In the future, parents may have the option to genetically engineer their children, and now is the time to discuss how this powerful technology should (or should not) be used.
Most people don't recognize how significantly and soon the genetic revolution will transform healthcare, the way we make babies, and the nature of the babies we make. The press release below is a thought experiment today. Within a decade, it won't be. * * *
Genomix Launches uDarwin, a New Business to Help Parents Optimize the Health, Well-Being, and Beneficial Traits of their Future Offspring
NEW YORK, July 29, 2029 /PRMediawire/ -- Genomix, a Caribbean-based health and wellness company, today announced the launch of uDarwin, a discrete, confidential service helping parents select and edit the pre-implanted embryos of their future children.
"Our mission is to help prospective parents realize their dream of parenthood in the safest manner possible while helping them optimize their future children's potential."
"We often fetishize nature," said Genomix Medical Director and Co-Founder Dr. Noam Heller, "but the traditional process of conception through sex confers risks on future children that can be significantly reduced through the careful and safe application of powerful new technologies."
Approximately three percent of all children are born with some type of harmful genetic mutation. Through its patented process of extracting eggs from the prospective mother, fertilizing these eggs with sperm from the intended father or from one of the superstar donor samples in the proprietary uDarwin gene bank, and screening up to twenty of these embryos prior to implantation, this risk can be brought down to under one percent.
"Having a baby is the most intimate and important experience in most people's lives," said Genomix CEO and co-founder Rich Azadian. "Our mission is to help prospective parents realize their dream of parenthood in the safest manner possible while helping them optimize their future children's potential."
In addition to screening pre-implanted embryos to significantly reduce disease risk, uDarwin uses its proprietary algorithm for the "polygenic scoring" of embryos to directionally predict potential future attributes including healthspan, height, IQ, personality style, and other complex genetic traits. Attributes once accepted as being the result of fate or chance can now increasingly be selected by parents from among their own natural embryos using this entirely safe process.
A premium product offering, uDarwin+, provides parents the opportunity to make up to three single gene mutations to their selected embryo to reduce a risk or confer a particular benefit. Among the most popular options for this service include increased resistance to HIV and other viruses, a greater ability to build muscle mass, and enhanced cognition. Additional edits will be made available as the science of human genome editing further advances.
Jamie Metzl's new book, Hacking Darwin: Genetic Engineering and the Future of Humanity, explores how the genetic revolution is transforming our healthcare, the way we make babies, and the nature of and babies we make, what this means for each of us, and what we must all do now to prepare for what's coming.
"uDarwin is proud to be the first company in the world offering the highest level of reproductive choice to parents," Mr. Azadian continued. "Genetic technologies are allowing us for the first time to crack the code of our health and identity. As pioneers in applying the most advanced genetic technologies to human reproduction, we recognize that prospective parents' desire for the services we offer exceeds societal levels of comfort with this technology. Our highest levels of customer service, comfort, and confidentiality ensure parents can secure massive benefits for their future children while avoiding unnecessary attention or any compromise of privacy."
All uDarwin services will be carried out in the company's state-of-the-art clinic aboard a super-luxury 500-foot yacht operating in international waters. After applying on the secure uDarwin website and gaining approval, clients are provided a date, time, and location to meet a company representative at a conveniently located Caribbean marina from where they will be shuttled to the uDarwin clinic. "Pioneers have always traveled beyond boundaries to create new possibilities," Mr. Azadian added. "Conceiving a child in a location where it can receive the greatest benefits of advanced science is no different."
"Pioneers have always traveled beyond boundaries to create new possibilities."
The cost of the basic uDawin service is $5 million, with half paid up front and half paid following the successful birth of a baby. Charges for uDarwin+, premium sperm or egg donors, surrogates, and other services are additional. "uDarwin is not for everyone," Mr. Azadian said, "but most parents of significant means understand that the benefits of optimal genetics far exceed almost any monetary cost."
"The genetic revolution has already begun," Medical Director Heller added. "The question for prospective parents is whether they want to be the last parents who left the health and identity of their future children to chance or the first to give their future children the greatest chance of optimal health and maximal fulfillment in the new reality that will arrive far sooner than most people appreciate."
If you could genetically alter your future children, would you? https://t.co/N0tqwX4Qd3— leapsmag (@leapsmag) 1564426548.0
As gene therapies and small molecule drugs are being studied in clinical trials, companies increasingly see the value in hiring patients to help explain the potential benefits.
Biomedical corporations and government research agencies are now incorporating patient advocacy more than ever, says Alice Lara, president and CEO of the Sudden Arrhythmia Death Syndromes Foundation in Salt Lake City, Utah. These organizations have seen the effectiveness of including patient voices to communicate and exemplify the benefits that key academic research institutions have shown in their medical studies.
“From our side of the aisle,” Lara says, “what we know as patient advocacy organizations is that educated patients do a lot better. They have a better course in their therapy and their condition, and understanding the genetics is important because all of our conditions are genetic.”
Founded in 2016, Tenaya is advancing gene therapies and small molecule drugs in clinical trials for both prevalent and rare forms of heart disease, says Ali, the CEO.
The firm's first small molecule, now in a Phase 1 clinical trial, is intended to treat heart failure with preserved ejection fraction, where the amount of blood pumped by the heart is reduced due to the heart chambers becoming weak or stiff. The condition accounts for half or more of all heart failure in the U.S., according to Ali, and is growing quickly because it's closely associated with diabetes. It’s also linked with metabolic syndrome, or a cluster of conditions including high blood pressure, high blood sugar, excess body fat around the waist, and abnormal cholesterol levels.
“We have a novel molecule that is first in class and, to our knowledge, best in class to tackle that, so we’re very excited about the clinical trial,” Ali says.
The first phase of the trial is being performed with healthy participants, rather than people with the disease, to establish safety and tolerability. The researchers can also look for the drug in blood samples, which could tell them whether it's reaching its target. Ali estimates that, if the company can establish safety and that it engages the right parts of the body, it will likely begin dosing patients with the disease in 2024.
Tenaya’s therapy delivers a healthy copy of the gene so that it makes a copy of the protein missing from the patients' hearts because of their mutation. The study will start with adult patients, then pivot potentially to children and even newborns, Ali says, “where there is an even greater unmet need because the disease progresses so fast that they have no options.”
Although this work still has a long way to go, Ali is excited about the potential because the gene therapy achieved positive results in the preclinical mouse trial. This animal trial demonstrated that the treatment reduced enlarged hearts, reversed electrophysiological abnormalities, and improved the functioning of the heart by increasing the ejection fraction after the single-dose of gene therapy. That measurement remained stable to the end of the animals’ lives, roughly 18 months, Ali says.
He’s also energized by the fact that heart disease has “taken a page out of the oncology playbook” by leveraging genetic research to develop more precise and targeted drugs and gene therapies.
“Now we are talking about a potential cure of a disease for which there was no cure and using a very novel concept,” says Melind Desai of the Cleveland Clinic.
Tenaya’s second program focuses on developing a gene therapy to mitigate the leading cause of hypertrophic cardiomyopathy through a specific gene called MYPBC3. The disease affects approximately 600,000 patients in the U.S. This particular genetic form, Ali explains, affects about 115,000 in the U.S. alone, so it is considered a rare disease.
“There are infants who are dying within the first weeks to months of life as a result of this mutation,” he says. “There are also adults who start having symptoms in their 20s, 30s and 40s with early morbidity and mortality.” Tenaya plans to apply before the end of this year to get the FDA’s approval to administer an investigational drug for this disease humans. If approved, the company will begin to dose patients in 2023.
“We now understand the genetics of the heart much better,” he says. “We now understand the leading genetic causes of hypertrophic myopathy, dilated cardiomyopathy and others, so that gives us the ability to take these large populations and stratify them rationally into subpopulations.”
Melind Desai, MD, who directs Cleveland Clinic’s Hypertrophic Cardiomyopathy Center, says that the goal of Tenaya’s second clinical study is to help improve the basic cardiac structure in patients with hypertrophic cardiomyopathy related to the MYPBC3 mutation.
“Now we are talking about a potential cure of a disease for which there was no cure and using a very novel concept,” he says. “So this is an exciting new frontier of therapeutic investigation for MYPBC3 gene-positive patients with a chance for a cure.
Neither of Tenaya’s two therapies address the gene mutation that has affected Borsari and her family. But Ali sees opportunity down the road to develop a gene therapy for her particular gene mutation, since it is the second leading cause of cardiomyopathy. Treating the MYH7 gene is especially challenging because it requires gene editing or silencing, instead of just replacing the gene.
Wendy Borsari was diagnosed at age 24 with a commonly inherited heart disease. She joined Tenaya as a patient advocate in 2021.
Wendy Borsari
“If you add a healthy gene it will produce healthy copies,” Ali explains, “but it won’t stop the bad effects of the mutant protein the gene produces. You can only do that by silencing the gene or editing it out, which is a different, more complicated approach.”
Euan Ashley, professor of medicine and genetics at Stanford University and founding director of its Center for Inherited Cardiovascular Disease, is confident that we will see genetic therapies for heart disease within the next decade.
“We are at this really exciting moment in time where we have diseases that have been under-recognized and undervalued now being attacked by multiple companies with really modern tools,” says Ashley, author of The Genome Odyssey. “Gene therapies are unusual in the sense that they can reverse the cause of the disease, so we have the enticing possibility of actually reversing or maybe even curing these diseases.”
Although no one is doing extensive research into a gene therapy for her particular mutation yet, Borsari remains hopeful, knowing that companies such as Tenaya are moving in that direction.
“I know that’s now on the horizon,” she says. “It’s not just some pipe dream, but will happen hopefully in my lifetime or my kids’ lifetime to help them.”