Moving Medicine Forward

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.

In honor of World Kidney Day, we’re rereleasing this timely and insightful episode of Moving Medicine Forward. In this conversation, we sit down with Dr. Helio Tedesco, a globally recognized transplant nephrologist from São Paulo, Brazil, who leads one of the world’s largest kidney transplant programs and is now part of the CTI team. Joined by Dr. Christopher Boshkos, Senior Medical Director at CTI, and clinical scientist Erika Aldag, the discussion explores the challenges and innovations behind managing more than 15,000 transplant patients—from telehealth expansion and donor matching to cutting-edge immunosuppressive strategies and the future of surveillance tools such as cell-free DNA and the iBox model. This episode offers a rare look into the science, systems, and human dedication driving transplant care forward.

00:35 Dr. Helio Tedesco (São Paulo), Dr. Christopher Boshkos, and Clinical Scientist Erika Aldag join to discuss global kidney transplant practices.

01:13 Dr. Tedesco shares his background, highlighting nearly 40 years in nephrology, performing over 1,000 kidney transplants annually, and a strong foundation in research.

03:17 Managing large transplant populations involves strategic follow-up care, decentralized nephrology networks, and an expanding role for telehealth.

06:30 How patients are matched to regimens, minimizing rejection and addressing regional challenges.

11:49 Dr. Tedesco explains their one-dose thymoglobulin approach and its benefits over traditional multi-dose methods.

18:34 New tools for monitoring graft health include donor-derived cell-free DNA, advanced biopsy techniques, and an examination of barriers to widespread adoption.

24:25 Discussion on the potential of the iBox model as a predictive tool for clinical trials and long-term graft survival.

28:15 Transplant science is headed toward improved long-term therapies, innovative biologics, and more patient-friendly immunosuppression options.

32:15 Successes and learnings from scaling telehealth to support a large patient base.

37:41 Appreciation for the collaboration between CTI and global transplant centers.

Christin Godale, Executive Director of LifeSciKY, and Savannah Doliboa, Chief Commercial Officer at CTI and LifeSciKY board member, explore how purpose built infrastructure can unlock the next generation of medical breakthroughs in our latest podcast episode.

Together, they discuss why infrastructure is often the missing link in drug development, how regional collaboration fuels innovation, and what success looks like when companies outgrow the incubator and scale locally. The conversation also shows the human side of ecosystem building by focusing on investing in people, nurturing talent, and turning scientific breakthroughs into real world impact.

LifeSciKY is a mission driven hub designed to lower barriers for startups, accelerate early research, and strengthen Northern Kentucky’s biotech ecosystem. Through affordable wet lab space, shared equipment, workforce development, STEM education, and community partnerships, it gives innovators the support they need to move discoveries from bench to patient.

00:30 An overview of LifeSciKY as a mission driven life sciences hub supporting early stage innovation.

01:45 Christin and Savannah share the vision behind LifeSciKY and how it benefits the region.

04:00 How the lack of wet lab space stalled regional growth and why infrastructure is essential to moving medicine forward.

06:45 Who LifeSciKY Is built for: startups, hybrid AI companies, and entrepreneurs looking to turn science into viable products.

08:25 What success looks like for biotech, including formation, fundraising, and scaling beyond the incubator.

10:15 How connecting academia, hospitals, industry, and government is building a regional life sciences ecosystem.

12:20 Affordable access, nonprofit mission, and hands on support sets LifeSciKY apart from other incubators.

14:20 How multiple years of state support and leadership brought LifeSciKY to its grand opening.

18:10 Workforce development and STEM outreach are building the next generation of life sciences talent.

22:00 The future of LifeSciKY and why Northern Kentucky could become a biotech hub.

23:10 Highlighting a LifeSciKY company developing more accessible CAR T therapies for children.

25:00 Ways founders, students, and community members can engage with LifeSciKY.

26:10 Reflections on momentum, mission, and future therapies.

28:05 In their closing thoughts, Christian and Savannah explore excellence and growth.

What happens when patients become true partners in healthcare innovation? In this episode of Moving Medicine Forward, we speak with Dr. Karin Hehenberger, physician, scientist, transplant recipient, and Founder & President of Lyfebulb, about how lived experience can reshape chronic disease care.

Diagnosed with type 1 diabetes as a teenager, Karin’s journey through medicine, biotech, and ultimately organ transplantation inspired Lyfebulb’s mission to empower patients through education, community, and innovation. We explore why patient voices must be included early in development, how patient entrepreneurs are driving real‑world solutions, and why reducing isolation is essential to better outcomes.

01:00 Welcome & Guest Introduction Meet Dr. Karin Hehenberger and the mission behind Lyfebulb.

02:10 Living With Chronic Illness Karin’s early diabetes diagnosis and how it shaped her path into medicine and research.

05:35 When Disease Progresses Anyway Facing kidney failure and vision loss despite careful disease management.

06:45 Transplant & New Perspective How kidney and pancreas transplants led to a renewed purpose.

07:55 Founding Lyfebulb Why patient education, connection, and innovation are central to empowerment.

10:45 Patients as Partners Moving beyond “patient‑centric” language to true collaboration.

12:20 Patient‑Driven Innovation Innovation challenges and real solutions created by patients for patients.

17:55 The Power of Community Reducing isolation to improve emotional health and daily disease management.

22:45 Industry & Research Collaboration Why patient insight must be integrated early—not as a checkbox.

27:15 Rethinking Chronic Disease A vision for managing chronic illness without constant mental burden.

31:15 Learn More How to get involved at LyfeBulb.com

In honor of Rare Disease Day, we sat down with Tammy Black, Chief Communications Officer at the Immune Deficiency Foundation (IDF), to discuss primary immunodeficiency (PI): a rare, lifelong condition that affects thousands of people and is frequently misdiagnosed. Tammy shares how living with a chronic illness shapes daily life and why education, advocacy, and strong community support are essential for patients and families.

Building on our ongoing conversations about rare diseases and patient access, this episode highlights the power of awareness and connection in improving outcomes for those living with PI. Tune in to learn more about how you can get involved and support IDF, CTI’s Q1 Charity of the Quarter.

01:00 Tammy Black explains the Immune Deficiency Foundation’s mission and primary immunodeficiency.

03:35 Why many patients go decades without answers and don’t appear ill despite serious health risks.

05:40 She connects PI experiences to pandemic‑era lessons and highlights how millions live in constant protection mode.

06:30 Barriers to adult diagnosis, lack of immune health conversations, and how IDF’s assessment tool helps patients advocate for themselves.

08:50 How IDF reduces isolation through support groups, peer matching, walk events, and national conferences.

10:30 Patient handbooks, webinars, school and clinician toolkits, and resources for newly diagnosed families.

13:55 Policy priorities including vaccines, healthcare access, and coverage challenges for immunoglobulin therapy.

15:40 How IDF partners with researchers and regulators to advance gene research and expand newborn screening for severe immune disorders.

18:10 Ways listeners can support IDF through donations, fundraising, volunteering, and plasma donation.

20:10 Tammy shares details about IDF’s documentary debuting publicly on YouTube for Rare Disease Day (Feb 28). 

In honor of the International Day of Women and Girls in Science, we sit down with Megan Kamm, Head of Global Clinical Project Management at CTI, and Dr. Amanda King, nurse practitioner, PhD, and clinical researcher in oncology and rare disease. Together, they explore their career paths, early scientific inspirations, the promise of personalized medicine, and how women are driving innovation across clinical research. Their conversation offers practical advice, powerful personal stories, and a look at breakthroughs shaping the future of health care.

01:12 — Megan’s Career Journey (Clinical Research → Global Leadership)
From research coordinator to leading CTI’s global clinical project managementfunction.

02:17 — Amanda’s Path (Clinician-Scientist in Oncology& Rare Disease)
A nurse practitioner and PhD with deep experience across pediatric critical care, oncology, and research.

03:07 — What Sparked Their Interest in ScienceEarly strengths in STEM, curiosity, and real-world exposure that shaped career direction.

04:53 — A Personal Story That Shaped a CareerAmanda shares how childhood experiences with cancer influenced her calling in healthcare and research.

05:49 — Turning Complex Science into Real-World TrialsMegan explains what it’s like to help move early concepts into tangible clinical studies with a path to patients.

07:12 — Why Rare Disease Research Hits DifferentAmanda describes the urgency—and reward—of trials where no standard of care exists.

07:38 — Where Innovation Is Headed: Personalized MedicineTargeted therapies, mutation-driven treatment decisions, and what that means for outcomes.

08:15 — The Promise of Cell & Gene TherapyHow technology is enabling potential one-time treatments and even cures—for previously untreatable diseases.

09:12 — Challenges Women Still Face in STEMBeing underestimated, navigating bias early in a career, and the role mentorship plays in resilience.

11:06 — Advice for Girls & Young Women Exploring STEMDefine success on your own terms, find the right environment, and learn what you truly value.

12:30 — Habits That Help: Mentorship + CuriosityShadow, ask questions, stay open to pivots, and keep learning as roles evolve.

14:12 — A Practical Takeaway: Don’t “Single-Track” Your SkillsetMegan on blending science with business skills to expand options and impact.

16:01 — Start Early: Intern, Shadow, ExploreAmanda’s recommendation to get real exposure early because STEM has more paths than most people realize.

In this episode of Moving Medicine Forward, we speak with CTI Founder & CEO Tim Schroeder and Managing Director of Latin America Jaqueline Aguiar about the region’s scientific strength, evolving regulatory landscape, exceptional patient retention, and growing role in rare disease and advanced therapies. They share real‑world examples of record‑speed approvals, high‑quality data, and the cultural dynamics that make LATAM a uniquely powerful environment for clinical trials. A must‑listen for anyone involved in global development or curious about the future of research in emerging markets.

01:22 Why LATAM is a strategic hub: scientific capability, cost efficiency, patient diversity, centralized healthcare, and increasingly fast regulatory timelines.

03:32 Tim on LATAM’s standout strengths — exceptional data quality, major specialty centers, and cost‑effective trial execution.

05:15 CTI’s expansion in the region: driven by rare disease needs, global sponsor demand, and Jaclyn’s leadership building a resilient LATAM infrastructure.

08:04 How LATAM supported global studies during COVID‑19and the ongoing complexities of logistics, sample transport, and country‑specific operations.

10:11 Significant regulatory acceleration across Brazil, Mexico, Colombia, Peru, and Chile — including <60‑day approvals forcomplex studies.

14:20 The role of CTI’s passionate teams and strong site relationships; unique investigator engagement in São Paulo.

18:38 Ethics committees’ central role in patient safety and informed consent; LATAM’s readiness for decentralized and hybrid trials.

22:14 Emerging local pharma capabilities and cultural factors influencing trial participation, including trust‑based doctor‑patient relationships.

25:15 LATAM as a global talent hub; future accelerators include digital harmonization, technology adoption, and better global awareness of the region’s capabilities.

In this episode of Moving Medicine Forward, Dr. Monica Shah, Chief Medical Officer at CTI and President-Elect of the American Heart Association's Greater Washington Region Board of Directors, reflects on why Wear Red Day holds personal significance to her.  Dr. Shah shares her path through cardiology and clinical research, the American Heart Association’s community impact across the region, and the organization’s priorities for advancing equitable care. She also shares her perspective on the future of cell and gene therapies and emphasizes the need for diverse representation in clinical trials.

01:08 Dr. Shah’s path through cardiology, research training at Duke, and career across academia, NIH, and industry.

03:38 Early involvement with the American Heart Association and the fellowship grant that launched her research career.

05:35 Responsibilities and priorities as President‑Elect of the AHA Greater Washington Region Board.

07:00 Leadership development and building a diverse pipeline of future AHA leaders.

08:10 Regional community impact: CPR education, school programs, nutrition initiatives, and policy advocacy.

10:07 Translating the AHA’s national mission into local, lifesaving change - including DC’s CPR Act.

11:32 Hypertension control and partnerships addressing nutrition security.

12:26 Barriers to cardiovascular health and how AHA programs support access and education.

13:22 COVID‑19’s long-term cardiovascular impact and AHA’s research and telehealth initiatives.

14:52 Advocating for equitable representation in clinical trials.

15:59 Emerging opportunities in cell and gene therapy and the importance of genetic testing.

17:34 How public–private partnerships accelerate cardiovascular innovation.

18:55 Advice for early-career clinicians and researchers, especially women and underrepresented groups.

20:00 Identifying and nurturing community and clinical champions.

21:52 Dr. Shah’s top priorities as incoming AHA president.

23:05 How listeners can get involved and closing reflections on advancing heart health.

In this episode of Moving Medicine Forward, we talk with Earnestine Walker and Ali Manson of ZERO Prostate Cancer about their mission to ensure every man—regardless of background—has access to early detection, support services, and quality care. They break down Blitz the Barriers, ZERO’s major initiative targeting disparities in the hardest‑hit communities, and share how programs like ZERO360 and caregiver resources are transforming patient experiences.

We also explore how policy advocacy, community partnerships, and patient voices are driving lasting change in prostate cancer outcomes nationwide. This is a powerful look at what equitable care truly requires—and how ZERO Prostate Cancer is helping move medicine forward.

00:00 – Welcome & Introductions

Meet Earnestine Walker and Ali Manson of ZERO Prostate Cancer and hear about their roles leading patient programs, education, advocacy, and policy.

01:00 – ZERO Prostate Cancer’s Mission

A look at how advocacy, education, awareness, and support guide every decision—and why “for all” is the organization’s guiding star.

02:00 – The Vision of Equitable Care

Discussion on what practical, equitable early detection and access look like in real communities.

03:30 – Introducing Blitz the Barriers

Why ZERO launched its most ambitious initiative ever—and the staggering disparities it aims to confront.

05:00 – The Cities Leading the Change

A breakdown of the phased rollout across 12 high‑impact cities, from Baltimore to Appalachia.

06:00 – Supporting Patients & Families

How programs like ZERO360 and the Caregiver Retreats provide emotional, financial, and navigation support.

08:00 – Navigating Financial Barriers

The role of patient navigation and how ZERO360 has delivered millions in financial assistance.

09:30 – Advocacy in Action ZERO’s long history on Capitol Hill and recent policy pushes to expand access to free prostate cancer screening.

11:00 – Empowering Patient Voices

Why patient stories and lived experiences are essential to driving legislative change.

13:00 – Closing the Gap for Black Men & Veterans

How ZERO Prostate Cancer is addressing the needs of the communities most disproportionately impacted.

15:00 – Measuring Impact & Expanding Reach

What ZERO has achieved so far—and how new data strategies will track long‑term change.

17:00 – The Power of Partnerships

From national organizations to local community groups, how collaboration amplifies ZERO’s equity‑driven mission.

19:00 – What’s Next for ZERO Prostate Cancer

A look at future goals, including the life-saving vision behind Blitz the Barriers through 2025.

In this episode, host Jeremy Schrand welcomes Tim Schroeder, Founder & CEO of CTI, Jaqueline Aguiar, Managing Director of APAC/LATAM, and Dr. Ludwig Baumann, Global Regulatory Strategist, for an in-depth conversation about the Asia Pacific region’s growing impact on clinical research. Together, they explore how population diversity, regulatory innovation, and advanced therapies are shaping the future of clinical trials. The discussion highlights real-world examples, regional strategies, and the evolving role of technology and local expertise in bringing new therapies to patients worldwide.

01:33 Welcome to the guests and setting the stage for the discussion.

01:50 Key drivers behind Asia Pacific’s growth: globalization of research, population scale, rare disease focus, and the need for global market access.

03:04 Panelists discuss the region’s diversity, infrastructure maturation, and regulatory harmonization efforts (ASEAN guidelines, mutual recognition).

05:10 Addressing past concerns about research qualityin Asia Pacific and how they’ve been resolved.

06:15 CTI’s strategic expansion into Asia Pacific:doubling of work, global footprint, and real-world examples from COVID-19 vaccine trials in Singapore and Vietnam.

08:17 Operational hurdles: regulatory fragmentation, cultural and language diversity, and government investment in science and digital health.

10:10 Managing regulatory variability: importance of local expertise, adapting to evolving guidelines, and leveraging relationships with regulators.

12:10 Harmonization and efficiency: Singapore’s reliance model, Australia’s streamlined processes, and their influence as regional role models.

14:26 Decentralized and virtual trials post-COVID: remote monitoring, reasons for choosing Australia (speed, tax credits) vs. China (population, cost).

16:07 Executing complex, multi-country trials: pivotal registration studies, remote monitoring, and the importance of experienced investigators.

18:05 Readiness for advanced therapies: academic medical centers’ preparedness, regulatory interest in cell and gene therapy, and the need for in-depth preparation for combination products.

20:27 Regulatory flexibility: pre-meetings with authorities, addressing language barriers, and successful integration of Asia Pacific into global trials.

22:01 Talent acquisition and retention: strategies for building consistent, high-quality teams across diverse countries.

23:53 Patient recruitment: cultural nuances and ensuring data quality and patient retention.

26:09 Looking ahead: the impact of AI, harmonization, and infrastructure development on accelerating clinical trial execution in Asia Pacific.

28:13 Recent success story: FDA approval of a rare disease drug with global patient participation, highlighting Asia Pacific’s role.

29:05 Closing thoughts: Asia Pacific’s leadership in innovation, regulatory adaptation, and inclusion of rare patient populations.