Q1. Tell me about yourself?

At ASC Therapeutics, I am responsible as Chief Medical Officer for all activities related to clinical development and regulatory submissions. We are conducting a phase I/II clinical trial, IND-cleared by the FDA, for our second-generation gene therapy for hemophilia A. We are about to initiate a phase II trial for allogeneic cell therapy, also IND-cleared, in graft-versus-host disease in hematologic malignancies. I also ensure that all our functional activities are coordinated with manufacturing, supply chain, preclinical, R&D and leadership.

My background is over 30 years in the biopharmaceutical industry working with large (AbbVie), medium-size and biotechs, focusing on biologics, cell and gene therapies, including CRISPR/Cas9 gene editing. Previously I worked in clinical and academic settings, leading to a tenured professorship in immunology.

Q2. What are the regulatory considerations and compliance requirements that organizations need to be aware of when outsourcing clinical trials in the USA?

Let me focus on my most recent experience when outsourcing clinical trials for gene and cell therapies. These therapeutic modalities require a specialized mindset by CROs, especially related to the need to focus on rare diseases. The FDA has been very gracious providing the industry with an increased flexibility regarding regulatory requirements while expanding their capabilities and providing numerous guidance documents. What this means is that although the compliance requirements are very demanding, the agency is flexible and open to discussion. This approach facilitates an open forum where different companies can share experiences and recommendations useful across the industry.

Q3. Can you share any success stories where the outsourcing of clinical trials in the USA led to significant advancements or breakthroughs in healthcare and medical research?

Focusing on the gene therapy space, we recently had two major breakthroughs in hemophilia A and B. For hemophilia A, BioMarin’s Roctavian and for hemophilia B, CSL Behring’s Hemgenix, have received marketing authorization in the US and Europe. This is an endorsement to the decades of basic and clinical research, as well as clinical development by many of my colleagues. Over the past years I have been supporting the phenomenal successes achieved by Avexis (Zolgensma, acquired by Novartis), and UniQure (Hemgenix, licensed by CSL Behring). These companies achieved breakthroughs that are paving the way for our second-generation gene therapy for hemophilia A.

Q4. How do you foster effective collaboration and communication among different parties involved in an outsourcing clinical trial?

The most critical driver to achieve effective teamwork for all stakeholders, again focusing on the extraordinarily complex gene therapies, is to have every individual displaying a positive attitude in their day-to-day activities. This means listening, trusting others, and focusing on accountability and commitment to the individual and team’s goals.