Emerging Therapeutics

Bioprinting Capabilities and Futures

Bioprinting has advanced rapidly through engineering step changes in the use of three-dimensional (3D) printing. With these developments, living cells can be positioned layer by layer to produce functional tissue structures. Key attributes of this emerging technology are its high scalability and modularity, which enable automated and repeatable manufacture of a wide variety of tissues. These high-throughput biofabrication capabilities equip companies with tools to develop 3D-printed tissues for broad applications, from in vitro drug testing models to therapeutic tissue implants,…

Emerging Treatments for Spinal Cord Damage

Injuries to the spinal cord can cause permanent paralysis and even lead to death, with little or no hope for patients to regain lost function after such trauma has occurred. News of my spinal cord research first came to prominence in the 1980s with a front-page story that chronicles a presentation at the annual Society for Neuroscience meeting (1). That reported the first time that crushed peripheral nerves had been regenerated back into a patient’s spinal cord. Regeneration of spinal…

Formulation Development of Microbiome Therapeutics Localized in the Intestines

Scientists have discovered that microorganisms associated with the body play a critical role in human health (1). The intestinal microbiome is the collection of all combined genetic material of microorganisms in the intestines. An intestinal microbiota, a term frequently used interchangeably with microbiome, is the collection of such microorganisms in the intestine. With the volume of research into the human microbiome expanding, so too is the number of examples of its benefits to our health (1). Like diseases of the…

Immunotherapy: Taking Aim at Solid Cancers

As cell and gene therapies arrive on the market, all eyes have focused on autologous chimeric antigen receptor (CAR) T – cell therapies. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, delegates looked at where the biopharmaceutical industry is going in the cellular immunotherapy space. Whether for off-the-shelf CAR T-cell products, personalized cancer vaccines, or modified natural killer (NK) cells derived from human induced pluripotent stem cells (iPSCs) — cell and gene therapy development is…

Building Toward Antibody–Drug Conjugate Success

“We are witnessing one of the most significant paradigm changes in oncology drug development, with some new types of immunooncology compounds inducing unprecedented increases in survival in certain solid and liquid tumor indications.” —Jagath Reddy Junutula (Cellerant Therapeutics) and Hans-Peter Gerber (Pfizer) (1) An antibody–drug conjugate (ADC) for cancer treatment needs four things to succeed clinically: It needs to target the right antigen using the right antibody, with the most powerful cytotoxin attached by best linker option. These features combine…

Antibody–Drug Conjugate News: From BioProcess Insider

The following news items have appeared on the BioProcess Insider site over the past year. Together they indicate the direction in which the ADC sector is moving. BEYOND ADCETRIS — SEATTLE GENETICS AIMS FOR BIG BIOPHARMA STATUS 30 April 2018: Seattle Genetics is building toward a bigger future. For the first quarter 2018, the company’s total revenues grew to US$141 million (€116 million) compared with $109 million in the same period last year. This was attributed to a 36% increase…

Analytical Tools to Improve Decision-Making During Product Development

Speed to clinic testing — and then speed to market — are highly significant metrics for companies developing biopharmaceuticals. By increasing the pace of drug development, these companies can reduce costs, obtain revenues early, and establish commanding positions in the market relative to their competitors. High-throughput development tools have contributed much to the acceleration of drug development in recent years. Such technologies enable the testing of many process parameters in parallel. Combining them with multifactorial “design of experiment” (DoE) analysis…

Antibody Derivatives: Deconstructing MAbs for the Next Wave of Biotherapies

Although they make up the largest and most successful category of biopharmaceuticals so far, monoclonal antibodies (MAbs) suffer from certain disadvantages. Some companies are addressing those limitations by deconstructing MAb molecules to create new emergent therapeutics. These antibody derivatives include: antibody fusions and fragments, bispecifics, trifunctional antibodies, and more. This eBook combines market analysis from consultant David Orchard-Webb with technical discussion from BPI cofounder and senior technical editor Cheryl Scott. It also includes commentary from editorial advisor Michiel Ultee and…

Cost of Goods Is Crucial for the Future of Regenerative Medicine: CAR-T Cell Therapy Provides a Case Study in Perspective

In the history of regenerative medicine, 2017 was a critical year. With approvals for Kymriah (tisagenlecleucel) from Novartis AG, Yescarta (axicabtagene ciloleucel) from Kite Pharma (a Gilead company), and Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics, cell and gene therapies finally made their mark on the regulatory landscape. Then in 2018, those products began both treating patients and bringing in revenues for their sponsor companies. “Patients are being treated, and biotechnology and pharmaceutical companies are being paid for treating them,” said…

Apparent Matrix Effects in an Iduronate 2-Sulfatase Specific Activity Assay

The recombinant fusion protein SHP631 consists of a chimeric monoclonal antibody binding to human insulin receptor and iduronate-2-sulfatase (I2S). This product is being developed as an enzyme replacement therapy to treat cognitive symptoms of Hunter’s syndrome. Because the current therapy (idursulfase, brand name Elaprase from Shire) cannot cross the blood–brain barrier (BBB), SHP631 is being developed to do so, enabling the presence of I2S in the brain. The enzymatic activity of this molecule is measured using the substrate 4-methyl umbelliferyl-α-L-idopyranosiduronic…