BioTalk Unzipped

Rob Dodge says AI has made him an expert Python programmer, even though he never took a class in it. But does having new capabilities change what it means to be a scientist?

Dr. Chad Briscoe sits down with Dr. Robert “Rob” Dodge at the AAPS National Biotechnology Conference in San Diego to discuss how artificial intelligence and machine learning are changing pharmaceutical research.

Thank you to our sponsors

TruTechnologies provides real-time clinical trial oversight, helping research teams improve protocol adherence and protect biospecimen integrity.

https://trutechnologies.ai/

Leucentra: Inspired by science, empowered by IT.

https://leucentra.com/

About the conversation

Rob remembers when spreadsheets changed how scientists calculated and plotted their results. Today, AI helps him write code and investigate scientific data with less reliance on specialist programming support.

He and Chad discuss where these tools are useful, where they fall short, and what scientists still need to bring to the work. Their conversation covers assay development, access to clinical data, scientific writing, and the challenges of using AI in regulated bioanalysis.

Rob also shares his predictions for how machine learning could change established modeling approaches, including quantitative systems pharmacology and design of experiments.

Throughout the discussion, he returns to a familiar principle: scientists still need to develop hypotheses, test them, and question their results.

In this episode

• How scientists are using AI to write Python programs
• What advances in mass spectrometry mean for complex drug modalities
• Machine learning for assay improvement and clinical data analysis
• Why data quality and laboratory experience matter
• Validation and quality assurance challenges in regulated research
• Using AI to draft and review scientific documents
• How independent consultants and smaller teams can use AI as a resource
• Rob’s predictions for the next year in pharmaceutical science

About Dr. Rob Dodge

Robert “Rob” Dodge, PhD, is an independent bioanalytical consultant focused on artificial intelligence and machine learning in regulated bioanalysis.

His more than 30 years in pharmaceutical science include biologics manufacturing, bioanalytical testing, and leadership roles at Bristol-Myers Squibb and Novartis. He co-founded Princeton Bio Lab and previously served as Director of Immunochemistry and Cell Biology at Pharmanet Development Group.

Connect with Rob:

https://www.linkedin.com/in/robert-dodge-phd/

About BioTalk Unzipped

Hosted by Gregory Austin and Dr. Chad Briscoe, BioTalk Unzipped uncovers the stories and science behind medical progress through conversations with scientists and leaders in biotechnology and drug development.

Find more episodes:

https://www.biotalkunzipped.com/

Follow BioTalk Unzipped on your podcast app to hear new episodes as they’re released. If this conversation brings a colleague to mind, send it their way.

Clinical trials often collect far more blood than an assay actually needs. Dr. Joleen White asks a more disciplined question: What information is required to make a sound decision, and how little sample can be collected without compromising that decision?

In this episode of BioTalk Unzipped, recorded at the 2026 AAPS National Biotechnology Conference in San Diego, Dr. Chad Briscoe sits down with Joleen to explore patient-centric sampling, dried blood microsampling, global bioanalytical capacity, and the values behind better drug development.

Joleen traces her path from a childhood friendship with someone living with cystic fibrosis to a career spanning BioMarin, BMS, Biogen, EMD Serono, the Gates Medical Research Institute, and BioData Solutions. She explains why bioanalysis became her way to help patients, why she eventually moved beyond the bench, and how global health work changed the way she thinks about laboratory infrastructure and clinical-trial design.

The conversation then turns practical. Joleen defines patient-centric sampling, describes the Mitra dried blood collection device, and discusses a first-in-human RSV monoclonal antibody study in which paired dried-blood and serum samples led to aligned PK conclusions. She and Chad examine regulatory acceptance, remote collection, pediatric sampling, local laboratory capacity in African countries, scientific inclusion, career choices, AI, mass-spectrometry sensitivity, and whether long-standing sample-volume and stability expectations still serve patients.

TruTechnologies provides live clinical trial execution and oversight through point-of-care data capture. Learn more: https://trutechnologies.ai/

Leucentra provides specialized IT consulting and services for life sciences and healthcare. Learn more: https://leucentra.com/

• How a personal connection to cystic fibrosis shaped Joleen's patient-first career

• Why bioanalysis connects analytical rigor to decisions about drug safety, exposure, and patient benefit

• What patient-centric sampling means beyond simply collecting less blood

• How dried blood microsampling can support PK, immunogenicity, and viral-neutralization assessments

• What Joleen's team learned by comparing dried blood with serum in a first-in-human study

• Why smaller samples and remote collection can reduce burdens on patients, caregivers, sites, and enrollment

• Why sustainable global health research depends on locally led scientific and laboratory capacity

• How legacy automation can slow innovation and create opportunities for newer laboratory networks

• Why inclusion and access expand both the scientific workforce and the quality of science

• How personal values can guide career decisions more effectively than a rigid five-year plan

• Why advances in mass spectrometry should prompt a fresh look at sample volumes and stability expectations

Dr. Joleen White is a Senior Bioanalytical Consultant at BioData Solutions with more than 20 years of experience across regulated bioanalysis, ligand-binding assays, immunogenicity, cell-based assays, neutralizing-antibody assays, and data science.

Connect with Joleen: https://www.linkedin.com/in/joleenwhite/

BioData Solutions: https://www.bdatasolutions.com/

Dr. Chad Briscoe is the co-host of BioTalk Unzipped and conducts this onsite conversation from AAPS NBC.

Connect with Chad: https://www.linkedin.com/in/chadbriscoe/

Open-access RSM01 dried blood microsampling study:

https://pubmed.ncbi.nlm.nih.gov/39696004/

Patient Centric Sampling Interest Group:

https://www.pcsig.org/

Website and all episodes: https://www.biotalkunzipped.com/

YouTube: https://www.youtube.com/@BioTalkUnzipped

BioTalk Unzipped unzips, unlocks, and uncovers the real stories behind medical progress.

Why is gene therapy still so difficult to manufacture at scale after decades of progress?

Phillip Ramsey, Chief Technical Officer at Sangamo Therapeutics, joins BioTalk Unzipped for a candid conversation about one of the central challenges facing advanced therapies: turning extraordinary science into reproducible, scalable medicines.

Leucentra - https://leucentra.com/

Inspired by Science. Empowered by IT. Leucentra provides specialized IT consulting and services for life sciences and healthcare, helping organizations use technology more effectively to support innovation.

TruTechnologies - https://trutechnologies.ai/

Clinical Trial Execution, Live. TruTechnologies provides live clinical trial execution technology that gives sponsors greater visibility into protocol activities, samples, and study data as they happen.

Recorded onsite at the AAPS National Biotechnology Conference in Boston, Phillip explains why gene therapy manufacturing has not yet reached the maturity of monoclonal antibody production.

As technologies have evolved across retroviral vectors, lentiviral vectors, adenovirus, AAV, lipid nanoparticles, and other advanced modalities, development teams have repeatedly been required to rethink fundamental manufacturing and analytical questions.

Phillip discusses why analytics are critical, why experience across multiple manufacturing lots and products matters, and why there simply is no shortcut to developing the scientific knowledge necessary to manufacture advanced therapies reliably.

He also reflects on the uncertainty of biotechnology.

Early in his career, Phillip was walking down a hallway when his CFO asked if he had cashed his paycheck.

When he said no, she told him not to.

It's a remarkable story that captures something rarely discussed about biotechnology: behind transformative science are people willing to operate through considerable uncertainty while trying to build something that has never existed before.

• Viral vector manufacturing and scale-up

• Why analytics are essential to gene therapy development

• AAV manufacturing challenges

• Lentiviral vectors and other evolving delivery technologies

• Lipid nanoparticles and changes in advanced therapy manufacturing

• Why gene therapy cannot simply adopt the established monoclonal antibody manufacturing playbook

• Building process knowledge across lots and products

• Career resilience in biotechnology

• Progress across AAV, CAR-T, and other advanced therapies

• Gene therapy affordability

• The importance of therapeutic durability

• Outcomes-based payment models for advanced therapies

Phillip's message for scientists and industry leaders is straightforward: don't assume the manufacturing problem has already been solved simply because a technology has existed for decades.

The field is still evolving, and some knowledge cannot be shortcut.

Phillip Ramsey is Chief Technical Officer at Sangamo Therapeutics and brings more than 35 years of experience across biotechnology and pharmaceutical manufacturing, technical development, operations, quality, and advanced therapies.

Connect with Phillip Ramsey:

https://www.linkedin.com/in/phillip-ramsey-a2074711/

Sangamo Therapeutics:

https://www.sangamo.com/

Gregory Austin on LinkedIn:

https://www.linkedin.com/in/gregoryaustin1/

Dr. Chad Briscoe on LinkedIn:

https://www.linkedin.com/in/chadbriscoe/

BioTalk Unzipped:

https://www.biotalkunzipped.com/

BioTalk Unzipped unzips, unlocks, and uncovers the stories behind medical progress through conversations with scientists, executives, entrepreneurs, and innovators advancing biotechnology, pharmaceuticals, and medical technology.

Recorded onsite at the AAPS National Biotechnology Conference in Boston.

Special thanks to AAPS and Rebecca Stauffer for supporting the BioTalk Unzipped interview series.

A mother’s search for answers to her daughter’s relentless brain tumor led her into natural killer cell biology, cancer immunotherapy, and biotech entrepreneurship.

Sponsored by Leucentra, https://leucentra.com/

Inspired by science, empowered by IT. Leucentra helps life science and healthcare organizations evaluate, implement, and get more value from technology that supports innovation.

Tracy Ryan, co-founder of NKore BioTherapeutics and former chief communications officer, joins Gregory Austin and Dr. Chad Briscoe to explain how her daughter Sophie’s pediatric brain tumor transformed her from a parent searching for options into a cancer advocate, research fundraiser, and biotechnology founder.

The conversation examines the scientific reasoning that moved Tracy from medical cannabis advocacy toward NK-cell immunotherapy. She describes an immune-system finding involving Sophie’s natural killer cells, the work of the late UCLA scientist Dr. Anahid Jewett, and the development of an experimental allogeneic NK-cell platform designed to enhance immune activity without genetic engineering.

The episode also explores the practical realities of translating an early scientific hypothesis into a therapeutic program, including donor selection, cell persistence, manufacturing, treatment outside the United States, regulatory requirements, clinical evidence, and the difficulty of raising capital for an emerging cell-therapy company.

At the center of the discussion is a difficult question: how do scientists, founders, regulators, and families preserve urgency when patients need better options, while still requiring the controlled evidence needed to determine whether a therapy is safe and effective?

What you will learn:

• How Sophie’s pediatric brain tumor changed the direction of Tracy Ryan’s life

• Why natural killer cells are important in immune surveillance and cancer biology

• What differentiates allogeneic NK-cell approaches from CAR-T and CAR-NK therapies

• How NKore’s experimental platform was designed to activate donor-derived NK cells

• Why persistence, donor biology, manufacturing, and lymphodepletion matter in cell therapy

• Why early human observations must remain distinct from controlled clinical-trial evidence

• How regulatory pathways and capital constraints shape whether promising science reaches patients

• What parents facing pediatric cancer need from the scientific and medical community

About Tracy Ryan:

Tracy Ryan is a pediatric cancer advocate, entrepreneur, public speaker, and biotechnology co-founder. She formerly served as chief communications officer of NKore BioTherapeutics.

Her daughter Sophie was diagnosed with a brain tumor during infancy, leading Tracy and her family into more than a decade of advocacy, research support, fundraising, and therapeutic exploration. Tracy and her husband also established Saving Sophie to support pediatric cancer research and help families pursue care. Their family’s experience with medical cannabis was featured in the documentary Weed the People.

Connect with Tracy Ryan:

https://www.linkedin.com/in/tracyryan23/

Connect with Gregory Austin:

https://www.linkedin.com/in/gregoryaustin1/

Connect with Dr. Chad Briscoe:

https://www.linkedin.com/in/chadbriscoe/

Watch more BioTalk Unzipped episodes:

https://www.youtube.com/@BioTalkUnzipped/videos

Visit BioTalk Unzipped:

https://www.biotalkunzipped.com

Learn more about NKore BioTherapeutics:

https://www.nkore.com

Support Saving Sophie:

https://www.savingsophie.org

This episode is brought to you by founding sponsor Lucentra. Lucentra provides information technology consulting for life science and healthcare organizations, helping teams modernize infrastructure, strengthen security, and build scalable technology environments that support scientific innovation.

Subscribe to BioTalk Unzipped for conversations with the scientists, founders, regulators, and business leaders shaping the future of medicine.

Medical and scientific disclaimer:

This conversation is provided for educational and informational purposes only. It does not constitute medical advice, diagnosis, or treatment guidance. The therapies and outcomes discussed may be investigational, preliminary, or based on individual experience. Early observations do not establish safety or efficacy. Patients and caregivers should consult qualified healthcare professionals before making medical decisions.

#CancerImmunotherapy #NKCells #PediatricCancer

Oncology drug development is becoming more complex, and bioanalysis can no longer be treated as simple drug measurement.

Sponsored by Leucentra, https://leucentra.com/

Inspired by science, empowered by IT. Leucentra helps life science and healthcare organizations evaluate, implement, and get more value from technology that supports innovation.

In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe speak with Dr. Lakshmi Amaravadi, Head of Oncology Bioanalysis at AstraZeneca, live from AAPS PharmSci 360 in San Antonio.

Dr. Amaravadi unpacks why biomarker validation is not one-size-fits-all, how context of use should guide scientific decision making, and why fit-for-purpose validation matters in modern oncology drug development.

The conversation explores:

00:00 Why oncology bioanalysis is becoming more complex

02:12 FDA biomarker validation guidance and industry response

04:27 What “fit for purpose” means in practice

06:38 PK assay validation vs biomarker assay validation

07:52 What drives Dr. Amaravadi’s work in translational science

10:48 Why validation is not a checkbox exercise

12:19 Advice for young scientists entering bioanalysis

15:12 Why oncology drug development is uniquely complex

18:17 ADCs, bispecifics, T-cell engagers, and conditional T-cell engagers

19:38 Why bioanalysis now requires understanding biology

20:46 Dr. Amaravadi’s path from molecular biology to bioanalysis

24:34 Critical reagent management in complex oncology assays

26:42 Validation, qualification, and context of use

29:03 Final thoughts from AAPS PharmSci 360

This episode is especially relevant for scientists, bioanalytical leaders, translational researchers, clinical pharmacologists, oncology development teams, biomarker scientists, and anyone working at the intersection of drug development, assay validation, and precision medicine.

Dr. Amaravadi discusses how oncology programs now involve ADCs, bispecifics, T-cell engagers, conditional T-cell engagers, complex linkers, multiple measurable species, immunogenicity considerations, and biomarker strategies that require deeper biological understanding. As she explains in the episode, the future of oncology bioanalysis is not simply measuring what is present. It is understanding what the measurement means in the context of the biology and the development decision.

Follow BioTalk Unzipped for conversations with leaders in biotech, pharma, bioanalysis, clinical development, translational science, regulatory strategy, and the future of medicine.

Guest

Dr. Lakshmi Amaravadi

https://www.linkedin.com/in/lakshmi-amaravadi/

Hosts

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

Sponsor: Leucentra

Related Links

Celerion

https://www.celerion.com/

Can artificial intelligence help make cancer therapies safer, more targeted, and more effective?

In this episode of BioTalk Unzipped, Gregory Austin sits down with Dr. Daniel Reker, Assistant Professor at Duke University, for a wide-ranging conversation on active machine learning, nanomedicine, drug delivery, and the future of AI in biomedical research.

This episode is brought to you by Leucentra.

Inspired by Science Empowered by IT

https://leucentra.com/

Dr. Reker works at the intersection of AI, chemistry, biomedical engineering, pharmacology, and molecular medicine. His lab develops computational and experimental approaches to better understand small molecules, nanoformulations, and drug delivery systems.

The conversation explores how machine learning can support drug discovery and development, especially in areas where datasets are small and the biology is complex. Dr. Reker explains why nanoformulations may be able to improve targeted drug delivery, reduce toxicity, and potentially revive therapeutic agents that previously failed because of safety or tolerability issues.

Gregory and Dr. Reker also discuss explainable AI, the risks of black box thinking, AI bias, predictive modeling, FDA considerations, non-animal models, and the responsible use of AI in education and science.

Topics include:

• Active machine learning in drug discovery

• AI and nanomedicine

• Cancer therapy and targeted drug delivery

• How nanoformulations may reduce toxicity

• Small datasets in biomedical AI

• Explainable AI and scientific trust

• AI bias and model limitations

• Regulatory implications for predictive models

• The role of AI in education and cognitive development

• The future of integrated data in drug development

Guest bio:

Dr. Daniel Reker is an Assistant Professor at Duke University. His research focuses on computational and experimental approaches to molecular medicine, including active machine learning, drug delivery, nanoformulations, small molecules, and translational pharmacology. He was named to Forbes 30 Under 30 Europe in Science and Healthcare.

Guest contact:

Dr. Daniel Reker

Email: [email protected]

LinkedIn: https://www.linkedin.com/in/danielreker/

Duke website: https://rekerlab.pratt.duke.edu/

Connect with BioTalk Unzipped:

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

BioTalk Unzipped uncovers the stories behind medical progress through conversations with innovators across biotech, pharma, medtech, bioanalysis, clinical research, regulatory science, and drug development.

Why haven’t we cured cancer yet? A Genentech scientist explains the real reason.

In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Bob Liu, Senior Principal Scientist at Genentech Roche, to unpack one of the most important and misunderstood questions in modern medicine.

This is a rigorous, scientifically grounded conversation on cancer biology, immunotherapy, and the real constraints shaping oncology drug development today.

Dr. Liu brings over a decade of experience across antibody drug conjugates (ADCs), bispecific antibodies, and CAR-T therapies, offering a rare, insider perspective on why a universal cure remains elusive and where meaningful progress is actually being made.

Thanks to our Founding Sponsor:

LEUCENTRA: Helping teams evaluate, implement, and get real value from IT solutions that support innovation, not slow it down. https://leucentra.com/

• Why cancer is not one disease, but more than 200 biologically distinct conditions
• What “curing cancer” actually means in clinical oncology
• How immunotherapies like checkpoint inhibitors and CAR-T are changing outcomes
• The biological limits of eliminating every cancer cell
• How tumors evade immune detection and adapt over time
• Why only about 20% of patients respond to immuno-oncology therapies
• The role of biomarkers, molecular profiling, and precision medicine
• Why early detection remains one of the biggest unsolved challenges
• The economic and regulatory pressures shaping next-generation therapies

Cancer is not simply something to eliminate.

It is a dynamic, adaptive system evolving within the human body. The future of oncology is not just eradication, but control, personalization, and intelligent engagement of the immune system.

“Cancer is a collection of more than 200 diseases, each requiring its own specific approach.”

“The cure for some cancers is within reach, but for many others, early detection remains the critical challenge.”

“Our immune system is constantly surveilling. The key is learning how to harness it effectively.”

00:00 – Introduction

02:42 – Bob’s passion - AACR

04:28 – Why we haven’t cured cancer

07:09 – Defining a cancer cure

10:26 – Cancer classification and molecular signatures

14:16 – Methylation profiling in diagnosis

17:22 – Patient resources and navigation

21:14 – FDA shifts toward randomized trials for CAR-T

24:15 – Cost and access challenges

26:33 – Cancer vs cardiovascular disease progress

33:39 – The challenge of early detection

37:48 – Biomarker limitations

39:14 – Immune system dynamics in cancer

45:19 – Bioanalytical challenges in modern therapies

51:08 – Progress and future outlook

Dr. Bob Liu is a Senior Principal Scientist at Genentech Roche specializing in bioanalytical sciences and immunogenicity assessment for advanced oncology therapies, including T-cell bispecifics and CAR-T.

FDA to tighten approval requirements for CAR-T therapies

https://www.raps.org/news-and-articles/news-articles/2025/12/fda-to-tighten-approval-requirements-for-car-t-cel

American Association for Cancer Research (AACR)

https://www.aacr.org/

National Cancer Institute – Molecular diagnostics and biomarkers

https://www.cancer.gov/about-cancer/diagnosis-staging/diagnosis

Pattern recognition technologies in diagnostics

https://toby.health

Dr. Bob Liu

https://www.linkedin.com/in/bob-liu-42b8b278/

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

The path to curing cancer is not a single breakthrough. It is a long, complex progression of scientific advances, better diagnostics, and deeper biological understanding.

The progress is real.

But the work is far from finished.

Recorded October 31, 2025

In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Kate Neville, immunologist turned seasoned biotech patent attorney at Marshall, Gerstein & Borun, to unpack one of the most misunderstood and mission-critical areas of life sciences: intellectual property.

If you are a biotech founder, scientist, executive, or investor, this conversation is essential listening.

We explore what patent prosecution really means, when startups should begin thinking about IP protection, how “freedom to operate” can determine commercial viability, and how emerging AI tools are reshaping the patent landscape.

Dr. Neville brings 25+ years of experience guiding university spin-outs, biotech startups, and global pharmaceutical companies through complex patent strategy. She has helped secure patents for FDA-approved drugs and offers a rare dual perspective as both scientist and attorney.

• The difference between patent prosecution and patent litigation

• Why it is never too early for biotech startups to think about IP

• The U.S. one-year grace period vs. Europe’s stricter disclosure rules

• What “Freedom to Operate” really means for commercialization

• Antibody patents, CDR regions, and the doctrine of equivalents

• How premature disclosure can impact global patent strategy

• The real-world back-and-forth of patent office “office actions”

• AI-assisted prior art search at the USPTO — opportunity or risk?

• How funding cycles influence patent filing decisions

• Women in biotech leadership and venture funding disparities

• The most rewarding part of protecting life-changing therapies

We also break down the USPTO’s new AI pilot programs designed to modernize patent examination and discuss how artificial intelligence may impact biotech patenting over the next several years.

Intellectual property is often the single most valuable asset in a biotech company.

Strong IP strategy can unlock funding, partnerships, and market exclusivity.

Weak or mistimed IP decisions can permanently limit global opportunity.

For founders and scientists: timing, geography, and disclosure discipline matter more than most people realize.

Dr. Kate Neville

Partner, Marshall, Gerstein & Borun

PhD in Immunology, JD

LinkedIn: https://www.linkedin.com/in/kate-neville-phd/

Firm Bio: https://www.marshallip.com/katherine-l-neville-ph-d/

Charity Highlight: Girls on the Run Chicago

An organization building confidence and resilience in young girls through mentorship and athletic achievement.

https://www.girlsontherun.org/

Dr. Chad Briscoe

Bioanalytical Scientific Leader

https://www.linkedin.com/in/chadbriscoe/

Gregory Austin

Director, Business Development | Bioanalysis

https://www.linkedin.com/in/gregoryaustin1/

If you enjoyed this episode, subscribe to BioTalk Unzipped on Apple Podcasts, Spotify, or your preferred platform and share with a colleague in biotech, pharma, or life sciences innovation.

In this episode of BioTalk Unzipped, hosts Gregory Austin and Dr. Chad Briscoe sit down with Glafabra CEO: Dr. Chris Hopkins, geneticist, biochemist, and biotech entrepreneur, to explore the science and strategy behind next generation cell-based gene therapies for rare diseases.

With more than 25 years of experience spanning gene augmentation, rare disease biology, CRISPR licensing, and biotech formation, Dr. Hopkins shares how autologous, ex vivo engineered cell therapies may overcome key limitations of current enzyme replacement and viral gene therapies, particularly for Fabry disease.

The conversation dives deep into:

• How lentiviral gene augmentation in patient derived cells enables sustained enzyme production

• Why redosing matters and where one time AAV therapies fall short

• The scientific rationale for early intervention, including potential newborn treatment

• Differences between autologous and emerging allogeneic approaches

• Regulatory pathways for rare disease therapies and recent FDA developments

• The role of non animal models in translational research

• Montana’s early access therapy law and its broader implications

• Building biotech platforms amid a challenging funding environment

Topics include cell based gene therapy, Fabry disease, lentiviral vectors, stem cell engineering, rare disease drug development, regulatory science, and translational medicine.

Subscribe to BioTalk Unzipped for in depth conversations with the scientists and leaders shaping the future of biomedical innovation.

00:00 - Intro

00:53 – Welcome to BioTalk Unzipped, Guest intro: Dr. Chris Hopkins

02:10 – Guest charity: Environmental Defense Fund

03:12 – His journey into rare-disease therapeutics and Glafabra

05:58 – Discovering a new enzyme-deficiency therapy

06:39 – Current standard of care

07:42 – How the new autologous cell therapy works

09:40 – Treating patients earlier (even newborns)

10:33 – Emerging therapies - AAV gene therapy vs. cell-based therapy

12:16 – Long-term results & repeat dosing

14:30 – Future plans: T-cells & allogeneic approaches

18:08 – New News: FDA resubmission for rare disease

20:00 – Navigating FDA pathways

22:06 – Non-animal testing & alternative models

25:50 – Montana’s early-access therapy law & medical tourism

29:03 – Could other states follow?

31:31 – Biotech’s current funding challenges

33:46 – New News: Gene therapy trial saves 4-year-old

37:09 – Long-term vision for expanding therapies

39:53 – Personal segment: outdoor life & skiing

44:43 – Guest question on international trade

Dr. Christopher Hopkins

https://www.linkedin.com/in/christopherehopkins/

Glafabra - https://www.glafabra.com/

Environmental Defense Fund - https://www.edf.org/

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

Celerion - https://www.celerion.com/

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

Celerion - https://www.celerion.com/

New News Articles:

US FDA asks Stealth BioTherapeutics to resubmit application for rare genetic condition therapy

https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-asks-stealth-biotherapeutics-resubmit-application-rare-genetic-condition-2025-05-29/

Gene therapy trial saves boy, 4, from 'death sentence'

https://www.thetimes.com/uk/healthcare/article/gene-therapy-trial-great-ormond-street-70l2sgqw

Montana, revolutionary law passed: unlimited research for longevity

https://en.ilsole24ore.com/art/montana-approved-revolutionary-law-researching-longevity-without-limits-AHmDI7BB?refresh_ce=1

#CellBasedGeneTherapy #RareDisease #FabryDisease #GeneAugmentation #LentiviralVectors #Biotech #TranslationalScience #GeneticMedicine #BioTalkUnzipped

https://youtu.be/VcPXZmK-XU8

In this episode of BioTalk Unzipped, Gregory Austin and Dr. Chad Briscoe sit down with Dr. Binodh DeSilva, Senior Vice President of Bioanalysis at Ultragenyx Pharmaceutical, to explore the science and soul behind rare-disease drug development.

From her early days studying electrochemistry at the University of Kansas to leading cutting-edge bioanalytical programs at Ultragenyx, Dr. DeSilva shares how curiosity and community shaped her four-decade career. She discusses the profound responsibility of working with limited, often irreplaceable patient samples with care.

A special thanks to AAPS (https://www.aaps.org/) for their help and support of this episode.

The conversation dives into:

• Balancing rigor and agility in small-population clinical studies
• Leveraging entrepreneurial mindsets from biotech within big pharma frameworks
• The promise of dried blood spots (DBS) and patient-centric sampling
• Mentorship, curiosity, and the future of scientific leadership
• Her return to Sri Lanka with KU faculty to recruit the next generation of scientists

Throughout the discussion, DeSilva underscores a recurring theme: science thrives when curiosity meets compassion. This episode is a masterclass in both.

Guest Links

Dr. Binodh DeSilva

https://www.linkedin.com/in/binodh-desilva/

Ultragenyx Pharmaceuticals - https://www.ultragenyx.com/

Hosts

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/

Celerion - https://www.celerion.com/

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

Celerion - https://www.celerion.com/

Keywords: BioTalk Unzipped, Binodh DeSilva, Ultragenyx, rare disease research, bioanalysis, dynamic drug development, dried blood spots, DBS sampling, biologics, AAPS NBC 2025, Gregory Austin, Chad Briscoe, Celerion, scientific leadership, mentorship in science, biopharma innovation, curiosity in research, Sri Lanka scientists, analytical chemistry, pharma innovation, drug development ethics.