Ankit Jain, CEO of Infinitus, discusses how his daughter’s rare disease exposed the obstacles families face, how the company’s AI agents work to reduce delays, and where human judgment and connection must remain central to care. @GlobalGenes #RARECast globalgenes.org/raredaily/us…

Arun Swaminathan @CoyaTx discusses how dysfunctional regulatory T cells contribute to neuroinflammation and neuronal damage in ALS, why the company is targeting them as a driver of disease progression, and the case for its dual immunomodulatory approach. globalgenes.org/raredaily/ta…

Geoff McDonough @RezeraInc discusses inflammation in heart disease, the company’s phase 3 trial of ruvonoflast in peripheral artery disease, and its next-generation, brain-penetrant NLRP3 inhibitor in development for neuroinflammatory conditions. thebioreport.podbean.com/e/t…

How can patient advocates and industry collaborate more effectively on drug development? Watch our panel from the @GlobalGenes Rare Drug Development Symposium @wendy_erler, Michelle Davis @ifopa, @TaiPasquini youtube.com/watch?v=B23GzMep…

Patient advocate Monica Dubeau of youmightbeazebra.com discusses fragmented care, diagnostic delay, and what must change for people with rare diseases. @GlobalGenes #RARECast globalgenes.org/raredaily/wh…

Here's what you may have missed in September from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. linkedin.com/feed/update/urn…

Ryan Beal @DyveBio discusses why acidity is an overlooked but foundational target in oncology, how its delivery technology may overcome the limitations of oral bicarbonate dosing, and indications beyond cancer that the company is pursuing. thebioreport.podbean.com/e/n…

Steven Ringel, founder and CEO of Nome, discusses helping families and patient groups evaluate genetic-medicine options, shape development plans, and connect with partners to advance individualized therapies toward the clinic. @globalgenes #RARECast globalgenes.org/raredaily/en…

Catharina Svanborg @HamletBioPharma , discusses Alpha1H—an experimental synthetic peptide inspired by a protein complex in human breast milk—designed to target bladder tumor cells while sparing healthy tissue. thebioreport.podbean.com/e/e…

Catharina Svanborg @HamletBioPharma , discusses Alpha1H—an experimental synthetic peptide inspired by a protein complex in human breast milk—designed to target bladder tumor cells while sparing healthy tissue. thebioreport.podbean.com/e/e…

Per Lundin of Evox Therapeutics, discusses the promise and limitations of exosome-enabled gene editing, how Evox is prioritizing its pipeline, and what it will take to establish this emerging therapeutic strategy in the clinic. @GlobalGenes #RARECast globalgenes.org/raredaily/us…

Eric Scheeff, CSO of Idefine, discusses progress in building the scientific foundation for a treatment for the neurodevelopmental condition Kleefstra syndrome, the role of its patient community, and its work ahead. @globalgenes #RARECast globalgenes.org/raredaily/bu…

Julie Raskin, CEO of Congenital Hyperinsulinism International discusses how an NICU crisis gave rise to the organization and its global fight for better treatments. @globalgenes #RARECast globalgenes.org/raredaily/th…

Sid Kerkar @AllerGeneAI on the company’s efforts to reset mast cells in people with severe allergies, the potential for mRNA and lipid nanoparticle delivery to simplify and broaden access to CAR-T therapy, and what it will take to advance the platform. thebioreport.podbean.com/e/r…

Here's what you may have missed in August from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. linkedin.com/pulse/gileads-s…

Doug Love @annexonbio discusses Guillain-Barré syndrome, the limits of current nonspecific care, and Annexon’s targeted approach to stopping the complement-driven inflammatory cascade at its source. globalgenes.org/raredaily/st…

Mayank Gandhi, co-founder and CEO of @NEOKBio , discusses the company’s bispecific ADC platform, its two clinical programs, and its strategy to develop more selective therapies for patients with advanced solid tumors. thebioreport.podbean.com/e/i…

Alan Trounson Evinco discusses the company’s effort to develop NK cell-derived particles to cross the blood-brain barrier, curb neuroinflammation, and promote amyloid clearance in Alzheimer’s. thebioreport.podbean.com/e/t…

Could sickle cell be cured before birth? @pshangaris @KingsCollegeLon discusses the PERICLES Project and prenatal gene editing’s promise. @GlobalGenes #RARECast globalgenes.org/raredaily/cu…

Unexplained weakness isn’t just aging. @Myasthenia Gravis Association’s Allison Foss and @ClevelandClinic John Morren discuss red flags for late-onset neuromuscular disease and a new clinical diagnostic tool. @globalgenes #RARECast globalgenes.org/raredaily/sp…