PharmaTalkRadio

This podcast dives into the history of creating a gene therapeutic for Duchenne Muscular Dystrophy. Dr Richard Jude Samulski has been studying gene therapy and the use of the AAV vector for decades. Recently, preliminary results from Pfizer were published on a small gene therapy trial for Duchenne Muscular Dystrophy with promising results, and Pfizer is scheduling a larger trial for later this year.

That gene therapy originated from Dr Samulski work and from AskBio, and uses an adeno-associated virus to carry a healthy copy of the dystrophin gene; the virus was injected into boys with DMD, broke into their muscle cells, and replaced their non-working gene.

Dr Samulski sat down with PharmaTalk's Danny McCarthy to discuss the process of creating an AAV vector to carry the gene therapeutic, the mechanisms of the therapy in DMD and what the future of the gene therapy landscape might look like. 

Guest:

R. Jude Samulski, PhD, is President, Chief Scientific Officer and Co-Founder of Asklepios BioPharmaceutical and Professor of Pharmacology in the UNC School of Medicine. Dr Samulski has worked with AAV for 40 years, and for 25 years, he was director of the University of North Carolina Gene Therapy Center.

Asklepios Bio's Chief Medical Officer, Martin Childers, DO, PhD, will be speaking at the Chief Medical Officer 360˚ summit, October 26-27, 2020, virtually.

This podcast tells the story of how a community-based COVID-19 research study was formed from a partnership between Oracle Health Sciences, Javara Research and Wake Forest Baptist Health, with the end goal of using regional infection patterns to understand the virus and develop strategies to contain it. 

Kathy Vandebelt of Oracle sat down to talk about the partnership enhanced Oracle's Therapeutic Learning System, which was set up to track how physicians were treating individuals infected with the virus and making the information available for others to learn from, for a real-world surveillance study. The collaboration resulted in the addition of a new component for the platform, the Patient Monitoring System, which aided in collecting daily information across a large population of participants and offering them the potential to participate in regular testing. That technology is being used for multiple studies on local, regional, national and now global levels. 

In the conversation, Ms Vandebelt also discusses the unexpected collaborations that emerged across the research spectrum as a result of the pandemic, how the Oracle team adapted their technology to keep pace with an evolving on-the-ground situation, and how clinical operations might benefit from adopting more decentralized measures, ultimately giving patients more choices. 

Guest:

Kathy Vandebelt is Global Head of Clinical Innovation for Oracle Health Sciences, where she uses the power of technology to introduce new experiences and expectations to make the clinical research ecosystem better for patients, healthcare providers, sponsors and regulators.

For more information about Oracle's COVID-19 response, visit Oracle.com. For more information about PharmaTalk Radio podcasts, go to theconferenceforum.org.

The goal of this podcast is to give you a brief overview on what you can expect from the 10th annual DPHARM conference in under 5 minutes.

DPHARM takes place on September 22-23 and is focused on clinical innovation and how to accelerate the translation of innovation for patients and physicians. DPHARM is the annual gathering where R&D reports on innovation that has advanced clinical trials in the last 12 months. Executive Director, Valerie Bowling shares the highlights on reporting from the trenches, modernizing clinical trial keynotes, plenary speakers, track choices and virtual interactivity.  

DPHARM: Disruptive Innovations to Advance Clinical Trials, September 22-23, Virtual EST. Mobile in Clinical Trials takes place the day before, September 21. For more information, visit theconferenceforum.org

PharmaTalk Radio’s Kate Woda welcomes guest, Erin Newburn, Director and Field Applications Scientist at Personalis on the topic of IO Biomarker Discovery and Personalized Cancer Therapy and how Personalis is enabling this. 

Guest Bio:

Erin Newburn, PhD joined Personalis as a Field Applications Scientist in 2013 with over 12 years of research experience in the areas of molecular biology, genetics, and biotechnology. As the Director of the Field Applications Scientist team, Dr Newburn’s group has the responsibility of providing both pre- and post-sale scientific and technical support for the Personalis ImmunoID NeXT Cancer Portfolio.

Dr Newburn completed her postdoctoral training at the National Institute of Mental Health (NIMH) investigating candidate susceptibility genes for major psychiatric illnesses. Dr Newburn received her PhD from the Ohio State University in Integrated Biomedical Science as a Presidential Fellow.

Information about Personalis, visit www.Personalis.com 

Information about upcoming conferences and podcasts on medicine development, visit theconferenceforum.org.

As the country goes through the phases of reopening, what does that mean for the patient and site experience in clinical trials? PharmaTalkRadio’s Valerie Bowling discusses the future of clinical trials with Dr Ray Dorsey, Matt Kibby and Craig Lipset. Together they address lessons learned from the rapid adoption of technologies to allow for more decentralized trials and their impact on study participation design moving forward, what sponsors can do to set themselves up for success, and the possibility of hybrid trials becoming the new normal for the near future. 

Guests: 

Ray Dorsey, MD, is the David M. Levy Professor of Neurology and Director of the Center for Health & Technology at the University of Rochester.

Matt Kibby is President, Principal of BBK Worldwide

Craig Lipset is the founder of Clinical Innovation Partners and the former Head of Clinical Innovation and Venture Partner at Pfizer

You can find all three of our guests speaking at either the upcoming Mobile in Clinical Trials conference or the 10th annual DPHARM conference virtually over September 21st-23rd. For more information about these conferences and other podcasts, visit theconferenceforum.org. 

For more information about BBK, visit BBKworldwide.com

Dr Dorsey's book on Ending Parkinson's Disease can be found on Amazon.

You can keep up with Craig Lipset at twitter.com/craiglipset

A special thanks to the team at BBK for their support in presenting this podcast.

In this podcast, Mark Scherzer, producer of Mobile in Clinical Trials gives an overview of what to expect at the 2020 virtual conference. The 7th annual meeting is co-chaired by Bayer's Michelle Shogren and Healthmode's Dan Karlin and reports on the latest progress in applying mobile-digital tools and patient-facing technologies for flexibility while connecting and empowering patients. It will feature collaborative, experience-based case studies to help R&D move faster to provide more choices for patients and investigators while obtaining better outcomes in drug development.

In this podcast, first recorded at the 2019 DPharm: Disruptive Innovations conference, Takeda's Dr Andrew Plump and Dr Jessica Scott discuss how they have developed KPIs that support innovation for patient-engagement.

Dr Plump leads Takeda's global R&D organization, where he provides strategic direction and oversights. He specializes in bringing and unwavering focus on patients and a deep commitment to innovation and positive change in the healthcare industry. 

Speakers:

Andrew Plump, MD, PhD, President, Research & Development, Takeda Pharmaceuticals

Jessica Scott, MD, JD, Head of R&D Patient Engagement, Takeda Pharmaceuticals

The 10th annual DPharm: Disruptive Innovations event is scheduled to take place on September 22-23, 2020. Learn more here.

In this podcast, first presented at the 9th annual DPharm: Disruptive Innovations conference, Starbucks' Robert Mercer sits down with Eli Lilly's Joe Kim to discuss the end-user experience in mobile technology. This out-of-industry example covers how they approach innovation and in particular how they designed their mobile experience with the customer in mind. More specifically:

Barriers and executive buy-inRolling out the mobile experienceBalancing the scale of new solutions across stores around the globe with the need to maintain existing operations and deliveryHow Starbucks engages with the customers to learn and develop innovative new solutions

Speakers:

Robert Mercer, Design Director, Global Digital products, Starbucks

Joseph Kim, MBA, Sr. Advisor, Clinical Operations and Digital Registry, Digital Health Office, Translational Technology & Innovation, Eli Lilly & Co.

The 10th annual DPharm: Disruptive Innovations event is scheduled to take place on September 22-23, 2020. Learn more here.

In this podcast, first presented at the 9th annual DPharm: Disruptive Innovations conference, Healthgrades' founder Kerry Hicks sits down with Craig Lipset for a keynote discussion on transparency and the future of patient expectations.

"Everyone likes progress and no one likes change." - Kerry Hicks

Kerry Hicks is the founder of Healthgrades, which puts the power of transparency into millions of patients' hands on provider visit experiences. The discussion begins with his vision for Healthgrades, what it was like to build it, early fears, and unintended consequences. Also covering: the intersection of patient experiences, technology, and clinical research as well as Kerry's insights on why you must consider transparency with everything you build moving forward.

Speakers:

Kerry Hicks, MBA, Founder, Healthgrades

Craig Lipset, MBA, former Head of Clinical Innvation, Pfizer

The 10th annual DPharm: Disruptive Innovation is scheduled to take place on September 22-23, 2020. Learn more here.

One of the goals of the Human Genome Project was to improve therapeutic options for those born with serious inherited diseases. Today gene therapy is beginning to realize this promise, with the ever-increasing level of clinical investigation in the space, and the first products coming to licensing in the last few years.

In this podcast, first recorded at the 2019 PODD: Partnership Opportunities in Drug Delivery conference, Dr Katherine High, Spark Therapeutics, reviews the current state of gene therapy, including the development of therapies for diseases that were previously untreatable, and, for diseases where therapeutics already existed, the development of therapies that greatly lessen the burden of treatment for the patient and the family. Hurdles in clinical development of gene therapy will be discussed. Examples will be drawn from already approved products and from investigational agents in late stage development.

To learn more about the 10th annual PODD: Partnership Opportunities in Drug Delivery conference please visit www.theconferenceforum.org