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what's up everyone and welcome back if you are new to the channel my name is chiraglatya hope you had a great holiday and a restful break today we're going to be talking about gene editing and gene therapy this topic peaked my interest when i read an article earlier this week about kathy wood the ceo of arkhanvest saying that the next fang stocks are in the genomic age and genomic stocks will drive returns for investment portfolios and innovation etf over the next five years first we're going to do a super basic high level crash course on gene therapy then we're going to talk about the applications of gene therapy as well as their impact to the broader healthcare industry and lastly i will give my business perspective on gene therapy and my thoughts as some random guy on the internet so before we begin please make sure to hit that like button if you haven't already and please subscribe before we talk about gene therapy we have to start with the human cell cells are the basic building blocks of all living things and the human body has trillions of them within our cells there is a nucleus and this nucleus has thousands of genes which provide the information for the production of specific proteins and enzymes that make muscles bones and blood which in turn support most of our body's functions such as digestion making energy and growing genes are segments of dna that produce proteins that perform a large number of different functions within the body there are many diseases today that are caused by genetics meaning that mutated genes provide incorrect instructions that cause a cell to either create an abnormal protein or in some cases not make the protein at all in gene therapy a copy of the correct gene is delivered to the patient's cells these new modified genes can then start to produce correct proteins that can potentially restore function the biggest question on scientists minds is how to actually deliver and target this therapy effectively to the human cell it turns out the body makes it very difficult for something to enter which is normally a really good thing but in this case makes the job of the scientists very difficult scientists have used things like viral vectors to do this and most recently crispr has emerged as a gene editing technology that can revise remove and replace dna in a highly targeted and specific manner okay so now you know about gene editing and gene therapy so now we're going to dive into some of the fun stuff why do people care so much about gene therapy there are over 6 000 known genetic disorders and more being discovered every day only around 600 of them are treatable additionally 10 percent of all adults have some sort of genetic defect so before you get too spooked i will caveat that most genetic defects are minor and not cause for worry now that you see how big gene therapy could be what is the goal gene therapy attempts to answer the question what if we could fix a patient's own cells and cure their disease once and for all instead of treating a chronic illness over their lifetime while gene therapy is very exciting there are a lot of remaining questions although it is a potential one-time cure for chronic illness it's still very much in development gene therapy has been around since the 1980s and aside from a few specific diseases we haven't quite nailed it yet gene therapy promises that it could potentially be long lasting but that remains to be seen it is also currently way too costly it is my hope that this will go down as the technology matures some scientists see gene therapy as a way to cure disease and others take it one step further and see gene therapy as a way to enable cells to do things that they weren't originally designed to do perhaps enabling new functions like identifying and attacking cancer there are however a lot of ethical discussions and concerns around this so what are the low-hanging fruit for gene therapy well any disease that can be traced to a single gene may be easier to fix there are however multi-gene disorders like heart disease and arthritis that will be harder and more complicated to fix secondly scientists are using gene therapy first to restore functions before thinking about enabling new functions okay so now you know about gene therapy how impactful it can be and why everyone's talking about it let's talk about the business perspective gene therapy has been around since the 1980s but hasn't really picked up steam until recently investors today are interested because new technologies like crispr dna sequencing and ai have emerged that might accelerate development my thoughts here are that while it may be exciting to compare the genomic space to fang facebook apple amazon netflix google we know that in healthcare you simply cannot move fast and break things like tech we must make sure that the development results in safe and effective therapeutics even if that means taking a little extra time i want to switch gears here okay so say you want to invest in a gene therapy company how do you determine how large the opportunity truly is to do this we must size the market this is a good way to see how many lives your treatment would impact i like to think of it as a funnel so starting from the top of the funnel you look at the overall population for example the u.s is approximately 330 million then you look at scientific epidemiology studies to see the disease prevalence these studies will tell you how many people actually have the disease then you have to determine once someone has the disease what is the chance they actually go into the doctor's office for a diagnosis and further what is the chance that they actually receive a treatment now that these patients have gone through this metaphorical funnel they land in what i like to call a cohort of eligible patients you have to consider now that in traditional therapy that is not curative this cohort of eligible patients will always be there but in gene therapy once a patient is cured they would be removed from this cohort and would no longer be eligible for treatment if you've been following along with me so far i want to show you one really advanced slide people don't understand how to value gene therapy right now because it's a one and done curative treatment it's completely different than other treatments that are available at the moment this is a big issue when you look at the payer space around the world a payer is a company that pays for an administered medical service an insurance company is the most common type of payer a payer is responsible for processing patient eligibility enrollment claims and payment when you have a new treatment you have to prove that it has some value to payers so that they will accept it payers around the world have different ways of determining value i won't talk through this slide because i want to keep things short and sweet but you can pause the video here if you want to read in more detail with that said thank you so much for watching i had a lot to say in this video but i had to cut a lot out to keep things simple comment down below if you have any questions or thumbs up if you want a part 2 on this i'll read all your comments and reply in the description i've added definitions and sources if you want to read further i've also placed a few links down below with my favorite services that i use on a daily basis and if you want to support the channel you can click through those i'm looking to grow this channel to 1 000 subscribers in the next year so please subscribe if you haven't yet with that being said thank you so much for watching and see you soon
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