Gene Therapy
topic on 10 shows · 20 statements across 18 episodes
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20 statements about Gene Therapy, every show
Hodak: Science's in-house retinal gene therapy probably enters human trials next year
“What we ended up doing is we developed indigenously a state of the art gene therapy, which is probably going to humans next year.”
Biotech must rebrand viruses for gene therapy treatments to succeed
“If we're going to get advances in viruses that deliver gene therapies or medical treatments, we got to rebrand virus. We got to come up with a new word, just like GLP one peptide that felt very safe.”
Chan: Self-Organized Patient Cohorts Advanced Gene Therapy in Under Five Years
“There's gene therapy that one disease group has moved forward over the course of, like, I want to say, like, three to five years rather than decades, and the speed is so fast. Because the patients themselves have organized the resources that a scientist or a c…”
Hodak: Optogenetic BCI approaches rely on risky, irreversible gene therapies
“We also don't need to genetically modify the, like, your brain. Some of the other ideas out there, for example, using optogenetics or things like ultrasound, this requires using a gene therapy to genetically modify the neurons in your brain, which First of all…”
Hodak: Million-dollar blindness gene therapy only marginally slows vision loss for 3%
“There's a gene therapy that's on market that costs almost a million dollars a patient, and it doesn't, first of all, it's only applicable for, like, three percent of patients that have this type of blindness, and it also just doesn't really work. Like, it very…”
Ricks: Lilly is considering SaaS-style recurring pricing for common gene therapies
“It's conceivable that one could create like a licensing concept, stealing from the SAS model, where you say, we'll do the procedure for free. And as long as it's working for you will deposit X amount in our bank account, and you're getting the value, and we're…”
Dave Ricks: CAR-T and gene therapies are difficult to scale to millions
“Those two techniques, CAR T and gene therapy, it's hard to think of like super scaled millions of people benefiting.”
Gil: Gene therapy is bottlenecked by delivery, not CRISPR editing
“When people talk about gene delivery, there's like three or four aspects of it that matter, and everybody focuses now on sort of the CRISPR side of it, which is, can you modify a gene? In a cell versus can you target it to the right cells? Will your immune sys…”
Diamandis: Anti-aging gene therapies will become cheap and globally accessible
“Yes, in the beginning when it doesn't work so well, it'll be paid for by the wealthiest and it'll be expensive. And then as it goes into production, it will be available for everybody. It will work extraordinarily well and it'll be cheap.”
Gene therapy delivery platforms enable modular repurposing across different diseases
“If we're able to make a medicine that does that, that can deliver one gene to a given cell type, it becomes increasingly likely that we'll be able to deliver a different gene to a different cell type for a different disease.”
Traditional gene therapies take 10-15 years and up to $3B to develop
“So every gene therapy on the planet, all the ones on that bullseye I just showed you on the last slide, whether they're from academia, industry, big pharma, doesn't matter. Every one of those is bespoke and personalized for a single indication, so that virus t…”
Acker: Muscular dystrophy gene therapy could potentially launch by mid-2023
“There's been no disease-modifying therapy, and now we have a gene therapy that could be launching potentially by the middle of 2023.”
Klausner: Cellular Rejuvenation Will Face Dangers Like Early Gene Therapy
“Actually it's going to be, you know, like with early gene therapy, there'll be surprising dangers and pitfalls. It's all about understanding how do you think about the deep scientific questions and investing in that.”
Andy Tran: AAVs are the only clinically approved gene therapy vectors
“To jump in, probably the natural place to start is on the AV side, as these adeno-associated viruses are the only clinically approved vector so far for gene therapy.”
Chang: Gene therapy modalities create moats providing sustained cash flows
“If you actually think about gene therapy and new technologies, they are creating the moat. Meaning, for example, Avexis and SMA, they are the, probably the leading gene therapy program out there, and they want to continue building that franchise and have not o…”
Novartis spent $15 billion on gene therapy acquisitions in 2018
“We've done fifteen billion dollars of acquisitions just last year in the space, not, not including all of our internal work in each of these areas.”
Conde: FDA advisory panels passed gene therapy and CAR-T unanimously 13-0
“Gene therapy, an area that historically has been considered to be very risky, has been shown to be so effective in this form of treating inheritable form of blindness, That that passed the FDA panel recommendation unanimously, a 13 to zero vote. CAR T similarl…”
Around 5,000 known diseases are caused by single-gene mutations
“So gene therapy has been the holy grail for treating the approximately 5000 known diseases that are caused by a single gene mutation.”
The first approved gene therapy treated a rare form of blindness
“And last year saw the approval of the first gene therapy for a treatment of a rare form of blindness.”
Precise editing and better delivery will expand gene therapy applications
“So as we advance our ability to deliver gene therapy, as we advance our ability to actually very precisely edit the genes, and only the genes that we need to edit, gene therapy will become applicable to a far broader range of diseases, not just limited to thos…”