The Genetics Podcast

This week on The Genetics Podcast, Patrick is joined by Daniel Chung, Chief Medical Officer of Beacon Therapeutics. They discuss lessons from developing the first ocular gene therapy (Luxturna), the complexities of designing and executing pivotal trials in inherited retinal disease, and how endpoint selection, delivery, and immunogenicity shape clinical and commercial success.
Show Notes: 
0:00 Intro to The Genetics Podcast
00:59 Welcome to Daniel
01:57 Daniel’s firsthand experience with the development and approval of Luxturna for inherited retinal dystrophy
06:13 Factors contributing to delays in approval for gene therapies in recent years 
08:13 Overview of ocular diseases and their suitability for gene therapy
11:40 Why Daniel joined Beacon Therapeutics and current priorities for the lead program
13:22 Key challenges in designing and executing a pivotal gene therapy trial for X-linked retinitis pigmentosa (XLRP)
15:49 Lessons from prior inherited retinal disease trials and selecting the right clinical endpoints
21:03 Expanding beyond the lead program into earlier patients and additional ocular indications
23:43 AAV packaging constraints and managing immunogenicity in ocular gene therapy
26:29 Lessons from academia–industry collaboration in developing gene therapies
28:23 Access to genetic testing in inherited retinal diseases and remaining gaps
32:10 Key scientific, regulatory, and commercial challenges facing gene therapy today
35:39 Closing remarks
Find out more:
Beacon Therapeutics

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This week on The Genetics Podcast, Patrick is joined by Zornitza Stark, Professor at the University of Melbourne and Co-Group Leader at the Murdoch Children’s Research Institute. Using early findings from the BabyScreen+ genomic newborn screening study, they examine feasibility, clinical impact, and family-wide implications beyond standard screening, and consider what these insights mean for infrastructure, policy, and equitable implementation at scale.
Show Notes: 
0:00 Intro to The Genetics Podcast
01:00 Welcome to Zornitza
01:55 Methods and findings of the BabyScreen+ study 
04:35 Scaling the BabyScreen+ study from pilot to population screening
07:46 Balancing benefits, risks, and downstream implications in genomic newborn screening
15:55 How the genes tested in BabyScreen+ were selected
19:00 Cascade testing and the family-wide implications of genomic newborn screening
22:05 What large-scale genomic newborn screening could reveal about penetrance 
23:57 Expanding genomic newborn screening over time and addressing equity, scale, and long-term value
27:47 Rapid genomic sequencing in critically ill newborns from pilot studies to national implementation
34:32 Building evidence infrastructure to interpret variants and support reimbursement decisions
37:25 Why global data sharing in genomics requires policy alignment and sustained infrastructure investment
39:55 Current priorities and the future direction of genomics in Australia
42:14 Closing remarks
Find out more:
BabyScreen+ study

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This week on The Genetics Podcast, Patrick is joined for a special recording at the Flagship Pioneering studio during JPM 2026 by Michelle Werner, CEO of Alltrna, and Mike Severino, CEO of Tessera Therapeutics. They discuss the molecular mechanisms behind Alltrna’s engineered tRNA and Tessera’s gene-writing platforms, why 2026 marks a major inflection point as both programs enter the clinic, and considerations around trial design, patient needs, and delivering therapies at scale.
Show Notes: 
0:00 Intro to The Genetics Podcast
00:59 Welcome to Michelle and Mike
01:26 Overview of Alltrna’s therapeutic approach to rare genetic diseases using engineered tRNAs
03:19 Overview of Tessera Therapeutics’ gene writing approach
04:51 Preclinical evidence supporting first-in-human testing of Alltrna’s lead candidate
07:50 Why Tessera’s preclinical models are predictive of clinical success
10:40 Key features that differentiate Tessera’s RNA-based gene writing
14:02 Advantages of using basket trials for engineered tRNAs
18:00 Clinical trial design and early efficacy signals for gene writing in alpha-1 antitrypsin deficiency
21:59 Genetic testing, patient identification, and patient advocacy in Alltrna trials
24:18 Differentiating Tessera’s gene writing approach for patients and investigators
26:35 Site readiness and expertise required for genetic medicine trials
28:32 Scaling Alltrna’s platform across mutations, tissues, and diseases
32:34 Expanding Tessera’s gene writing platform beyond alpha-1 antitrypsin deficiency
35:57 Perspectives on biotech funding, pharma partnerships, and rare disease investment
39:08 The data pharma looks for when partnering on novel genetic therapies
42:49 Emerging technologies Michelle and Mike are watching beyond their own platforms 
47:19 Closing remarks
Find out more:
Alltrna (https://www.alltrna.com/)

Tessera Therapeutics (https://www.tesseratherapeutics.com/)

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https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link

This week on The Genetics Podcast, Patrick is joined by Dr. Travis Hinson, Professor and physician at the University of Connecticut and investigator at the Jackson Laboratory. They discuss how genetics is reshaping the understanding of heart failure, why sarcomere biology has become a central target for new gene-based therapies, and how advances in genome editing and preventive genetics could redefine cardiovascular care.
Show Notes: 
0:00 Intro to The Genetics Podcast
01:00 Welcome to Travis
01:44 Overview of gene editing for inherited cardiovascular disease
05:01 Delivery methods and viral capsid engineering to target heart tissue
07:29 Role of the titin gene in dilated cardiomyopathy and treatment opportunities
10:32 Genetic variants in titin and differences in phenotype
13:51 Promising therapeutic approaches for targeting titin 
16:21 Possibility of using a mini titin gene for replacement
17:31 Sarcomere proteins and their role in cardiomyopathy
20:28 Limits of current understanding in heart failure, including cardiac regeneration and congenital heart disease
24:50 Predicting drug-induced cardiotoxicity using stem cell models, animal studies, and digital twins
30:02 How Travis balances clinical genetics at the University of Connecticut with translational research at the Jackson Laboratory (JAX)
32:03 Where genetic testing is used in cardiology today and what’s limiting broader access
35:19 Understanding polygenic risk and unexplained heritability in cardiovascular disease
37:07 Managing inherited cardiomyopathy risk in families after a pathogenic variant is identified
40:36 Genetic testing as a prevention strategy and the public health case for earlier intervention
43:03 Balancing early genetic screening with penetrance, uncertainty, and patient anxiety
45:51 Closing remarks
Find out more:
Review on cardiovascular gene editing approaches
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This week on The Genetics Podcast, Patrick is joined by Dr. Beth Shapiro, Chief Science Officer at Colossal Biosciences. They discuss her path into ancient DNA and evolutionary genetics, how advances in genome engineering are reshaping de-extinction and conservation science, and why restoring lost ecological functions could transform the future of biodiversity.
Show Notes: 
0:00 Intro to The Genetics Podcast
00:59 Welcome to Beth
01:43 Beth’s path from journalism to ancient DNA research
04:46 Beth’s first project on American bison and near extinction
08:26 How Beth worked on a dodo sample at Oxford and what ancient DNA could reveal
11:09 How de-extinction entered the field and why resurrecting species became a scientific goal
14:54 Why de-extinction efforts could strengthen ecosystems and accelerate species conservation
18:33 How cloning a mammoth works and why genome engineering replaces traditional cloning
25:05 Understanding the genome of a woolly mammoth
28:06 What functional de-extinction means in practice
30:55 Genetic clues behind the woolly mammoth’s coat
33:25 The technical hurdles behind de-extinction
38:23 Building a stepwise path to de-extinction through near-term conservation tools
39:36 Ethics risk management and working with local and Indigenous communities in de-extinction projects
44:59 Scientific and technological breakthroughs needed over the next decade to make de-extinction and biodiversity preservation viable
49:20 Closing remarks
Find out more:
Colossal Biosciences (https://colossal.com/)
Please consider rating and reviewing us on your chosen podcast listening platform! 
https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link