Economics

Good Biotechnology Governance to Combat Bad Blood: Using Cautionary Tales and Storytelling to Catalyze Start-Up Success

In January 2022, after months of proceedings and years of speculation, a jury found Theranos founder Elizabeth Holmes guilty of conspiracy to commit fraud and multiple charges of wire fraud (1). The trial of her former business partner Ramesh (also known as Sunny) Balwani began in March 2022. Theranos, a private medical device company in the healthcare sector, aimed to change the face of blood testing through benchtop in vitro diagnostics (IVDs) that were rapid, highly sensitive and specific, and…

Hurdles Ahead for Cell and Gene Therapy Makers

Significant growth of the cell and gene therapy (CGT) pipeline in recent years demonstrates the enormous potential of these modalities to treat or even cure otherwise intractable diseases. Several CGT products have been approved for clinical use over the past five years. More than 75 such products have come to the market around the world so far. They include chimeric antigen receptor (CAR) T-cell therapies that involve genetic engineering of patient cells ex vivo as well as in vivo gene therapies…

Bioprocessing Facilities in Asia Consider Domestic Alternatives to Western Suppliers

The global biopharmaceutical industry had been growing robustly even before the COVID-19 pandemic. According to the BioPlan Associates Top 1000 Biofacility Index and Biomanufacturers Database (1), bioprocessing capacity worldwide increased an average of 12% over the past decade. China has seen nearly double that rate. India’s bioprocessing segment also is showing strong growth. Because those regions represent 37% of the world’s population — and with rapidly growing middle-class economies, — demand for biologics there is outstripping that elsewhere. Historically, Western…

Collaboration Agreements: Critical Issues and Common Pitfalls

The number of collaboration arrangements for emerging technologies is increasing significantly, especially among companies developing COVID-19 diagnostics, vaccines, and therapeutics. Consequently, a massive amount of capital was invested in the biopharmaceutical industry in 2020. These deals range in value from the low millions to several billion dollars, representing significant risks and value for investors. Some recipients of that capital are seeking partners with whom to collaborate, sharing both the costs and risks associated with their activities and bringing innovative technologies…

Focusing on the Patient Journey Can Increase Access to Lifesaving Therapies

Cell and gene therapies (CGTs) are positioned currently as last-chance, “miracle” cures for patients who have severe illnesses. Such promises require innovation. Despite the cutting-edge science and significant investment that goes into CGT development, fundamental challenges remain, including patient access. The highly personalized nature of autologous-therapy development presents myriad logistical, financial, and manufacturing challenges to ensuring global access to treatment. Understanding a patient’s journey to treatment is vitally important to achieving that goal. Barriers to Cell and Gene Therapy Access…

Microbiome-Based Therapeutics: Negotiating Key Development Challenges

Microbiome-based therapeutics have evolved significantly in recent years, with several promising candidates advancing through the clinical pipeline. That progress is the result of growing evidence showing that targeting and manipulating the microbiome could improve human health and treat more than 25 conditions by restoring healthy bacterial populations (1). The human microbiome comprises a diverse community of microorganisms, helpful and harmful. It differs by person based on factors such as genetics, environmental influences, diet, and immune function. Microorganisms play a role…

The Next COVID Challenge: Building an Arsenal of Vaccines

For months, international partners have pressured the United States about intellectual property (IP) concerns and access to SARS-CoV-2 vaccines. As the country emerges from the worst of the pandemic, President Joe Biden now has an opportunity to shift his administration’s focus to realizing its longstanding promise of making the United States “the arsenal of vaccines for the world” (1). However, the country cannot accomplish that goal just by exporting mRNA vaccines and waiving IP protections. The current generation of vaccines…

eBook: Cell and Gene Therapies —
A 2021 Industry Update

The US Food and Drug Administration (FDA) reports that as of June 2021, 22 advanced therapy products have received regulatory approval in the United States. The first such product gained regulatory approval in 2010. Since then, hundreds of cell and gene therapies have advanced to clinical evaluation, but few products have reached commercial stages — and those that have done so have been hindered by manufacturing problems. In this eBook, writers from the BioProcess Insider and Project Farma analyze trends…

Mesenchymal Stromal Stem Cells: Next Steps and Considerations for CGMP Manufacturing

Massimo Dominici is scientific founder of Rigenerand srl, a joint venture between RanD (a biomedical company producing bioreactors for liver support and chemohyperthermic technology for cancer) and experts in cell and gene therapy at the University of Modena and Emilia Region in Italy. Rigenerand develops and manufactures medicinal products for cell-therapy applications (primarily for regenerative medicine and oncology) and three-dimensional (3D) bioreactors as an alternative to animal testing for preclinical investigations. The company also produces its own pipeline of cell…

Toward the Point of Care: Flexibility and Decentralization Are Key to Making Autologous Therapies More Readily Available

Part of the advanced therapy medicinal products (ATMPs) class of therapeutics, cell and gene therapies (CGTs) can be either autologous, using the patient’s own cells, or allogeneic, using master banked donor cells. Global biotechnology company Orgenesis focuses on autologous therapies, with processes and systems developed for closed and automated processing that have been validated for regulatory-compliant production at the point of care for patient treatment. This technology could help overcome the limitations of traditionally cost-prohibitive CGT manufacturing methods that do…