Per Lundin, the company’s co-founder and CEO, discusses the promise and limitations of exosome-enabled gene editing, how the company is prioritizing its pipeline, and what it will take to establish th...

Eric Scheeff, chief scientific officer of Idefine and parent of a child with Kleefstra syndrome, discusses the organization’s progress in building the scientific foundation for a potential treatment, ...

Congenital hyperinsulinism can turn a newborn’s first days into a medical emergency. The body produces too much […]

Here's what you may have missed in August from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. The Bio Report Innovating ADCs with Dual Targeting and Smarter Delivery Antibody-drug conjuga...

Doug Love, CEO of Annexon, discusses the biological role of the classical complement pathway in Guillain-Barré syndrome, the limitations of current nonspecific standards of care, and the company’s eff...

Raza Bokhari, CEO of Medicus Pharma, discusses the company’s approach to developing a localized, non-surgical treatment for people with Gorlin syndrome, how the patch is intended to induce tumor-cell ...

Panicos Shangaris, a clinical senior lecturer and consultant in maternal and fetal medicine at King’s College London, discusses what daily life looks like for people living with sickle cell disease to...

Myasthenia Gravis Association Executive Director Allison Foss and Director of the Neuromuscular Center at Cleveland Clinic John Morren discuss the everyday impact of conditions like myasthenia gravis,...

Giacomo Chiesi, head of Chiesi Rare Diseases, discusses how the business has grown through acquisitions, its move into CRISPR gene editing and blood–brain barrier crossing enzyme platforms, and its br...

Warner Biddle, CEO of Kyverna Therapeutics, discusses the company’s B cell–targeting cell therapy platform, the range of autoimmune diseases it aims to address, and its next-generation therapies in de...

Jeff Coller, director of the Johns Hopkins RNA Innovation Center why the promise of bespoke gene editing therapies will be constrained less by science than by regulatory, manufacturing, and reimbursem...

Sunstone Health founder Joshua Resnikoff discusses how his son’s rare disease diagnostic odyssey gave rise to the company, how Sunstone is working to transform the path to a diagnosis, and its busines...

Craig Leppert, founder of the non-profit Shadow Jumpers, discusses his experience with EPP, the benefits he’s had as a participant in a clinical trial of an experimental therapy to treat the condition...

Miquel Vila-Perello, CEO of SpliceBio, discusses the company’s platform technology that enable to the delivery of large genes in AAV vectors, its work to date in Stargardt disease, and preclinical dat...

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 se...

PacBio CEO Christian Henry discusses the company’s long-read HiFi whole-genome sequencing, how it is reshaping the rare disease diagnostic journey, and the growing acceptance of the technology among p...

Friedreich’s Ataxia Research Alliance CEO Jennifer Farmer and Solid Biosciences chief medical officer Gabriel Brooks discuss the lived reality of Friedreich’s ataxia, Solid Bioscience’s next-generatio...

Kasey Walsh, founder and CEO of Winsights, discusses her daughter's diagnostic journey, the critical gaps in how patient experience informs drug development, and how Winsights empowers rare disease co...

Keith Berelowitz, founder and CEO of Trialport, discusses his vision for a future where clinical trials are discussed alongside standard of care as a routine healthcare option, the gap between clinica...

Matching phenotype to genotype at scale could transform how rare diseases are found, understood, and treated. Komodo […]

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Akash Kumar, co founder and chief medical officer of Myome, discusses where genome sequencing now fits into care pathways, how it captures hard to detect variant types; and what it means for treatment...

Nicole Johnson and Nasha Fitter, co-founders of the FOXG1 Research Foundation, discuss how a parent-driven foundation became a virtual biotech capable of advancing a gene therapy into human clinical t...

Jim Foote, CEO of First Ascent biomedical, discusses how combining genomics, functional assays, and AI offers a realistic path to better outcomes, fewer lifelong toxicities, and more rational use of e...

Sanofi is exploring similarities in rare autoimmune conditions that may allow it to treat a number of different disorders with a single therapy. Pablo Sardi, head of rare disease research at Sanofi, discusses the company’s oral BTK inhibitor rilzabrutinib, the challenges of testing one drug in different rare diseases, and how this kind of approach might push doctors to focus less on symptoms and more on the root causes of a rare disease.