Moving Medicine Forward

What happens behind the scenes to ensure new therapies are safe for patients? In this episode of Moving Medicine Forward , we are joined by John Kane, Vice President of Pharmacovigilance and Safety Services at CTI, to explore the critical role of drug safety throughout the clinical development process. John shares how safety teams collect and evaluate adverse event data, navigate complex global regulations, and help protect patients from clinical trials through post-market surveillance. He also discusses the growing impact of AI in pharmacovigilance, the continued growth of North Carolina's Research Triangle, and the rewarding career opportunities available in clinical research. 01:13 – Finding a Career in Clinical Research John shares how he transitioned from nursing into the clinical research industry. 02:14 – CTI's Growth in Raleigh Why North Carolina's Research Triangle continues to attract biotech companies and research talent. 04:55 – Understanding Pharmacovigilance What pharmacovigilance is and how safety teams collect, evaluate, and report adverse events throughout a drug's lifecycle. 07:33 – Protecting Patients Through Drug Development How CTI safety teams monitor risks, identify safety signals, and communicate important findings to regulators and investigators. 09:55 – Navigating Global Regulations The growing complexity of global safety reporting requirements and regulatory compliance. 11:51 – The Role of AI in Drug Safety How AI may improve data processing, signal detection, and scientific literature review. 13:53 – A COVID-19 Success Story John reflects on supporting a large COVID-19 trial and managing high volumes of safety data. 14:38 – Collaboration at CTI Why teamwork and collaboration are key to successful clinical trials and patient impact. 15:32 – Advice for Future Research Professionals Career opportunities in clinical research and how CTI helps new professionals enter the field.

What does it take to set a complex global clinical trial up for success? In this episode of Moving Medicine Forward , we are joined by Megan Kamm, Senior Vice President of Client Delivery, and Josh Schroeder, Vice President of Site & Field Operations, to discuss how strong planning, site partnerships, and cross-functional collaboration drive successful trial execution. From feasibility and study startup to patient centered decision-making, biometrics strategy, and global expansion, they share insights into the operational challenge —and opportunities—behind bringing innovative therapies to patients faster. 01:22 Megan Kamm and Josh Schroeder discuss their expanded leadership roles and growing collaboration across teams. 03:44 Why feasibility, country strategy, and study startup are critical to trial success. 05:07 The importance of site partnerships, patient experience, and reducing operational burden. 06:16 How project managers, CRAs, data managers, and site teams work together to execute complex trials. 07:01 Common startup challenges and strategies for keeping studies on track. 08:13 Managing patient-centered logistics in rare disease and cell & gene therapy programs. 11:15 Real-world examples of strong coordination improving enrollment and accelerating timelines. 13:19 The growing role of biometrics, statistics, and risk-based monitoring in study execution. 15:15 Scaling global teams through consistent training, leadership alignment, and operational excellence. 16:48 What differentiates CTI: collaboration, service, and a deep commitment to patients. 18:59 Looking ahead: AI, technology, startup optimization, and continued global growth. 21:05 Opportunities and challenges of expanding operations in China and the APAC region.

Asia Pacific has become one of the fastest-growing regions in clinical research, offering sponsors new opportunities to advance clinical development in an increasingly complex global environment. In this episode, Pavels Anetko, Director of Clinical Operations and Clinical Project Management at CTI, shares insights from across the region, including Australia's strengths in early phase studies and CTI's recent expansion into China and Japan. 01:24 Pavels’ background, career journey, and leadership role overseeing CTI's project management teams across Asia Pacific. 02:13 Why APAC is one of the fastest-growing regions for clinical development and the increasing influence of China. 02:41 CTI's growth strategy in Australia and the region's advantages for early-phase research, including the CTN regulatory pathway and R&D tax incentives. 04:06 The rise of healthy volunteer and Phase I studies in Australia. 04:27 Building CTI's presence in China and Japan and navigating more complex regulatory environments. 05:05 Staying aligned with regional regulatory requirements while maintaining global clinical trial standards. 05:40 How CTI helps sponsors select the right countries, sites, and patient populations across APAC. 06:20 Why local infrastructure, language expertise, and in-country teams are critical to trial success. 07:09 Challenges sponsors face when entering new APAC markets and how local support reduces risk. 07:36 The strategic value of including Japan and China in global studies, including access to patients and regulatory considerations. 08:58 How APAC can accelerate development timelines and strengthen regulatory submissions. 09:38 Key operational considerations for global studies, including logistics, specimen handling, and country-specific requirements. 10:08 Emerging opportunities across APAC, including growth in Southeast Asia and expanding clinical research markets. 10:54 Pavels shares career highlights, including gene therapy studies in Australia and supporting COVID-19 vaccine trials in Singapore. 12:44 Closing thoughts on the future of APAC and its growing influence on global clinical development.

Summer Farmen and Jeff Sturm share their personal cancer journeys and the mission behind ALK Positive, CTI’s Q3 Charity of the Quarter. ALK Positive is a patient-driven organization dedicated to improving outcomes for individuals living with ALK-positive cancer. Together, they discuss how they became advocates within a global patient community, the importance of genetic testing and second opinions, and the role of research initiatives and clinical trials in advancing treatment options and improving patient outcomes. 00:00 Introduction to ALK Positive and today's guests, Summer Farmen and Jeff Sturm. 01:14 Summer and Jeff share their personal cancer journeys and what led them to become advocates within the ALK Positive community. 02:33 The mission of ALK Positive: connecting patients, funding research, and ensuring no one faces the diagnosis alone. 03:49 Why second opinions matter and how specialized expertise can significantly impact treatment decisions. 06:19 Research initiatives supported by ALK Positive, including clinical trials, grants, and collaborations focused on new treatment approaches. 10:46 Challenging the perception of ALK-positive cancer as a “rare” disease and advocating for greater representation in research and clinical trials. 13:22 How targeted therapies have transformed outcomes and extended survival for many ALK-positive patients. 15:47 The realities of survivorship: managing side effects, quality of life, and the role of palliative care. 17:27 The importance of genetic testing, monitoring disease progression, and making informed treatment decisions. 21:15 Clinical trial participation: common barriers, improving access, and helping patients connect with opportunities. 26:16 Building community through support groups, education programs, Healing Arts initiatives, and patient connections. 28:46 Addressing the emotional and mental health challenges that often accompany a cancer diagnosis. 32:50 Looking ahead: emerging therapies, vaccine research, precision medicine, and the future of ALK-positive cancer treatment. 36:51 Resources available through ALK Positive and how patients and caregivers can get involved. 37:54 Closing thoughts on advocacy, community, and the patient voices helping shape the future of cancer research.

This week, we're revisiting a previous conversation with Jon Koch, CEO, and Brian Lawrence, CFO and President, as they share their perspective on what’s next for CTI as the organization enters a new phase of growth. They reflect on CTI’s legacy of partnership and execution while outlining how they plan to expand its impact across complex clinical trials. From scaling capabilities and investing in talent to strengthening operational discipline and embracing data-driven decision-making, Jon and Brian offer an inside look at how leadership alignment is shaping strategy. They also explore the evolving demands of the clinical research industry, from the need for predictability and site support to the practical application of technology and AI. At its core, this conversation is about balancing growth with purpose: delivering for sponsors, strengthening teams, and ultimately improving outcomes for patients. 00:36 CTI Rewind: Revisiting CTI's leadership transition and future vision 00:58 Expanding CTI's reach in complex and advanced therapy trials 02:23 Disciplined growth strategy and sustainable global expansion 04:38 CTI's legacy of care, credibility, and partnership 06:14 Driving performance through metrics and accountability 09:03 Leadership alignment and partnership in action 12:13 Talent development, infrastructure, and AI enablement 14:14 Positioning CTI for continued growth in complex trials 17:12 Key industry trends: predictability, sites, and technology 20:40 Sustaining culture and employee experience 22:08 Long-term vision: scale, impact, and global presence 25:15 Closing takeaways: quality, people, and execution

What does it take to design clinical trials that truly put patients first—especially when those patients are children? In this episode of Moving Medicine Forward , Dr. Radhika Peddinti, Medical Director at CTI, discusses her journey from pediatric hematology/oncology at the bedside to shaping clinical research at a global scale. Drawing on more than two decades of experience caring for children and their families, Dr. Pennant shares how those deeply human moments continue to influence how she approaches trial design today. From the emotional realities of pediatric oncology to the promise of gene therapies and targeted treatments, this conversation highlights the balance between scientific rigor and compassion—and why both are essential to advancing medicine. Together, they discuss how clinical trials can be designed to reflect real patient lives, the evolving landscape of rare disease research, and the leadership perspective required to make decisions that impact patients far beyond the clinic. If you're interested in the future of pediatric research, patient centered innovation, and what it truly means to move medicine forward, this episode is for you. 00:37 Meet Dr. Peddinti | Background in pediatric hematology/oncology and transition to CTI. 01:17 Finding Her Path | From training in India to a career shaped by patient and family experiences. 03:37 From Bedside to Research | Shifting focus to impact patients at a broader scale. 04:00 The Reality of Pediatric Oncology | Challenges, resilience, and redefining normal for children and families. 05:39 Expanding Impact Through Trials | Sickle cell research and the move into clinical development. 07:33 Designing with Patients in Mind | Bringing real-life patient needs into clinical trial design. 08:58 Balancing Science & Compassion | Why rigor and humanity are both critical to successful trials. 09:30 Leadership Lessons | Showing up with honesty, empathy, and adaptability. 10:47 The Future of Medicine | Breakthroughs in targeted therapies, gene therapy, and cure-focused research. 13:15 Advice for Clinicians | You don’t need to check every box—your experience matters.

In this episode, we’re joined by Rafael Miñana Prieto, Senior Manager of Operations for Laboratory Services at CTI, who oversees global biorepository operations across the U.S. and Europe. He walks through the full specimen lifecycle—from kit design and sample collection to storage, retrieval, and delivery—and explains why biological specimens are the foundation of clinical research. Rafael shares how consistent, end-to-end management, global standardization, and integrated laboratory services help protect data integrity, reduce risk, and improve outcomes across trials. He also highlights how small upstream decisions can impact downstream results, along with the operational challenges and evolving trends shaping modern biorepository services. 01:26 Rafael’s role and the end-to-end specimen lifecycle 02:15 Why sample integrity underpins clinical evidence and patient impact 03:26 Lifecycle complexity and global logistics challenges 04:16 Standardization, monitoring, and risk reduction strategies 05:32 Coordinating retrieval and global shipment 06:31 Upstream decisions (kit design, instructions) shaping outcomes 07:23 Integrated lab services and reduced fragmentation 07:59 Scaling challenges and pre-analytical gaps 09:24 Key trends: complexity, globalization, and data-driven operations 10:07 CAP accreditation and delivering consistent global standards

In this episode of Moving Medicine Forward , Dr. Robert Fisher—one of the pioneers of modern transplant surgery—explores the rapidly advancing field of xenotransplantation and why it may represent a true turning point in medicine. From CRISPR-driven genetic engineering to breakthroughs in immunosuppression and scalable organ production, Dr. Fisher breaks down how decades of research are converging to make cross-species organ transplants a clinical reality. The conversation also dives into what this means for patients, how it could transform transplant access, and the critical role of rigorous, collaborative clinical research in translating breakthrough science into real-world care. 01:23 Dr. Fisher’s path into transplant surgery 03:05 Why xenotransplantation is reaching a turning point (CRISPR + immunosuppression) 04:47 Advances enabling scalability, safety, and compatibility 08:32 Ethics, risks, and clearing misconceptions 10:27 Real-world patient impact and clinical considerations 13:10 Logistics, coordination, and clinical execution 15:49 New immunosuppression strategies driving success 17:44 Collaboration, ethics, and managing risk 20:49 Eliminating waitlists: expanding access to transplant 22:54 Milestones toward adoption and future outlook

What does a recovering clinical research market actually look like in 2026? In this episode, Joel White , Owner of Market Cap Consulting, and Ryan Gifford , Vice President, Global Laboratory Services, Business Development & Client Management at CTI, join us to unpack the state of the industry—from the rebound in biotech funding to the growing importance of execution, site relationships, and smarter trial design. We explore how increasing complexity is shaping partnerships, how labs and decentralized models are evolving, and what it will take to keep trials on track. Looking ahead, the conversation offers a practical perspective on what the next 12–18 months could bring. 01:00 Guest backgrounds: Joel White (Market Cap Consulting) and Ryan Gifford (CTI) 02:45 Market recovery and funding trends 05:15 Trial success: early collaboration and site relationships 08:15 Funding challenges and execution impacts 10:30 Reducing site burden through lab operations and logistics 14:15 Decentralized trials and patient experience 16:25 Regulatory shifts, AI, and growing data complexity 19:30 Future outlook: partnerships and global trial expansion 23:30 Key takeaways and rapid-fire insights

In this MMF Rewind, we’re bringing back an important conversation with Lynda Lee Smith—one that shines a light on the ongoing challenges transplant families face and the critical role of research in improving outcomes. Lynda Lee Smith, CEO of Enduring Hearts, is featured on the Moving Medicine Forward Podcast to discuss the mission and impact of the nation’s only nonprofit dedicated to funding pediatric heart transplant research. In this conversation, Lynda addresses the challenges transplant families face and how research is improving outcomes and quality of life. Listen now and consider donating to Enduring Hearts here. 00:55 The mission behind the only organization dedicated exclusively to pediatric heart transplant research. 01:19 How Lynda found Enduring Hearts and its mission-driven work. 02:45 The personal story that exposed a major research gap in pediatric heart transplantation. 03:50 Why heart transplants are lifesaving, but not a cure. 04:22 More on home blood tests and their role in reducing invasive biopsies and improving quality of life. 05:39 Why Enduring Hearts focuses on making one transplant last a lifetime. 07:06 How Enduring Hearts supports bold research ideas before larger funders step in. 09:40 The unique challenges teenagers face after transplant and how research helps. 12:05 Addressing the financial, emotional, and logistical burdens families face. 15:21 Programs that connect families navigating life after pediatric transplant. 15:49 Enduring Hearts’ innovative approach to engaging a new generation through gaming. 18:04 The move toward DNA informed, individualized treatment plans. 19:19 What real success looks like for pediatric heart transplant patients. 21:08 Stories of resilience, progress, and hope for the future.

In this episode, Jon Koch, CEO, and Brian Lawrence, CFO and President, share their perspective on what’s next for CTI as the organization enters a new phase of growth. They reflect on CTI’s legacy of partnership and execution while outlining how they plan to expand its impact across complex clinical trials. From scaling capabilities and investing in talent to strengthening operational discipline and embracing data driven decision-making, Jon and Brian offer an inside look at how leadership alignment is shaping strategy. They also explore the evolving demands of the clinical research industry, from the need for predictability and site support to the practical application of technology and AI. At its core, this conversation is about balancing growth with purpose: delivering for sponsors, strengthening teams, and ultimately improving outcomes for patients. 00:35 CTI leadership transition and future focus 01:23 Expanding reach in complex and advanced therapy trials 02:48 Disciplined growth strategy and global expansion 05:03 CTI’s legacy of care, credibility, and partnership 06:39 Driving performance through metrics and accountability 09:28 Leadership alignment and partnership in action 12:38 Talent development, infrastructure, and AI enablement 14:38 Positioning for growth in complex trials 17:37 Key industry trends: predictability, sites, and tech 21:05 Sustaining culture and employee experience 22:33 Long-term vision: scale, impact, and global presence 25:39 Closing takeaways: quality, people, and execution

What does effective medical leadership look like in today’s complex research environment? In this episode of Moving Medicine Forward , Dr. Sliman discusses his path from patient care to clinical research leadership. Drawing on decades of experience, Dr. Sliman shares lessons on staying patient‑centered, navigating regulatory realities, and leading teams within evolving healthcare systems—offering practical insights for physicians and researchers shaping the future of clinical development. 00:36 Dr. Sliman’s background spanning clinical care, research, and medical leadership. 01:10 Pop culture influences ( M A S H*, Star Trek ) and family roots in healthcare that sparked Dr. Sliman’s interest in becoming a physician. 03:42 Early research experiences and witnessing healthcare disparities in developing countries. 05:05 Why individual patient journeys still matter—even as studies scale and become more complex. 06:55 Leading within financial, regulatory, and operational constraints while staying patient‑centered. 08:32 Lessons on navigating rules, expanding boundaries, and achieving outcomes through collaboration. 10:36 Building infrastructure, sharing institutional knowledge, and avoiding repeated mistakes in clinical development. 11:53 Endpoint strategy, patient population selection, and negotiating within regulatory frameworks. 13:19 Understanding evolving regulatory expectations and operating effectively within them. 14:26 Why learning the system—regulatory, operational, and corporate—is essential. 16:02 Balancing passion for patient care with research experience and real‑world trial exposure. 19:27 Reflections on medicine, leadership, and the importance of staying connected to patient impact.

Dr. Robert Wagner, Senior Medical Director and Principal Investigator at CTI’s Clinical Research Center, shares more about clinical trial operations at research sites. Reflecting on nearly three decades in internal medicine and hospital‑based care, Dr. Wagner shares his path from patient care into clinical research, how that experience shapes his approach as an investigator, and why strong research sites are critical to trial quality and outcomes. The conversation also touches on the day‑to‑day realities of running trials and how site operations, technology, and patient‑centered practices continue to evolve. 01:00 His past experiences and current role as a Principal Investigator at CTI. 03:00 How clinical experience shaped his transition into research. 05:30 Daily responsibilities and the importance of sites. 08:30 Technology, recruitment, and patient access. 10:45 Guidance for physicians and patients; the future of clinical research.

Halle Raicevich, Research Associates Manager; Rick Renko, Project Manager, Regulatory & Scientific Affairs; and Alexa Kehoe, Senior Clinical Project Coordinator, are featured on this episode of the Moving Medicine Forward podcast to discuss CTI’s Research Associate (RA) Program. This pathway is designed to help emerging talent gain real‑world experience while exploring opportunities across the industry. Tune in to explore the value of mentorship and early exposure for professionals beginning their careers and learn how CTI’s people‑first culture supports growth at every stage. 00:51 Meet the guests: Halle Raicevich, Ricky Renko, and Alexa Kehoe 01:23 Overview of the RA program and its role as an entry point into clinical research 03:00 Why CTI invests in early‑career development and hands‑on learning 04:08 How cross‑department exposure builds confidence and strengthens collaboration 04:49 A typical day for a Research Associate 05:57 Skills gained through the RA role 07:06 The importance of flexibility and mentorship 08:20 How the RA program supports long‑term career growth across functions 09:18 Expanding the RA program internationally and lessons learned from global implementation 10:56 Developing future leaders through mentorship 12:23 Advice for those considering the RA program or a career in clinical research 14:27 Community involvement and CTI’s people‑first culture 16:37 Why the program is a powerful career foundation

Vincent Drouillon, Vice President of Global Central Lab Strategy and Growth at CTI, discusses how central laboratory services support clinical trials. He shares his path into laboratory medicine, explains why early collaboration and partnership are key to trial success, and outlines how global alignment and integrated lab solutions help sponsors move medicine forward. 00:36 Overview of Vincent’s role and background in laboratory medicine and clinical research. 01:18 Vincent’s career path and what drew him to central laboratory services. 02:37 The global impact of central labs and their role in developing new medicines. 03:44 Why global collaboration and diverse perspectives matter in central lab work. 04:28 Recognizing laboratory professionals and the importance of Lab Week. 05:26 Leading growth, scalability, and strategy for CTI’s central lab services. 06:55 How CTI’s quality-focused, flexible, and holistic approach differentiates its lab services. 07:54 Supporting complex and integrated clinical trials across specialized therapeutic areas. 09:29 Why lab planning and logistics are often underestimated in trial design. 11:19 The value of early engagement to reduce risk and prevent downstream delays. 14:39 Emerging capabilities shaping the future of laboratory services, including AI, analytics, and personalized medicine. 17:12 Closing reflections on partnership, patient impact, and the role of laboratories in moving medicine forward.

Shakirah Bradford, Senior Director of Vendor Management and Sourcing at CTI, discusses the key relationships that support clinical research. She also shares her unconventional path into sourcing and highlights why vendor management is uniquely complex within regulated environments. Since joining CTI, Shakirah has helped shift vendor relationships from transactional to strategic, equipping teams with the partnerships and tools needed to move medicine forward. 00:33 Overview of Shakirah’s role and cross industry background in vendor management. 01:25 How relationship building led Shakirah into sourcing and vendor management. 03:09 Selecting, contracting, and overseeing third-party vendors across the organization. 04:00 A housebuilding analogy to explain the role in everyday terms. 04:42 The impact of global regulations and quality requirements on vendor oversight. 05:31 How trust and long-term relationships improve execution and efficiency. 06:47 What defines true partnerships in clinical research. 07:52 Shakirah’s evolving approach to collaboration and stakeholder needs. 08:37 Why creativity, curiosity, listening, and asking questions matter. 09:32 Choosing an industry you’re passionate about and staying connected to patient impact. 11:17 Final reflections on passion and partnership.

Lynda Lee Smith, CEO of Enduring Hearts, is featured on the Moving Medicine Forward Podcast to discuss the mission and impact of the nation’s only nonprofit dedicated to funding pediatric heart transplant research. In this conversation, Lynda addresses the challenges transplant families face and how research is improving outcomes and quality of life. Listen now and consider donating to Enduring Hearts here. 00:00:55 The mission behind the only organization dedicated exclusively to pediatric heart transplant research. 01:19 How Lynda found Enduring Hearts and its mission-driven work. 02:45 The personal story that exposed a major research gap in pediatric heart transplantation. 03:50 Why heart transplants are lifesaving, but not a cure. 04:22 More on home blood tests and their role in reducing invasive biopsies and improving quality of life. 05:39 Why Enduring Hearts focuses on making one transplant last a lifetime. 07:06 How Enduring Hearts supports bold research ideas before larger funders step in. 09:40 The unique challenges teenagers face after transplant and how research helps. 12:05 Addressing the financial, emotional, and logistical burdens families face. 15:21 Programs that connect families navigating life after pediatric transplant. 15:49 Enduring Hearts’ innovative approach to engaging a new generation through gaming. 18:04 The move toward DNA informed, individualized treatment plans. 19:19 What real success looks like for pediatric heart transplant patients. 21:08 Stories of resilience, progress, and hope for the future.

What drives success at a clinical research site? Adam Roth , VP of Research Site Services, and Nick Finan , Director of Business Development and Client Management, share insights on site operations at CTI’s Clinical Research Center. They discuss feasibility, patient experience, trust based partnerships, and why operational realism is key to delivering high-quality clinical trials. Throughout the conversation, Adam and Nick also explain how strong collaboration among sites, sponsors, and CROs ultimately drives better outcomes for patients. 01:00 Adam Roth and Nick Finan share their roles supporting site operations and sponsor partnerships at CTI. 02:30 An overview of day-to-day site operations and resourcing throughout all phases of clinical trials. 04:00 How feasibility, therapeutic expertise, and operational readiness guide trial selection. 06:00 Why understanding patient and staff realities leads to better‑designed, better‑executed studies. 09:30 Building long‑term relationships through honest conversations about consistency and delivery. 11:00 How CTI preserves ethical boundaries while supporting productive collaboration. 13:00 Using site experience to improve CRA training and organizational best practices. 16:00 Why the strongest sponsor, CRO, and site relationships are built on shared goals. 20:45 Reflections on teamwork, trust, and moving medicine forward together.

Antibody drug conjugates (ADCs) are transforming cancer research — but how do they actually work, and what does it take to bring them safely to patients? In this episode of Moving Medicine Forward , we speak with scientist Lidia Blasco about the science and promise of antibody drug conjugates (ADCs) in cancer research. Lidia shares her journey from pharmacy and forensic analysis to oncology research, explaining in clear terms how ADCs work, why they’re transforming treatment, and what it means to develop them responsibly. The conversation also highlights the human side of clinical trials and the patients who make medical progress possible. 01:18 – Lidia Blasco’s background and path into ADC research 02:30 – Balancing PhD work with hands‑on industry lab experience 03:40 – Antibody drug conjugates explained simply 04:45 – Why ADCs are a promising cancer treatment approach 06:26 – The goal and impact of Lidia’s PhD research platform 07:30 – Improving dosing decisions and patient safety in clinical trials 08:06 – Emerging trends and innovations in ADC development 09:27 – Advice for those considering a career in oncology research 10:43 – Honoring the patients behind the research samples 12:53 – Closing reflections and final takeaways

Paul Ayoub, co‑founder of Rarity PBC, joins this episode of Moving Medicine Forward to take us behind the scenes of developing sustainable, patient‑first gene therapies for rare genetic diseases. Paul explains how the guidance of influential mentors, combined with his experience in cell and gene therapy, shaped who he is and ultimately guided Rarity’s mission. This episode explores ADA‑SCID, the realities of protective isolation for families, and the potential for a one‑time gene therapy to transform children’s lives, while highlighting why resilience drives innovation in rare disease care. Rarity PBC is a mission-driven public benefit corporation focused on developing accessible and affordable gene therapies for rare diseases. Learn more by visiting their website: Rarity PBC | gene therapy 1:00 Paul introduces Rarity PBC and its mission to deliver gene therapies for rare diseases. 3:00 Challenging the misconception that rare disease therapies can’t be sustainable. 4:00 The scientific promise of gene therapy and the personal motivation behind the work. 6:30 Why rare disease innovation can (and must) balance purpose and viability. 8:00 What ADA-SCID is, why it’s urgent, and how early intervention saves lives. 9:40 The isolation, vigilance, and emotional toll of living in a protective bubble. 10:45 A story that highlights what “normal life” truly means for families. 12:00 A one-time, autologous gene therapy that addresses the root genetic cause. 13:45 Why gene therapy represents a shift toward curative, value-based care. 14:00 How Rarity’s structure embeds patient-first decision-making. 17:00 Why expertise, continuity, and culture matter in gene therapy development. 18:20 Long-term outcomes, manufacturing scale-up, and the path to FDA approval.