
Onyx @ Union Square: Shankar Musunuri, Ocugen
What if the key to curing blindness wasn't targeting individual genes, but entire genetic networks?
What if the key to curing blindness wasn't targeting individual genes, but entire genetic networks?
Lexicon's latest drug for obesity and weight management has a novel mechanism and is backed by Novo Nordisk.
Neil Warma explains how Mongoose Bio is pioneering TCR-T therapies that may last longer, tackle more cancers, and require less from patients.
Harnessing Nobel Prize-winning insights on telomeres, Geron’s CEO Dr. John A. Scarlett is taking on some of hematology’s toughest challenges—and winning FDA backing in the process.
“Almost all of the data that we use comes from our own laboratories - data generation doing real experiments in human biology… The AI side of things will be commoditized eventually, and the winners will be those who have the strongest data moat in life sciences.”
A Grammy-winning CEO is taking gene therapy to new heights.
A recent multi-billion dollar deal with Novo Nordisk in diabetes and obesity, and an injection of significant funding through a $200 million government partnership - Aspect is worth watching as they enter 2025.
By targeting toxic protein clumps in the brain, Dr. Martin Tolar, founder-CEO of Alzheon, aims to outsmart Alzheimer’s decades ahead of symptoms - and reshape global neurology.
Ron Cooper, ex-Albireo CEO, is betting on enGene’s novel gene delivery platform to sidestep BCG shortfalls and transform bladder cancer treatment worldwide.
SpyGlass Pharma is poised to revolutionize glaucoma treatment by implanting an innovative long-term drug delivery system directly inside the eye. Their treatment promises to continuously release eyedrop medication for 3 years after surgery, as opposed to using eye drops manually every day.
GERD affects 65 million Americans, yet innovation in treatment had stagnated. Phathom Pharmaceuticals' FDA approval of VOQUEZNA® (vonoprazan) is a big moment for patients.
If left untreated, Stargardt disease and Geographic Atrophy almost always lead to vision loss and blindness - but a groundbreaking oral treatment in the last stage of clinical trials offers new hope.
“I think I'm blessed with truly the best team in all of pharma… we are doing something that's bigger than all of us!”
“The cost of gene therapies can reach millions of dollars per patient… Our goal is developing cost-effective treatments that can be accessible globally.”
"One of our first patients was treated over five years ago. He had received 109 injections over 10 years, and five years after a single Ixo-Vec injection, he is free of injections with stable vision and no fluid in the eye."
CAR T-cell therapies are often lauded as one of the industry’s most important frontiers, and these therapies have seen amazing success in treating hematological cancers — will autoimmune diseases yield next?