The people
The team turning a rare disease into a curable one.
Rare To Scale brings together a builder who funds the impossible, the scientist who knows Delaney's disease better than anyone, and a gene-editing pioneer shaping the tools that can one day fix it.
RM
Ross McCrayFounder, McCray Foundation
mccray.org
A father of four who built VideoAmp into a $1.7B+ company, then pledged the majority of his wealth to science through the McCray Foundation. He founded Thesis, which builds autonomous wet labs and computational biology to accelerate discoveries, the engine that can make one-of-a-kind cures possible for children the system overlooks.
Dr. Bruce GelbGogel Family Chair · Director, Mindich Child Health & Development Institute
Icahn School of Medicine at Mount Sinai
A world authority on Noonan syndrome and the RASopathies, the very family of disorders Delaney was born with. His lab uses patient-derived stem cells to model rare heart conditions and hunt for therapies where none yet exist.
Kiran Musunuru, M.D., Ph.D.Barry J. Gertz Professor · Co-Director, Penn Medicine/CHOP Orphan Disease Center
Perelman School of Medicine, University of Pennsylvania
A pioneer of CRISPR gene-editing therapies for cardiovascular disease, working to turn a single corrective edit into a lifelong cure, and to extend the same toolkit to rare diseases once deemed too small to treat.
BB
Dr. Barry ByrneDirector, Powell Gene Therapy Center
University of Florida
A pediatric cardiologist who leads one of the most experienced gene therapy programs in the world, carrying corrective genes into the cells that need them. His team has treated more patients with this approach than any other program in the United States or Europe.
Dr. Kristen WigbyClinical Geneticist · Precision Medicine Clinic
Rady Children's Hospital-San Diego
A clinical geneticist and dysmorphologist who co-founded and directs Rady Children's Precision Medicine Clinic. Her work turns rapid genome sequencing into an actionable diagnosis, the first step toward a therapy that has never been made before.
Dr. Matt BockPediatric Cardiologist · Medical Director, Heart Failure & Transplantation
Rady Children's Hospital-San Diego
A pediatric cardiologist who leads the heart failure and transplantation program at Rady Children's Hospital-San Diego, caring for children whose heart muscle has grown too thick to pump safely.
Ryan J. Taft, Ph.D.Chief Scientific Officer · Head of Precision Medicine, EspeRare
Genetic Alliance
A geneticist who leads global programs expanding access to advanced genomic diagnostics and tailored therapies for children with rare and undiagnosed diseases. He is the scientific founder of iHope, a worldwide network bringing clinical genome sequencing to patients who would otherwise go untested, and leads a consortium delivering custom antisense therapies to children outside the United States. Previously he held senior genomics leadership roles at Illumina and Tempus AI.
Robyn Saskia Cortese, PharmDPediatric Clinical Pharmacist · Co-Founder
Rubinstein-Taybi Syndrome Foundation
A pediatric clinical pharmacist, Air Force veteran, and rare disease advocate who co-founded the Rubinstein-Taybi Syndrome Foundation with two fellow RTS mothers. Through her clinical practice and advocacy, she works to connect science with the needs and priorities of rare disease families.
Jessica BeatusDoing Well by Doing Good
doingwellbydoinggood.agency
Jessica helps businesses and non-profits succeed through purpose-driven strategies and partnerships, with 15 years of experience across media, tech, and social impact.
