New Science

Mapping the patient journey uncovers a wealth of insights into care, diagnosis and intervention. The onset of advanced analytics tools and sensors now augment the pharma industry’s understanding of the challenges of traversing health care systems. Layering AI into the mapping process not only has the power to provide insights into the current clinical pathway, it can be used to predict and intervene in clinical care.

The US FDA could approve as many new gene therapies in 2023 as it has in the previous five years. Development in the field has rapidly accelerated, but still faces hurdles.

CEO Scott Braunstein, who took leadership of Marinus in 2019 following clinical trial setbacks, believes the company is on a route to success with its ganaxolone program, targeting chronic use and hospital markets. 

Bad actors, lack of funding and skilled workforce shortages threaten to hinder the growth of cell and gene therapies. To unleash the transformative power of cell and gene therapies requires the concerted effort of the entire CGT community.

Infex CEO Peter Jackson discusses the state of antimicrobial resistance in the UK, and globally. He highlights the potential of a new “Netflix” style subscription model for antibiotic reimbursement, as well as future challenges for biotechs in the anti-infectives space. 

Despite decades of R&D, there are no disease modifying therapies on the market for Parkinson’s disease, and few efficacious products for non-motor symptoms. Clinical setbacks have been rife but breakthroughs in diagnostics and a better understanding of disease pathology brings hope for the future. In Vivo speaks to some of those responsible for bringing innovation to the field. 

2022 was a turbulent year for start-ups, especially in medtech. However, Rutger Zietsma has persisted, refocused and is ready for further success with his company, Manus Neurodynamica.

One of In Vivo’s 2023 Rising Leaders, Pheast Therapeutics’ chief development officer Amira Barkal, is also busy becoming a qualified physician. She is in the unusual position of leading an early-stage drug development program and being hands-on helping patients at the same time.

Anthony DeBoer, head of business development at Synaffix, joined the company in 2014 and has helped develop a platform technology business model in the antibody drug conjugate space, where Synaffix has become one of the leading technology providers enabling best-in-class therapeutic candidates.

Rachel Haurwitz, president and CEO of CRISPR-focused biotech Caribou, sat down with In Vivo to reflect on the thrill of the early days of CRISPR technology, Caribou’s second-generation approach, and the benefits of being a CEO with no prior corporate leadership experience.

Roche’s decision to discontinue a Phase III program in idiopathic pulmonary fibrosis was the latest of several recent late-stage clinical failures, emblematic of the difficulty in finding new approaches to treat the disease.

The antibody-drug conjugate field is beginning to reach its full potential and big pharma’s attention has been caught.

Pfizer and GSK are expected to gain approval and launch in time for this year’s flu season, but must educate public and physicians

The FDA Modernization Act 2.0 should make it easier for companies to gain regulatory authorization using non-traditional testing methods, like organs-on-a-chip. But biopharma will need to make the leap into technology to help build knowledge and data if the drug development world is to move away from animal testing entirely. 

Messenger RNA (mRNA) prophylactic vaccines have made major breakthroughs in 2020 and 2021 with both the Pfizer/BioNTech and Moderna COVID-19 vaccines gaining approval in more than 150 countries. These mRNA COVID vaccines have been developed much faster and with superior clinical profiles when compared to other more conventional modalities.

French-founded biotech Smart Immune has made significant headway in stimulating the thymus, a small gland with a huge immunological impact which rarely finds itself as a key clinical target. The company is now investigating the impact that two of its ProTcell products in patients with severe combined immunodeficiency and leukemia could have.