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.
https://t.co/v3xecInGMX
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
https://t.co/USWgcKEItu
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.
https://t.co/Zgyb7bpURd
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.
https://t.co/Zgyb7bpURd
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
https://t.co/EU2wtHfOWT
@ErikIngelsson@WaveLifeSci explains how an RNA-based approach to fat loss could reshape obesity treatment—by targeting the biology that regulates fat breakdown and redefining what success looks like.
https://t.co/AiwaLVNKnY
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
https://t.co/7mfeU6F1zz
@Jack_Silb , CEO of Deck Bio, discusses the company’s efforts to create multi-target T-cell engagers that reach more cancer cells, reduce the likelihood that tumors will evade treatment, and minimize harm to healthy tissue.
https://t.co/D1sXCAwoQ5
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
https://t.co/TKzR5TgcNv
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. https://t.co/ovgHXeoEQ2
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.
https://t.co/U1axaTR1wt
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.
https://t.co/zZtAmRRD0E
@R_Bokhari@MedicusLtd discusses the company’s effort to develop a localized, non-surgical treatment for people with Gorlin syndrome, and how the patch may induce tumor-cell death while limiting systemic exposure. @globalgenes.bsky.social #RARECast https://t.co/tf8zaWW0xG
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.
https://t.co/v8HAUh2LjE
@GileadSciences CMO Dietmar Berger on using targeted acquisitions to build platforms across solid tumors, autoimmune disease, and viral threats.
https://t.co/LsrT0xPzbW
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
https://t.co/u0Lklpd2DN
Half of ischemic stroke patients face poor outcomes. Revalesio’s Greg Archambeau discusses how the company’s oxygenated saline therapy, RNS60, may help protect the brain after stroke, reduce tissue damage, and change the economics of stroke care.
https://t.co/xJngeA1ggw