00:03:40.040When you're with McKinsey, you know you have connections with everybody.
00:03:43.180So imagine the contact of, hey, we're trying to get this product into this McKinsey relationship.
00:03:48.840Countries, foreign, other companies, investments.
00:03:52.320It's the right lineage of opportunities for this company.
00:03:56.800And they've already, as of 2023, more than 200 people have been treated with experimental CRISPR therapies.
00:04:03.380I'll give you a couple of testimonies and stories here.
00:04:05.380First one is Victoria Gray, 37-year-old survivor of sickle cell disease, spoke about her transformative experience with gene editing treatment.
00:04:12.660When she described a childhood and adolescence marked by severe symptoms and dreams deferred, after receiving a transfusion of genetically edited cells from her bone marrow, which she calls supercells, she felt an immediate sense of rebirth and gradually saw significant health improvements over seven to eight months.
00:04:31.500Her story, marked by newfound enjoyment of life, deeply moved the audience, including typically stoic scientists.
00:04:38.220What's important to know is, some of maybe, so is this stem cells?
00:04:57.920In October 2021, CRISPR Therapeutics gave preliminary results for individuals with lymphomas who had been treated and followed for at least four weeks after treatment.
00:05:07.200Side effects were not severe, and the safety profile was superior to other CART products.
00:05:12.240In these patients, almost 60% showed a positive response to treatments, with 21% showing no signs of disease for six months after a single treatment.
00:05:45.240Team of researchers, led by Shukart Mitalipog, a genetic at Oregon Health and Science University in Portland, reported that human embryos carrying a mutation could be repaired using this method.
00:05:59.720The researchers generate embryos from a union between two cells, a sperm carrying a mutation that can make it harder for the heart to pump blood.
00:06:08.920But in an egg with a healthy version of the gene, the doctor and his team used CRISPR-Cas9 to cut the broken copy of the gene to see if the intact version would guide its repair.
00:06:21.040They reported the experiment a success and published it in the journal Nature.
00:06:27.280If you think about what it's saying, you go to the doctor, the doctor tells you your baby has a 70% chance of being Down syndrome.
00:06:35.520These guys can use CRISPR-Cas9 to cut the gene and edit it with the right gene so the kid will be okay, won't end up being Down syndrome.
00:06:47.740So this is personal to me because we've gone through it in our family.
00:06:51.020Think about the possibilities of what this could do.
00:06:53.560Again, you are playing God, but the capabilities are unbelievable.
00:06:58.380Here's another one for you, adverse results.
00:07:00.24027-year-old Terry Horgan was participating in a clinical trial for a CRISPR treatment aimed at Duchenne muscular dystrophy, tragically passed away.
00:07:10.500Duchenne muscular dystrophy is a fatal condition characterized by progressive muscle degeneration, the exact cause of Terry's death.
00:07:17.800And whether it was related to the CRISPR treatment remains unclear.
00:07:22.020By the way, the other negative adverse results of this could be the wrong arrangements of DNA.
00:07:27.260So imagine they're rearranging the DNA and all of a sudden the sequencing is off, which could lead to random mutations by error.
00:07:35.340You don't know what that's going to look like.
00:07:38.460So maybe a person that's willing to use this is on the last straw and they're saying, look, I'm willing to be tested for this because the alternative is I'm dead.
00:08:06.480The experiment was widely condemned as irresponsible and dangerous in large part because many of the ways in which CRISPR-Cas9 can affect cells remain poorly understood.
00:08:18.360The doctor was found guilty of conducting illegal medical practices in China and was sentenced to three years in prison.
00:08:24.800Apparently, he said these infants are built in a perfect way where they'll never have AIDS.
00:08:30.720How are you going to know if they're never going to have AIDS?
00:08:32.360This is the part where this technology in the wrong people's hands, what are they going to do with it?