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: https://t.co/fOe6pR4LKr
Most antibodies act as antagonists—but biology isn’t that simple. @MetaphoreBio's function-first platform uses live-cell data + ML to design antibodies that agonize, bias, or multi-target pathways. CEO Angela Hwang explains: https://t.co/CIt4NfEm0R
FARA CEO Jen Farmer and @Solid_Bio 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
https://t.co/gTwQ2ptc70
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.
https://t.co/meRr2ndQvl
Winsights' Kasey Walsh discusses her daughter's ultra-rare genetic disorder diagnostic journey, gaps in how the patient experience informs drug development, and the Winsights data platform empowers rare disease communities to drive research priorities.
https://t.co/hUhwXL3WdO
@EricElenko@PureTechH 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.
https://t.co/6aTWufIXFl
Trialport's Keith Berelowitz discusses his vision for a future where clinical trials are routinely discussed alongside standard of care, the gap between clinical trial availability and patient awareness, and how the Trialport platform informs patients.
https://t.co/rCrjqiP4GB
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.
https://t.co/omP2EG9Rt8
John Wollman @KomodoHealth 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.
https://t.co/Lu91t2Hmbw
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.
https://t.co/gw9MhfTY56
@Bayer CDIO @BijoyCido 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 .
https://t.co/FSVGhL89IX
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#RARECast
https://t.co/jLC4E0kM9C
When inflammation spirals out of control, traditional drugs often don’t work. @Mesoblast's Ryoncil—the first MSC therapy in the US—targets GVHD in children. CEO @DrItescu discusses how MSCs can calm cytokine storms and what’s next for the platform.
https://t.co/CfjfEqzVqG
Most MS drugs control relapses—but don’t stop the neurodegeneration driving long-term disability. Daniel Vitt, CEO of @ImmunicInc discusses how his company’s experimental once-daily oral therapy targets both inflammation and neuronal damage.
https://t.co/18Nij63348
Jim Foote, CEO of First Ascent Biomedical, explains how combining genomics, functional assays, and AI could transform rare pediatric cancer care @GlobalGenes#RARECast
https://t.co/p0Aw6makIP
@garidel_de CEO of @Abivax_ discusses how a once-failed HIV drug evolved into a late stage oral IBD therapy that may deliver durable remission, how it acts upstream of key inflammatory pathways, and its potential in a crowded but underserved IBD market.
https://t.co/Q0EBSK3Xet
Pablo Sardi @sanofi discusses the potential of the oral BTK inhibitor rilzabrutinib to treat multiple rare autoimmune conditions, and how this strategy could shift the field from treating symptoms to targeting root causes of these conditions. @GlobalGenes
https://t.co/lfjnzY24ll
Dennis Salotti @JazzPharma discusses how use of AI for mundane but high-impact work speeds studies and improves trial-site experience and how AI can be a thinking partner, rather than a shortcut, to elevate people from doers to critical thinkers.
https://t.co/nHsdoycrWq
For people with Lynch syndrome, cancer is often a question of when—not if. Nouscom CEO Marina Udier discusses the company’s efforts to develop cancer vaccines to stop tumors before they start by training the immune system to recognize early cancer signals.
https://t.co/tIPLZ566Xb