The Ledger, every show

“We still haven't seen, and I think it's coming, but it hasn't happened yet. We haven't seen artificial intelligence or other technologies massively compress the amount of time it takes us to do the clinical development, the clinical trials, the enrollment of p…”

“You know, in the case of treating a baby that had, you know, something like somatic muscular atrophy that would have otherwise died, a one and done gene therapy from Novartis, this is a therapy that's worth about, it's cost about 2.1 million dollars, effective…”

“And so if I had to bet, I would bet that in the next 10 years, we will have solved sickle cell anemia, much like we've solved cystic fibrosis, that had a very similar profile in terms of what we understood about the disease.”

“The FDA is looking to the skies and taking a page out of the FAA's approach to aviation safety, rigorous yet adaptive. Recently launching its own new office for therapeutic products and piloting Operation Warp Speed for rare disease to create more transparent …”

“The breaking news is that the first CRISPR therapy has been approved. This is a therapy for sickle cell anemia and beta thalassemia that was developed by a biopharmaceutical company called Vertex Pharmaceuticals, working with another company called CRISPR Ther…”

“It's barely been 10 years since the concept of CRISPR was discovered and described in the scientific literature. So we went from the initial discovery of CRISPR as a potential use as a medicine, all the way to an approval in just over 10 years. That is lightni…”

“And really what drove that wasn't some fundamental discovery on the biology or the science. What drove that was Illumina was able to apply Three very specific and distinct engineering disciplines and converge them. One was the use of microfluidics, the, just a…”

“And I think one of the things that we're seeing is that now when we can start to design cells, we can actually start to design cells that have logic, that will know where to go in the body, what to do when they encounter disease, and how to essentially termina…”

“A great data point that I heard recently was a partnership that Facebook made with the Michael J. Fox foundation to find ways to pilot the use of social networks to recruit patients for Parkinson's disease trial using social media. They were able to reduce the…”

“And as we can see in leukemia, as we can see in inherited forms of blindness, as we can see in PKU, these living medicines are finally leading us to where we've always wanted to be, which is to find cures.”

“And so when you add all of this up, it takes an incredible two and a half billion dollars and 10 to 15 years of investment to get the average drug to market when you account for all of the failures along the way.”

“Well, if we get really good at understanding biology, and we get really good at engineering biology, on the horizon, we have the ability to begin to program cells so that we can imbue them with logic. So we can take a cell and have it understand how to find di…”

“So Facebook, for example, as I mentioned earlier, is using their social network to help us identify and recruit patients for clinical trials. And in fact, they did a pilot project with the Michael J. Fox foundation where they were able to reduce the cost and t…”

“And that's not to say that hospitals will disappear altogether, but I think what's very surprising is the kinds of procedures and activities that we'll see in hospitals will be specialized to the things that are so incredibly complex, like surgeries that requi…”

“And I think one of the most surprising things we'll see is the emergence of new specialties that come from the use of technology in healthcare, much like radiologists, of course, came on the backs of the ability of the availability of x-ray technologies.”

“It's been fascinating to see the broad range of applications that you can do on, on an iPhone today, whether it's an EKG or to manage blood pressure, or you can do DNA sequencing even today.”

“Six million cases a year of diagnosed cancer in the United States in dogs.”

“I think one of the big things in synthetic biology has really been, you try 10,000 things to get one thing to work.”

“Today, because of improvements that were born of the application of engineered disciplines and three very specific ones, we can now sequence a human genome in what used to take 13 years in a matter of hours, and what used to cost three billion dollars for less…”

“One of the very interesting things about this particular space is these companies will look very tech-like in terms of their ability to create new markets and scale and dominate those markets, and those are obviously very attractive and interesting opportuniti…”

“As we move biology more and more into an engineer-based discipline, I think we're going to see more powerful modalities like CRISPR.”

“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…”

“Biology is no longer an industry in and of itself. It's something that's going to touch every single industry, and we're already seeing applications in energy, in textiles, in food, of course in health, and in data storage.”

“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…”

“And the first clinical trial to do this is already ongoing. A company called Synlogic has engineered a microbe that allows it to break down A, an amino acid in a disease called fetal, phenolketonuria, PKU, in patients that are unfortunately not able to break d…”

“Pharma companies are swimming in targets, right? The problem they're trying to solve for is how do I fill in my pipeline?”

“Today it accounts for about 20% of our GDP, and that number is going to double over the course of the next three decades.”

“As we move beyond software, we're at this point where we're seeing this incredible convergence between biology, computer science, and engineering. And it's this convergence that's going to give us broad new superpowers to understand and manipulate biology. Ess…”

“So we're living at this incredible point in time where biology is going to look a lot like information technology, and that it's going to be able to tackle and transform and perhaps even disrupt multiple industries.”

“What scientists were able to do is they were able to take this video, the pixels from this video, and using CRISPR can coax a bacterial cell to ingest the information and store it in its DNA. And on the right, what you're seeing Is scientists' ability to retri…”

“CRISPR is a concept barely registered just a few years ago, and now it's an indispensable tool for drug discovery, for, actually, for understanding biology, as we've seen the cycles of iteration in biology accelerate.”

“We'll see more generics approved this year than in any year in history previously.”

“And so those chemistry based medicines have served us in countless ways to treat and manage disease, but there's a limitation to what we can do with chemistry. So beyond developing our own medicines, we had to find ways to produce medicines that we couldn't ma…”

“But with advances in synthetic biology, advances in our ability to use things like gene and cell engineering, we're increasingly finding ways to make those same cells and genes and microbes, not the target of medicines, but the medicines themselves.”

“In the case of cancer, it's unfortunate that 40% of the men and women in this room will likely be diagnosed with a form of cancer somewhere in their lives, sometime in their lives.”

“And last year saw the approval of the first gene therapy for a treatment of a rare form of blindness.”

“In this world, it's one in 10,000 becomes a product.”

“So scientists at the Broad Institute at Harvard and MIT, they've created an atlas of various different cell types, so we can understand how different cells function and dysfunction.”

“The emergence of the biotechnology industry gave us the ability to coax organisms to make proteins for us, and as a result, seven of the top 10 selling drugs today are protein-based drugs.”

“Up to 40% of drugs that go through a phase three trial fail. 40%. This is after 10 years, a decade, and hundreds of millions of dollars invested.”

“These are averages of averages, of course, but on average, it could take anywhere between 10 to 15 years to develop a drug to get it to patients.”

“And specifically, I want to talk about our increasing ability to program living things to serve as medicines for us.”

“But for the most part, today, this is limited to leukemias.”

“So gene therapy has been the holy grail for treating the approximately 5000 known diseases that are caused by a single gene mutation.”

“The U.S. Biopharmaceutical industry spends about seventy five billion dollars every single year in research and development.”

“The market for drug sales in the United States alone is about a half a trillion dollars annually.”