition, secondary structure, and applied modifications. Here we explore how Horizon Discovery is leveraging Dharmacon’s 20+ years of RNA synthesis experience to solve the challenges of long RNA oligo synthesis to address unmet research needs.

Analytical Challenges: Characterization of Oligonucleotide Therapeutics

Recent approvals of oligonucleotide therapeutics are a clear signal for optimism for this product class. This is supported by the strength of the current pipeline which has over 180 active oligonucleotide clinical programs in various phases of development. Improvements in analytical technology and know-how have played a key role in enabling suitable characterization and quality control strategies to overcome the difficulties associated with testing these complex molecules. Despite the lack of dedicated regulatory guidelines related to characterization or quality control,…

Tangential-Flow Electrophoresis: Investigation of Factors Involved in an Effective Separation of Human Serum Albumin from Human Plasma

Human plasma is a complex mixture of biomolecules such as serum albumin, immunoglobulins, coagulation factors, and others (1). These important protein biomolecules often are present at low levels or lacking in affected patients with certain life-threatening conditions. To extract those valuable proteins, a number of purification methods have been developed over time. Plasma fractionation can be traced back to the middle of the 20th century, when Edwin Cohn of Harvard University developed the first industrial process to purify proteins from…

Rolling with the ‘Tides: Elucidating the Role of Peptides and Oligonucleotides in the Biopharmaceutical Industry

In earlier issues of BPI we published a few “Elucidation” closers that we called “Defining Moments.” Since then, we have tried to distinguish key confusable terms from one another. Those presented (and sometimes “elucidated”) have been analytical and bioanalytical, spectroscopy and spectrometry, and biosimilars and biobetters. They are just a few of the many confusable terms in the biopharmaceutical industry. For example, when someone says “drug delivery,” a formulator will think of a syringe or transdermal patch, but a logistics…

Special Report on Antibody-Drug Conjugates: Technical Challenges and Opportunities

Among the emerging targeted therapies in biotechnology, antibody–drug conjugates (ADCs) hold a unique position. An ADC consists of a monoclonal antibody (MAb) with affinity to tumor cells, a cytotoxic small-molecule payload, and a linker connecting the two. Together the MAb, conjugation chemistry, and cytotoxin increase the complexity of ADCs several-fold relative to unmodified MAbs — and exponentially relative to chemotherapies. Viewing ADCs as hybrids of antibody- and chemotherapy-based cancer therapies is tempting. That description applies chemically and structurally, but ADCs’…

Bacteriophages, an Alternative to Antibiotics: Challenges and Possible Solutions for Bringing Them to Market

Bacteriophages are viruses (consisting of a genome contained within a protein coat) that specifically infect bacteria. They are the most abundant living entities on earth — the estimates range from 1030 to 1032 in total — and play key roles in regulating the microbial balance in every ecosystem where that has been explored (1). Bacteriophages are genotypic and phenotypically different from viruses that infect Archaea (Archaeovirus) and Eukarya (Eukaryovirus). The name bacteriovirus has been proposed as scientifically more accurate (2).…