Last week marked a major milestone for The Genetics Podcast: our 200th episode. To celebrate, host Patrick Short revisited some of the most impactful conversations from the show’s early days. The result is a retrospective that not only highlights how far the field has come, but also reminds us of the timeless ideas and bold predictions that continue to shape genetics and precision medicine today.
In recent years, the therapeutic landscape for genetic hearing loss (GHL) has advanced rapidly, with several programs reaching clinical development. However, trials in this space can present unique challenges. Recruiting patients often requires careful engagement with the deaf and hard-of-hearing community, where cultural and linguistic considerations are paramount. The rarity of certain genetic variants can make it difficult to recruit patients who meet inclusion criteria. In addition, newborn genetic screening is a critical pathway for early diagnosis, but it may not always be available or welcomed by parents.
In the last episode of the Genetics Podcast, Patrick speaks with Dr. Xin Jin, a pioneering neuroscientist at Scripps Research, about her team’s cutting-edge method for decoding the genetics of complex brain disorders. Their approach, in vivo Perturb-seq, combines CRISPR-based gene perturbations with single-cell sequencing to map how genetic risk factors affect specific cell types across the brain.
After a decade of strong investment, the biotech sector in 2025 is navigating a markedly constrained funding environment. Early-stage companies, particularly those in genomics, cell therapy, and precision drug development, are facing longer timelines to raise capital, more scrutiny from investors, and increasing pressure to demonstrate clear ROI across development stages.
Clinical trials have always been burdensome on sponsors, but in the current biotech landscape, the pressure to execute trials promptly, efficiently, and in a cost-effective manner may be more intense than ever. Even companies with comfortable budgets report feeling resource-constrained, and for early-stage companies, executing a trial with limited funding has become a make-or-break challenge. Investor caution, shrinking pipelines, and rising operational costs have forced teams to do more with less, without compromising speed, scientific rigor, or patient safety.
In the latest episode of The Genetics Podcast, we sat down with Dr. Benny Sorenson, CEO of Hemab Therapeutics, to explore a relatively overlooked therapeutic area: bleeding disorders beyond hemophilia. While innovation in hemophilia care has surged over the past decades (with more than 30 approved therapies today), many other serious clotting conditions have seen little progress since the 1960s. Benny and the team at Hemab are aiming to change that.
The promise of precision medicine has been intensifying over the past decade. In 2025, the field appears to be at an inflection point due to a confluence of various factors. Sequencing costs have plummeted with consistent technological development, biobanks have amassed data from millions of individuals, artificial intelligence (AI) is being embedded into discovery pipelines, gene therapy is accelerating, and regulators are evolving to meet the complexity of modern therapeutics. Below are five defining trends that are shaping the scientific landscape of precision medicine this year.
In the latest episode of The Genetics Podcast, we spoke with Dr. Heiko Runz, SVP of Neuroscience at insitro. With a background spanning clinical genetics, academic research, and leadership roles at Merck, Biogen, and now insitro, Heiko has helped pioneer the integration of human genetics into drug discovery. Today, he’s at the forefront of applying AI and large-scale biobank data to unravel the complexity of neurological disease and accelerate therapeutic development.
As part of new efforts to improve FDA processes and enhance communication with stakeholders, they hosted a roundtable on cell and gene therapy on June 5th, 2025. Various academics, physicians, patient advocates, and industry experts were present. With major recent advancements in cell and gene therapy, this initiative signaled that the FDA is keen on building the momentum and spearheading the next wave of therapeutics. In this blog, we provide an overview of the main themes and key takeaways from the 3-hour discussion.
In the latest episode of The Genetics Podcast, we spoke with Dr. Molly He, CEO and co-founder of Element Biosciences. With over a hundred patents and leadership roles at Illumina and Pacific Biosciences, Molly has been a force behind some of the most transformative technologies in genomics. At Element, she’s now leading a revolution in sequencing and multi-omics that could reshape how we understand biology, develop therapeutics, and personalize healthcare.