# RARECast RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy. Page: Feed: --- ## Curing Sickle Cell Before Life Begins _2026-08-13_ Sickle cell disease is caused by a single mutation in the beta-globin gene that leads to painful crises, anemia, and organ damage. Despite advances in treatment, it remains a devastating and often overlooked global health challenge, particularly in low-resource settings where children frequently go undiagnosed until life-threatening complications arise. The PERICLES project is an ambitious… [Listen](https://rss.art19.com/episodes/8ed1a96a-f60e-4ce9-b441-8ed3c53c8065.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Spotting Neuromuscular Red Flags _2026-08-06_ Many older adults may dismiss dropping objects, struggling with stairs, or tiring on short walks as a matter of just getting older, but it can be an early sign of a serious group of disorders known as late-onset neuromuscular diseases (LONDs). The American Neuromuscular Foundation’s Why Behind Your Weakness campaign seeks to raise awareness of LONDs and the shared symptom patterns that too often… [Listen](https://rss.art19.com/episodes/e4ca370d-4c65-4723-a10a-8974e4e15747.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Expanding into a Global Rare Disease Player through Deal-Driven Innovation _2026-07-30_ Chiesi Global Rare Diseases has rapidly evolved from a small, regional rare‑disease business into a global organization, leveraging strategic deals and development bets to reshape standards of care for patients worldwide. Giacomo Chiesi, head of the unit, discusses how the business has grown through acquisitions, its move into CRISPR gene editing and blood–brain barrier‑crossing enzyme platforms,… [Listen](https://rss.art19.com/episodes/e4ec8a3c-c76e-41e4-9d3e-e0261673f219.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## When Geography, Cost, and Policy Become as Life-Limiting as a Disease _2026-07-23_ When Tom Sayiner was diagnosed with the fatal neurodegenerative disease ALS, he and his wife, Tamara, learned that tofersen had been approved in Europe as a therapy that could slow the progression of his genetic form of the disease. But the Sayiners, who live in Sweden, soon discovered they could not access the drug because a Swedish health technology assessment council determined there was… [Listen](https://rss.art19.com/episodes/ba4cd802-900a-4076-9c2b-370821734baa.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## A One-Time Cell Therapy to Reset the Immune System in Autoimmune Diseases _2026-07-16_ People with rare, severe autoimmune diseases often live for years with progressive, disabling conditions managed by chronic immunosuppression that rarely addresses the underlying cause. Kyverna is developing an autologous CAR T-cell therapy designed to deliver a deep immune reset by broadly depleting pathogenic B cells, followed by repopulation with naïve, non-pathogenic B cells and normalization… [Listen](https://rss.art19.com/episodes/315ac594-9982-4a63-b6e1-1f38273d97f3.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Building the Infrastructure for Made‑to‑Order Gene Therapies _2026-07-09_ The case of baby KJ Muldoon, an infant born with a lethal genetic metabolic disorder, demonstrates the potential to compress years of therapeutic development into months using an in vivo base editing approach. Jeff Coller, director of the Johns Hopkins RNA Innovation Center, wrote about the case in a New York Times op-ed, arguing that CRISPR-based base editors—delivered via lipid nanoparticles as… [Listen](https://rss.art19.com/episodes/c04217ff-31b9-49bd-a9db-6029e3be8d3b.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Rewiring the Rare Disease Diagnostic Odyssey _2026-07-02_ Families seeking a diagnosis for a rare disease often face a protracted diagnostic odyssey that can include ER visits, specialist referrals, and dead ends, even at world-class medical centers. Parents bounce from doctor to doctor while payers absorb mounting costs, and the pivotal moment of putting a name to a disease—which can reduce unnecessary care and emotional distress—arrives late, if at… [Listen](https://rss.art19.com/episodes/59915023-9190-40c1-b6e1-3fca2bdc2f36.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Emerging from a Life in the Shadows _2026-06-25_ Erythropoietic protoporphyria (EPP) is a rare, inherited metabolic disorder that triggers a toxic photochemical reaction in skin when exposed to light. A short time in the sun for someone with the condition can result in excruciating, second-degree–like burns and leaves patients sidelined from normal outdoor activity. Craig Leppert, who has EPP, saw his childhood shaped by constant vigilance… [Listen](https://rss.art19.com/episodes/6553f69c-d236-49a9-bf5d-c20f93c38c06.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## Fitting Big Genes into Small Vectors _2026-06-18_ Many disease‑causing genes are too large to be packaged into standard AAV gene therapy vectors, leaving a long list of otherwise gene-therapy-ready conditions without viable treatments. SpliceBio is leveraging a protein splicing platform based on engineered split inteins to overcome the cargo limitations of AAV gene therapy vectors, enabling delivery of large genes to potentially treat a broad… [Listen](https://rss.art19.com/episodes/a15f067f-1fb9-44f0-b55a-7caf1c75ccce.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e) ## [Non-custodial Crypto Payments & Escrow (Sponsored)](https://crawlproof.com/a/qNBJCE8jGnxZ) _2026-06-18 · **Sponsored**_ Accept multi-chain crypto with trustless escrow, instant confirmations, and no KYC ## Targeting Iron Dysregulation in the Neurodegenerative Condition MSA _2026-06-11_ Multiple system atrophy is a rapidly progressive neurodegenerative condition that is often misdiagnosed as Parkinson’s disease but carries a far grimmer prognosis. MSA has a median survival of just seven to eight years after symptom onset. Toxic aggregates of alpha‑synuclein and excess brain iron create a vicious cycle of neuronal damage that drives the multisystem motor and autonomic decline… [Listen](https://rss.art19.com/episodes/a6c068d5-76bc-4ae4-937e-5fd3cf614ae7.mp3?rss_browser=BAhJIhRSU1NBbXBsaWZpZXJCb3QGOgZFVA%3D%3D--6345a2e4a0991d4cbe92da92e0fa95ff9d12de2e)