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next up is Nicole Paul I'm really excited for this talk I think you guys will find a lot of optimism in where
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technology is playing out in um in biology and bioengineering to cure fundamental human disease
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for nearly 20 years she's been developing Next Generation aav platforms for Gene repair Gene transfer and Gene
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editing and chief directed Evolution for novel engineered capsids and comparative
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multiomic approaches to interrogate translational aav biology she's going to share a little bit about her work with
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her startup siren biotechnology which came out of stealth last year please join me in welcoming Dr Polk to the
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stage we'll let your winners ride Rain Man [Music] [Applause] we open source and they've just gone crazy
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[Music] all right quick show of hands how many of you have ever even heard of the
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phrase gene therapy or viral gene therapy before Oh My Gosh would you guys read in Wall Street Journal and The
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Economist all right pretty good usually it's like two hands um so who the heck am I why am I up here
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talking to you about this so like I said my name is Dr Nicole Paul come until like a hot minute ago I was a professor
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of viral gene therapy at UCSF in San Francisco and decided to spin out a company based off some work in my lab
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and I'm here to talk to you guys a little bit about using viruses as medicine so so what is gene therapy
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broadly speaking uh this is using viruses as medicines so historically we have used these to treat single Gene
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genetic disorders but we can use these much more broadly now and I'll share a little bit about that but historically
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we've used these when you were born either missing a gene in your genome or you had a mutation in a particular Gene
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in your genome and all you needed in order to be completely healthy was to have a functional copy of that Gene
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given back to you or to have um that the particular mutation in that Gene corrected and restored for you and
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we can do all of these with viruses and so if that sounds a little bit crazy and a little bit new and a little bit
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revolutionary it's because it is we're kind of quite literally living through um one of the most recent and kind of
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newest eras of modern medicine and so in order to talk about where we are we kind
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of need to understand where we came from so nearly every one of you in this room
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this first era of modern medicine started probably with your either your parents or your grandparents this is
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with chemical medicines this is when we realized that gosh every time I have a stomachache I can go eat this one
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particular Leaf from a tree and when I eat that leaf I feel much better and then the scientists realize
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well we don't have to eat the leaf we could isolate the chemical compound that comes from that leaf and then we could
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produce that in mass synthetically in large Vats in the lab and that way we don't have to all go outside and eat and
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eat leaves all the time um and so the Advent of chemical medicines and realizing that we could
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isolate these and synthesize these in Mass was an absolute transformation this is nearly every drug you've ever taken
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in your life these are all the pills you can go find at your local pharmacy uh particularly the ones that are over the
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counter and so this is the vast majority of medicines you've ever taken in your entire life
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and the way these chemical medicines work right you take that pillow you swallow it it goes into your stomach it
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gets dissolved by the acids in your stomach it then gets absorbed into the bloodstream in your GI tract and then
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goes throughout your entire body and it will bind to and affect kind of either the shape or the function or the ability
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of various proteins in your body in a way that's useful to reduce your symptoms and so then in the next kind of era of
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medicine we had the brilliant idea well let's just cut out the middle man let's not give you a chemical that alters the
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shape or function of a protein let's just give you the protein let's let's advance to protein medicines and let's
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now take a protein that has the particular shape in the function that you might need in order to provide some
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kind of therapeutic benefit for the disease state that you have and give you that as a drug and so that's kind of
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been the next kind of most recent wave of modern medicine and so this is things like enzyme treatments and antibody
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therapies many of you in the room have probably heard of the most successful drug of all time anyone know what it is
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anyone want to shout out a guess what's the most successful drug of all time that's raised the most money
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it's not Viagra guys Humira someone said it so this is an antibody that's used to treat a particular form
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of arthritis this antibody cells on average a little over 21 billion dollars worth of just
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that drug every single year and has been the most successful drug for the last 20
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years this is what we call a blockbuster this is what everyone is shooting for in
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their portfolio is a blockbuster drug and it's an antibody but now we're entering kind of this
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newest third modern uh form of medicine which is what we call living medicines this is using things like viruses
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bacteria cells things that are quote unquote alive that we can now use to go in and impart changes in your body why
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would we do that well both chemical medicines and protein medicines we typically either give orally or we give
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intravenously which means they're going to go throughout your entire body but in
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some cases we don't want a drug to go to your entire body like for example when you take chemo boy you feel like trash
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right it's because it's also affecting healthy cells in your body so sometimes we want the medicine to only go into a
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simple place in your body or a single place so that way you only experience the therapeutic effect in a single
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location the other really cool thing we can do with things like viruses and cells is we can engineer I know many of
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you here are like Tech or Tech adjacent we can engineer in logic circuits so we can add things like if then or and and
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all these types of kind of Boolean logic we can engineer those circuits into viruses and cells and have them perform
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those same types of decisions uh within your body like if you experience this release the drug If you experience this
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don't so you can engineer in these types of circuits in order to get this really
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really precise delivery of any particular medicine that you're interested in delivering and you can
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package almost anything in a virus and so a really common misconception is that all viruses are bad for you all
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viruses make you sick couldn't be farther from the truth the vast majority of viruses on the planet
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are very good at getting inside of you but very few of them make you sick I bet most of you can't name more than 20
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viruses and all the viruses you can name are viruses that make you sick right measles mumps small polio smallpox these
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types of things we only know of them because we study them because they make us sick but the vast majority of the
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viruses on the planet can actually kind of be considered our allies they're tools that we can use that don't make us
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sick but they're still very good at getting inside of us that we can use to deliver all kinds of medicines
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so all of this is talk much more fun to look at as an actual video of this in action and so what I'm going to show you
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on this screen is a patient from a clinical trial from a patient that has a rare form of
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inherited genetic blindness this is a disease called Libra's congenital amaurosis these patients are born
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basically medically blind they cannot see at Birth and you're going to see two videos the one on the left is going to
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be this patient pre-treatment attempting to navigate a maze you can see that maze
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on the floor at very very low light so the reason that this looks very yellow is because it's taken at one luminal
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unit or low light all of us have much poorer visual Acuity at low light than we do at highlight so it's not that this
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video is taken in 1985 it was just in very very low light that's why it looks yellow
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so I'm actually going to start that video and I'll kind of talk over this this patient while they're going so this
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patient's medically blind they cannot see the arrows on the ground that they're being told to navigate nor can
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they see the little obstacles that they're being told like you should step over so they're feeling for them with
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their feet just like you would if you were blind and you didn't have your walking stick you would just kind of
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feel in front of you to see what was there so that you wouldn't manage to trip and fall
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so I'm not actually going to show you this entire video because it took this poor patient 214 seconds to attempt this
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navigate this eight foot eight foot by eight foot maze and all this patient needed they were
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missing a single protein in the back of their eye all they needed was a single viral infusion for that virus to express
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a single protein that that patient was missing in their eye and this is that exact same patient
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again this was a 10 year old just one year after treatment they would have been able to do this within seven days
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though so they have absolutely perfect vision so we're able to see pure blindness
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this same young patient has now gone on to get their driver's license they are they are walking quite
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literally they are walking amongst us leading a completely normal life so this feels crazy and revolutionary like oh my
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gosh I hadn't heard about this when is this when is this coming for the rest of us and all of our indications
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soon where are we now this is a one-year-old Slide the next prospectus from Wells
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Fargo is expected to come out any day now I was hoping it would come out before this talk but it didn't but this
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is the annual prospectus they put out every year of kind of the advancement of these gene therapy companies they do
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this for every industry but this is for gene therapy so this is all of the logos
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of all of the companies that are working in gene therapy for various diseases the
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outer rings are kind of the earliest stages of development those phase one phase two trials and as you move towards
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the center towards the bullseye that's when you're FDA approved and you can sell your drug for hopefully 21 billion
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dollars a year and be a Blockbuster but as you can see right there is a wave of Gene therapies coming from so even those
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few of you who didn't raise your hand earlier that you had ever heard of gene therapy before I promise you in your
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lifetime no matter how old any of you are in this audience in your lifetime you are going to receive a viral gene
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therapy as medicine so there's a particular virus Dave mentioned it earlier that I like to work
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on this virus called aav and it's typically used like we were mentioning before to treat rare genetic disorders
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some of the disorders that we've been able to cure are the ones shown here on this slide
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and again we've typically only done rare single Gene disorders and in my lab at UCSF and now at the company we wanted to
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ask a little bit Bolder question typically we've only used these to treat rare genetic disorders could we use
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these to treat other indication spaces ones with many more patients where the need is much greater and the benefit to
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humanity would be much more so we wanted to ask a rather audacious question rather than having a virus that might
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cause cancer could you use a virus to cure cancer Could you actually use a virus as a medicine that could be used
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to treat cancer so we've been working on this for the last seven years to try to go against
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conventional gene therapy so every gene therapy on the planet all the ones on that Bullseye I just showed you on the
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last slide whether they're from Academia industry big Pharma doesn't matter every one of those is bespoke and
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personalized for a single indication so that virus that you make can only be used to treat that one disease and no
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other disease and every one of those viruses takes about 10 to 15 years to develop so this is very time intensive
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usually on average about two to three billion dollars so maybe not necessarily as expensive as the gentleman who just
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left the stage but a very very expensive technology nonetheless and typically treat very very small patient
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populations the definition of a rare disease in the United States is anything that occurs in fewer than 200 000
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patients a year but most of those Gene therapies are going after indications with like a hundred patients 200
00:11:09
patients so very very rare disorders and so we wanted to go after something that would help much more of humanity
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and so we wanted to ask the question could we make a single gene therapy that could be used to treat millions of
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patients across a variety of indication spaces things like cancer where they're often very similar to one another
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could you make a universal gene therapy is that even a thing and so we wanted to kind of set out on a
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fairly audacious project to not only bring viral gene therapy to the oncology world but to blend it with one of the
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newest forms of oncology which is this idea or this concept of immunotherapy many of you have maybe even heard of
00:11:46
this before there's a variety of different ways you can accomplish this they all essentially work the same way
00:11:52
where they retrain your immune system to fight cancer and I'm using that word retrain very purposefully
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every single one of you in this audience has a tumor you've had a tumor every single day of your life
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it's usually a single cell you often have many of them throughout your body your immune system is doing what's
00:12:12
called background cancer immunosurveillance your immune system is circulating throughout your body every
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single day and it's actually able to detect and find and sniff out where those tumors are and get rid of them why
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they are while they are still single cells how do they do this so that single tumor
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cell wherever it is in your body it sends out little chemical cues to the local environment as well as the immune
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system and the healthy cells that surround that tumor are also able to detect something's weird about that guy
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and they'll send a little message over to the immune system and they'll be like come over and do an investigation check
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him out what's up with him your immune system will come over sniff it out and be like yeah I agree
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something's up with him and they'll kill that cell and this is happening all the time
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throughout your entire body it's your immune system right now today your immune system knows how to fight
00:12:58
cancer it's Exquisite at it so how in the heck do you ever get a baseball-sized tumor how does that
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happen it happens because that individual tumor cell through a variety of different mechanisms there are many
00:13:07
ways this happens but typically somehow through one or two mechanisms it will end up basically randomly generating a
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mutation that will give it essentially an invisibility cloak that invisibility cloak makes it so that those neighboring
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healthy cells can't detect that something's up with it so they don't send a message to the immune system and
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because it's a little bit invisible your immune system also can't just like randomly swim by and detect it and see
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that something's wrong and so because it can't be seen it can't be destroyed and
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so then it starts to grow and become your big baseball size mass and then you become symptomatic right and then you go
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to the doctor but you can retrain your immune system to be able to see that invisible tumor
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and if you can do that with immunotherapy then now you've got a way to destroy a tumor that doesn't involve
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chemo that doesn't involve radiation but basically uses your own immune system to fight Cancers and so we wanted
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to blend the power of viral gene therapy and the Precision delivery and kind of those logic circuits around like do this
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when you experience this these types of things with immunotherapy to see if we couldn't do something
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important in the cancer space so we developed a platform a variety of different Universal Gene Therapies in my
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lab at UCSF and spun those out into the company and the name of the company is actually based off the mechanism of
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action here so we can take these viruses have them deliver a variety of different
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kind of cancer announcing payloads that will announce to immune system the cancer is here and so that way both the
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cancer cell as well as the neighboring cells will basically set off the fire alarm pull the fire in and let the
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immune system know this is where the tumor is come in and fight it and you'll have these three different waves of
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tumor killing that can happen both with your innate and your adaptive immune system
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so while we were still at UCSF we were like okay we have this really cool idea we want to try this but we're agnostic
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to any particular cancer type because again we weren't an oncology lab we were a viral gene therapy lab so we went over
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to our colleagues at the Cancer Center and we're like what's the gnarliest cancer what's the one that nothing works
00:15:03
we love a challenge tell us something that we can work on that will be interesting and they said go after brain
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cancer so we decided we'd start collaborating with Folks at UCSF to determine a brain cancer kind of path
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but how do you test a drug in order to make sure it works on human brain cancer when you can't test it yet
00:15:19
on humans because the FDA wants you to have a data package before you go up for your clinical trial
00:15:24
so the way you test it is you actually make mice with human brain cancer so you can take brain cancer samples from
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humans from biopsies and resections Transplant those into mice they'll grow a mini human brain tumor you can come in
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and now treat them with your human drug not your mouse drug your human drug to test if it works and look to see whether
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or not you get a response and this is a quick little sample from three Mice from a treatment group where
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you can see uh those big colored blobs or the tumors and the brains of these myosis is live non-invasive
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bioluminescent Imaging where the control treated mice that receive either a control virus or just receive sterile
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saline have massive tumors these mice die versus the mice that have been treated with our virus where we can
00:16:04
completely eliminate these tumors as you would imagine if you can eliminate the tumors with imaging that might just
00:16:09
correspond to improvements and life expectancy so this is the one and only graph that I'm showing you very simple
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it's a survival curve the line is vertical you died the line is horizontal you lived so we can massively improve
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the lifespans of these animals and for all intents and purposes cure these mice of brain cancer and we're cueing up for
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a clinical trial in 2025. so we're excited to start our clinical trial in 2025 and very quickly I'm going
00:16:40
to share with you guys maybe like 15 three little 15 second vignettes on what else could we do with viruses so we see
00:16:47
a gentleman here sleeping how many of you are short on sleep every one of you raise your hands come
00:16:53
on uh so there is a rare mutation that happens in patients these are people walking amongst us who have a mutation
00:16:58
in a gene called dec2 these patients again single mutation single Gene these patients only need four hours of sleep a
00:17:06
night to be completely rested the same way that we have the need for eight to nine hours of sleep a night
00:17:12
you interested in getting this gene therapy we could do this today we haven't yet but we could do this
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today another example most of us in the room pretty probably interested in longevity
00:17:23
um we could use viruses to regenerate the tissue in your in all of your joints maybe get rid of things like cellulite
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viruses brain fog all these types of things this is probably a few more years out where we're probably five to ten
00:17:36
years out on being ready to do this but this is absolutely coming in your lifetime you could get a Rejuvenation
00:17:41
gene therapy you really want to talk far out we could talk about maybe 30 years from now we
00:17:47
could absolutely engineer Humanity to withstand life in much more drastically harsh environments so we could alter
00:17:54
your skin color to be able to reflect more UV light so you wouldn't get as much damage if perhaps say you were
00:17:59
living on Mars we could engineer the cells in your immune or in your gut and the bacteria that live in your gut to be
00:18:05
able to metabolize foods that are more easily grown on a martian environment and these types of things so really your
00:18:12
imagination is the limit so could we enhance human potential we absolutely can it's just a matter of are you
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interested in when so happy to discuss the future foreign [Music] [Music] times yeah
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can you just um help everyone understand the timing and the regulatory hurdles um
00:18:56
viral gene therapy had a moment where there were setbacks and just share that with everyone how
00:19:03
that's affected how this technology has been held back how it's progressing now and what timelines generally look like
00:19:10
for getting these uh these uh breakthroughs to Market absolutely so today if you were to start
00:19:17
a brand new gene therapy company called siren biotechnology or anything else and
00:19:22
you wanted to develop a viral gene therapy and you wanted to get to Clinic you're still probably looking for that
00:19:27
first program in like a 10 to 15 year mark and it's not because it's going to take you that long to run the clinical
00:19:32
trials it's not because it's going to take you that long to grow up the Vats of the drug it's it's mostly going to be
00:19:38
a regulatory timeline so in between each one of these clinical trials even before
00:19:41
you do your very first ones I mean we're ready today we could start our brain cancer clinical trial today but we're
00:19:46
looking at 18 months of paperwork in order to file to do that first phase one and then you need to do your data
00:19:51
readouts in between each one of those trials there's this kind of massive amount of paperwork that goes in front
00:19:57
of the FDA that needs to be reviewed and there's back and forth and so the regulatory hurdles both from
00:20:03
if you weren't even doing anything you know really drastic like those last three vignettes that I talked about is
00:20:08
still going to be 10 to 15 years but if you wanted to propose something like the
00:20:12
Sleep one that I mentioned that I think we'd all I'm certainly interested in um there you're probably looking at an
00:20:17
ethical review board and whether or not they let you do something like that because that's not a disease so fun fact
00:20:23
the FDA only lets you queue up clinical trials to test drugs that are for diseases but aging is not a disease
00:20:32
state wanting to sleep fewer hours is not a disease State insomnia is but wanting to
00:20:40
just sleep fewer hours and be fully rested is not a disease state so we kind of have to get to this moment where
00:20:47
does the FDA start changing their definition of disease do we have a new regulatory body who will review these
00:20:54
types of things that are more like augmentations and enhancements how are we going to Grapple with this as a
00:20:59
society are we are we okay with this and let me just ask one more question it's a
00:21:05
great it's a great question you pose because there is increasing consensus that maybe we
00:21:10
should think about aging itself as a disease obviously and that there's a lot of approaches now to addressing that
00:21:16
what about the other challenges to completing the research and the trials and scalability what are the bottlenecks
00:21:24
we've talked about I've talked on our show about car T therapy and the challenge and getting manufacturing
00:21:30
scaled up to treat enough patients each year even though the Technologies here it's FDA approved those drugs are in
00:21:35
Market we just can't make enough what are the chat and I've heard a lot about uh lentovirus production and all these
00:21:41
other kind of viral vectors being 18 month delay super bottleneck how bottleneck are we in being able to do
00:21:47
the research that you guys are doing and ultimately to get these products to Market make enough of the stuff to treat
00:21:52
patients so the single biggest bottleneck is actually not technological at all it's Financial it's access to
00:21:57
Capital um right so these are like I said very very Capital intensive the average viral
00:22:02
gene therapy is about two to three billion dollars that's about three times more than making one of those chemical
00:22:06
medicines some of that has to do with time how long that process takes right every year your company is operating uh
00:22:13
that GNA oh it's expensive um it adds up really fast and so some of this is just access to Capital and
00:22:19
there's right the any company every one of us in this room this has a company there's depending on your sector there's
00:22:24
a valley of death for some of you it's very very early stage for some of you it's a little bit more later stage but
00:22:28
there's a valley of death where you just can't access Capital before you hit this
00:22:31
like big de-risking milestone where the bigger checks will come in and private equity and those types of groups and
00:22:36
crossover funds in your IPO so there's a value of death for many of these early stage companies where usually either
00:22:42
after your seed stage or your series a but before you've got clinical trial readouts where people are very nervous
00:22:47
to give you a really big check so that's certainly the biggest hurdle right now and why you see many companies going
00:22:52
under across the biotech space regardless of if they're selling Gene therapies and then from the
00:22:56
technological side it's still manufacturing is just like the drum beat being able to manufacture these things
00:23:02
at scale continues to be a challenge we need folks who come from like the mechanical engineering and these types
00:23:09
of backgrounds who don't historically necessarily think of Biotech as a place where they could apply their their
00:23:15
knowledge and Technologies to please come over to biotech and help us make you know new generations of bioreactors
00:23:22
and types of things that will allow us to produce these at scale cleanly cheaply and easily and it's just not
00:23:29
intuitive making chemicals at scale is something that we've been doing for 40 years so we're really good at it genenta
00:23:35
can make kilos of drugs in an afternoon with a robot but viruses are still something like if I queue up the biggest
00:23:42
cdmo in the world catalent Pharma and ask them to make us a 5 000 liter bioreactor a virus they'll
00:23:48
absolutely say yes but they won't even touch it for two and a half years um because I wanted to double click on
00:23:54
the concept of making life better for people who aren't sick the Marty Seligman from
00:24:01
the American Psychological Association actually posed this question 20 years ago and he said well we try to make all
00:24:07
of psychology and Psychiatry is taking depressed people and anxious people and making them less depressed and anxious
00:24:14
what about meaning and fulfillment for people who are content what about more joy and so when you look at the field
00:24:20
and it's amazing the progress you're making and thank you for doing all that work aside from hey can we sleep less
00:24:27
what else is you know do you do you talk about wow if we had the mandate to take
00:24:33
healthy people and and give them a gift of augmenting them making them into superheroes almost when we're talking
00:24:39
about like X-Men mutations here what are the fun things that you dream about that
00:24:45
you could do for Humanity could we all just have Vision like Hawks or oh night vision is totally possible night vision
00:24:51
yeah easy not literally without the goggles yeah easy I mean a world in which that's just
00:24:59
an FDA like I need a I need the green light but no that's easy so okay that feels like Professor X kind
00:25:06
of level I like it what else you got keep going um night vision would be possible the
00:25:13
ability to metabolize um you know a food that grows perhaps in a different environment that not only
00:25:19
you can't get any nutrients from but you can't even absorb it um so we could make
00:25:23
it so that you could you know pretend pretend Soylent tasted good um you know uh you could eat anything
00:25:30
and not only could it taste good to you but it could provide you all the nutrients that you need and be able to
00:25:35
grow with you know much less much less water and in the types of environments that we'll have in in the future planets
00:25:40
that you know given the climate change challenges that we all have um as far as other augmentations I mean
00:25:45
there's there's all of the obvious Beauty applications uh that I think all of us are already doing uh now this we
00:25:52
would just be doing this with a different modality right so removing you know there's a there's a gene therapy
00:25:56
company in San Diego working on male pattern baldness uh there are folks working on you know all the wrinkles and
00:26:01
cellulite and you know getting your hair color back and like all of the normal Beauty things that you would expect
00:26:05
that's absolutely coming but as far as beyond the obvious Beauty things like what other types of augmentations I
00:26:10
think the mental Health space is a huge one because it's it's usually a very very simple thing and it's complex but
00:26:17
it's simple they're usually missing a single receptor or a single protein somewhere in their brain and if we were
00:26:23
just able to restore that for them in just that location and not everywhere in their body
00:26:28
um you could restore Joy you could restore happiness all of these types of things do you want to talk about the
00:26:34
capital markets piece because you mentioned you need Capital yeah we're fundraising hi yeah and uh tomorrow
00:26:41
maybe maybe you can help out here but you know the biotech Market's been decimated oh yeah since November of 21
00:26:47
and it's really intricately linked to interest rates because of how far out you have to make a bet on a biotech
00:26:54
company and so as a result the valuations have plummeted 80 90 percent in many cases a lot of stuff just not
00:26:59
getting funded right now um maybe tomorrow you can share your point of view on where things are headed
00:27:05
there and um Nicole maybe you can share a little bit about what the experience has been fundraising even though you
00:27:10
have a great technology that's got some proof points how hard it is well I I have a I have a kind of a technical
00:27:15
question but which is just you had to when you picked aav you had to make a decision about small
00:27:22
edits and not large edits I guess right so just explain to these guys about what
00:27:27
payloads are actually possible in AV and are you thinking about what happens if you actually have to go beyond four KB
00:27:34
and how are you going to solve that so because a lot of the diseases that these guys will actually probably
00:27:39
understand are not small Snips they're going to be large you know cut and paste two types of gene therapy
00:27:45
there's Gene transfer gene therapy where we give you the whole Gene that you were
00:27:49
missing like perhaps you were missing that portion of your chromosome at Birth so that's Gene transfer gene therapy
00:27:53
we're literally transferring a functional copy of the gene back to you and then there's Gene editing gene
00:27:57
therapy where we go in and actually correct a mutation in place uh within your genome and don't give you anything
00:28:02
extra so every virus on the planet has a carrying capacity it has a size just like a sedan can't carry as much as a
00:28:10
minivan and so aav viruses the viruses that I work on are amongst one of the smaller viruses so it can only package
00:28:16
about 4.75 KB so the numbers of nucleotides that you can fit inside of it and different viruses have larger
00:28:21
packaging capacities and can fit more studs that's 4700 letters of DNA and so different viruses will have different
00:28:28
packaging capacities and so if you're doing Gene transfer gene therapy where you need to provide the entire copy of
00:28:33
the gene you can only put in genes that will fit with all of the other regulatory elements for expression
00:28:38
within that virus so of all protein coding genes since we're typically going after diseases that are in influencing a
00:28:46
protein of all protein coding genes eighty percent of those genes can fit within my virus so actually the vast
00:28:50
majority of genetic diseases can be treated with aav gene therapy with Gene transfer gene therapy with Gene editing
00:28:56
gene therapy we can treat anything because now we're not size limited we only need to deliver either the knicker
00:29:01
or the cutter right many of you have heard of things like crispr that's one of the many tools we can use to Nick or
00:29:06
cut DNA and we can either make a Nick and have your body repair the mutation we can go in and make a Nick or a cut
00:29:13
and we can cut something out that wasn't supposed to be there or we can go in and
00:29:18
Nick or cut something in your genome cut something out and then put something that was meant to be there the whole
00:29:23
time back in all three of those forms those Gene repairs and Gene edits are all kind of broadly classified as a lot
00:29:29
of Gene editing gene therapy and all of those would fit within a lot of any virus there's a high percentage of
00:29:34
disease I don't know the percentage you probably know which is really just like one point mutation right yeah huge
00:29:40
transcribe AC or something is typically the biggest one right can you just explain to folks in when you think the
00:29:46
tool chain will exist for us to do those single point edits and actually just today you can actually go in and
00:29:52
accurately rewrite an a to a c okay today there's a yeah there's a few in yeah there yeah
00:30:00
um guys we got to wrap and I want to really thank Dr Nicole Paulk for her amazing work
00:30:06
um fantastic thank you so much thank you thank you thank you [Music] [Applause] [Music]
00:30:23
rain man [Music] we open source it to the fans and they've just gone crazy [Music]
00:30:37
I tried