Tessera

Multispecific antibodies unlock new biology—but safety risks are often designed in. @labgeni_us' @AngusSinclair discusses how its AI platform engineers multi-specific T‑cell engagers that are both potent and selective in solid tumors. thebioreport.podbean.com/e/turning-mu...

Families seeking rare disease diagnoses face years of dead ends. Sunstone Health uses AI and rapid WGS to speed up answers for high-risk families. @globalgenes #RARECastListen to founder Joshua Resnikoff on @globalgenes.bsky.social #RARECast: globalgenes.org/raredaily/65...

Rewiring the Rare Disease Diagnostic Odyssey

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...

globalgenes.org

Acute ischemic stroke is common yet undertreated—few patients get thrombolytics or thrombectomy due to narrow treatment windows and bleeding risk. Basking Biosciences is developing a reversible vWF-targeting thrombolytic to expand access and safety. thebioreport.podbean.com/e/addressing...

EPP is an ultra-rare disorder where brief sun exposure causes extreme, burn-like pain. Shadow Jumpers founder Craig Leppert discusses growing up with undiagnosed EPP and how a clinical trial therapy offers new hope. #RARECast @globalgenes.bsky.social globalgenes.org/raredaily/em...

Emerging from a Life in the Shadows

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...

globalgenes.org

Plasma gelsolin, a natural regulator of inflammation depleted in severe illness, may offer a first-in-class, non-immunosuppressive way to tame cytokine storms in ARDS and beyond. Hear BioAegis CEO Susan Levinson on this pipeline-in-a-product. thebioreport.podbean.com/e/a-pipeline...

AAV gene therapies have cargo limits that prevent them from being used to treat large disease genes. @SpliceBioHQ uses split inteins to deliver oversized genes, with a dual-AAV program CEO Miquel Vila-Perello explains: @globalgenes.bsky.social #RARECast globalgenes.org/raredaily/fi...

Fitting Big Genes into Small Vectors

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...

globalgenes.org

High quality targets remain a bottleneck in drug discovery. Regeneron is addressing this by combining human genetics, proteomics, clinical data, and AI to scale target discovery. Aris Baras, head of the Regeneron Genetics Center, explains. thebioreport.podbean.com/e/building-a...

David Stamler, CEO of Alterity, discusses the biology of multiple system atrophy, the company’s promising clinical results to date, and why its therapeutic approach may also have application in other neurodegenerative diseases. @globalgenes.bsky.social #RARECast globalgenes.org/raredaily/ta...

Targeting Iron Dysregulation in the Neurodegenerative Condition MSA

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...

globalgenes.org

Epigenetics may be cancer’s operating system. K36 is targeting a key enzyme that lets multiple myeloma cells grow, adapt, and resist treatment—aiming to re-sensitize tumors to existing drugs. A shift from chasing mutations to rewriting how cancer reads DNA. thebioreport.podbean.com/e/stopping-s...

Rare disease diagnosis is at a turning point. Long-read HiFi sequencing is boosting diagnostic yield by 10–15% by capturing structural variants, epigenetics, and regulatory signals—making the case for first-line use. @PacBio CEO Christian Henry explains. globalgenes.org/raredaily/lo...

Long Reads, Shorter Journeys

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...

globalgenes.org

Most antibodies act as antagonists—but biology isn’t that simple. Metaphore’s function-first platform uses live-cell data + ML to design antibodies that agonize, bias, or multi-target pathways. CEO Angela Hwang explains: thebioreport.podbean.com/e/rewriting-...

FARA CEO Jen Farmer and Solid Biosciences CMO Gabriel Brooks explore the lived reality of Friedreich’s ataxia, the promise of next-generation gene therapy, and how true patient industry partnerships can shape trial design and development. @globalgenes.bsky.social globalgenes.org/raredaily/th...

The Critical Role of Patient Community-Industry Partnership in Advancing a Gene Therapy

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...

globalgenes.org

Cells constantly sense and respond to stress—adapting, recovering, or dying. Soley uses AI to map these responses at scale, unlocking “undruggable” targets. Soley's Yerem Yeghiazarians discusses their platform, pipeline, and first-in-class candidates. thebioreport.podbean.com/e/mapping-ce...

Kasey Walsh of Winsights discusses her daughter's ultra-rare genetic disorder diagnostic journey, critical gaps in how the patient experience informs drug development, and the Winsights data platform empowers rare disease communities to drive research priorities. globalgenes.org/raredaily/ca...

Capturing the Lived Experience of Rare Disease

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...

globalgenes.org

Eric Elenko, president and co founder of PureTech, discusses the company’s disciplined approach to rescuing promising but discontinued therapeutics, its hub and spoke structure, and how this model can turn partially derisked assets into commercial successes. thebioreport.podbean.com/e/turning-ab...

Trialport's Keith Berelowitz discusses his vision for a future where clinical trials are discussed alongside standard of care as a routine option, the gap between clinical trial availability and patient awareness, and how the Trialport platform informs patients. globalgenes.org/raredaily/ra...

Raising Awareness of Clinical Trials

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...

globalgenes.org

Most cancer therapies hit 1 or 2 pathways, giving tumors a chance to adapt. Nuago CEO Robert Schickel discusses how his company is developing short RNAs to silence multiple survival genes at once—aiming for more durable tumor killing with less toxicity. thebioreport.podbean.com/e/targeting-...

John Wollman of Komodo Health discusses how the company and GeneDx are leveraging longitudinal patient data with genomic and phenotypic data to shorten rare disease diagnostic odysseys, accelerate natural hist studies, and enable better clinical decisions. globalgenes.org/raredaily/us...

Using AI and Longitudinal Data to Transform Rare Disease Care

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

globalgenes.org

Crystalis CEO James Mackay discusses gout, why therapies often fail to get uric acid to target levels, and how the company’s next generation URAT1 inhibitor may fill the treatment gap and change daily life for patients living with moderate to severe gout. thebioreport.podbean.com/e/addressing...

Bayer CDIO Bijoy Sagar discusses why AI must be treated as a tool to solve defined business problems, why prompt engineering is an ongoing process rather than a one-shot querry, and how expert teams are empowered to experiment and build AI solutions . www.youtube.com/watch?v=5922...

Building an Outcomes First AI Strategy

YouTube video by Life Sciences DNA Podcast

youtube.com

Genome sequencing is moving from last resort to frontline in rare disease care. Akash Kumar of Myome explains what it means for treatment decisions, trial access, and the emotional burden on families searching for a diagnosis. @globalgenes.bsky.social #RARECast globalgenes.org/raredaily/cu...

Cutting through the Diagnostic Maze for Rare Diseases

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...

globalgenes.org