Executive Director, Gene Therapy Clinical Development, PTC Therapeutics
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
TRNDS Speaker Spotlight
Why are you participating in TRNDS 2019?
SCHILLING: I am excited to hear the perspectives of other people working to bring gene therapies to patients, and hope that sharing what I’ve learned will be helpful in return.
What do you see as the biggest opportunities for accelerating therapeutic development in the 21st century?
SCHILLING: Close collaboration between industry and regulatory authorities will help to define a pathway with clear expectations.
How is the increasing use of modern technologies changing the field of clinical research?
SCHILLING: Wearable technologies are allowing for the collection of a more complete picture of a patient’s daily experience. With greater implementation in clinical trials and acceptance of wearable technologies by regulators, we in development will be able to better ensure that our compounds are addressing key patient needs.
Share with us some of the most exciting developments in your organization.
SCHILLING: PTC is rapidly growing, and we have several innovative products in development. We are filing an BLA for a gene therapy in AADC deficiency, and an IND for a gene therapy in Friedreich ataxia. We have compounds in earlier phases of development for Huntington’s disease, rare epilepsies, and other rare conditions.
What is a critical topic you’d like to see discussed at TRNDS 2019?
SCHILLING: Innovative ideas for the evaluation of gene therapies in rare diseases, and the implementation of wearables in clinical trials.
What challenges lie ahead in the rare disease space?
SCHILLING: The lack of natural history data available combined with the small numbers of patients available for clinical trials makes assessing effect size of a potential therapeutic very difficult.