Dr. Pierre Kory had an article about how little has been studied and is known about human biology, but we’ll let these pharmaceutical megacorps run by sociopaths come up with unvetted new gene therapies? I wouldn’t fall for that one. And remember, these white coats are promoting euthanasia and harvesting of organs as well. And RFK, Jr has been a disappointment, but then he was always a controlled opposition asset, and they probably have enough dirt on him from his heroine days along with his wife’s suspicious suicide to where he’ll never go off script. Consequently, we just learned that a 49 year old acquaintance passed away, found on the side of the freeway by a friend who attempted CPR, and we had dinner with her a few years ago when she told us she took the gene therapy to not wear the mask. And she was slim and in good health when we knew her before she left he area. So how many lives has the first gene therapy taken, and still taking?
https://financialpost.com/technology/fda-gene-editing-therapy
Health Secretary Robert F. Kennedy Jr. called the treatment ‘a powerful glimpse into the future of personalized medicine’
A top United States regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare diseases.
Vinay Prasad, who oversees gene therapies at the Food and Drug Administration, said scientific advances, like Crispr, have forced the agency to relax some of its strict rules. As an example, he cited the case of 10-month-old KJ Muldoon, who this year became the first person in history to have his genes custom edited to cure an inherited disease.
“Regulation has to evolve as fast as science evolves,” Prasad said in an interview with Bloomberg News. The agency is “going to be extremely flexible and work very fast with the scientists who want to bring these therapies to kids who need it.”
Prasad plans to publish a paper in early November outlining the FDA’s new approach. He predicted it will spark interest in developing treatments for conditions that may affect only a handful of people.
“The moment we publish our paper, the investment in this space will flow,” Prasad said. “It will turn the spigot on.”
The shares of companies working on gene editing rose on the news. Crispr Therapeutics AG rose as much as eight per cent, Editas Medicine Inc. climbed as much as 12 per cent and Intellia Therapeutics Inc. increased as much as 9.1 per cent
The team behind Baby KJ’s treatment appears to be the first to benefit.
In a paper published Friday, Rebecca Ahrens-Nicklas, a physician at the Children’s Hospital of Philadelphia, and Kiran Musunuru, an expert in genome editing at the University of Pennsylvania, explained how the new process is working for them. They’re designing clinical trials they can use to gain FDA approvals with a gene-editing platform that can be tweaked for patients with different genetic mutations.
Historically, every treatment for every disease has needed its own separate study. The advent of Crispr, the Nobel Prize-winning gene-editing technology, has made it theoretically possible for scientists to develop cures for many of the 7,000 rare diseases, a small fraction of which have treatments. But the FDA’s demands made it seem too expensive and time-consuming.
Now, the FDA is allowing a combined trial of patients with related genetic disorders, Ahrens-Nicklas and Musunuru wrote in the American Journal of Human Genetics. It’s a fundamental shift in how the agency approaches genetic therapies, potentially freeing them from traditional rules that slowed innovation.
“It allows for the amazing ability to tackle multiple genetic diseases at once,” Ahrens-Nicklas said in an interview.
The researchers said they took the unusual step of sharing their interactions with the FDA to show drugmakers there’s a regulatory path for custom-made Crispr treatments. The result may be one-time therapies that cost a few hundred thousand dollars, Musunuru said, rather than the current multimillion dollar price tags.
“It’s important to highlight how quickly things are changing,” Ahrens-Nicklas said. “The FDA today is not the same FDA that existed six months ago.”
For years, scientists have dreamed of using Crispr this way. By simply modifying one part of the drug — the guide RNA — they could treat a variety of conditions, like the same Keurig machine allows people to brew individual cups of coffee.
Health Secretary Robert F. Kennedy Jr. called the treatment for Baby KJ “a powerful glimpse into the future of personalized medicine.” But for many families in the rare disease community, the news was bittersweet. Thousands of parents contacted the team that crafted the treatment asking if their children with devastating diagnoses could be next. The answer, generally, was no.
“It was celebrated, yet it was also sad day for a lot of folks,” Ahrens-Nicklas said. “The science may be advancing, but a lot of people feel like their child has been left behind.”
Musunuru and Ahrens-Nicklas are asking for FDA permission to start clinical trials for a liver disorder known as phenylketonuria, or PKU, which can cause brain damage so severe that doctors prick the heels of every newborn to test for it, and urea cycle disorders like Baby KJ’s, which can lead to seizures, poor muscle tone and coma.
They’re beginning with those conditions in part because they can be treated by editing genes in the liver, a known target for Crispr. UCD can also require a US$1 million liver transplant, so the Crispr approach may also save money, Musunuru said.
There are several subtypes of urea cycle disorders, which collectively affect about one in 30,000 newborns. Drugmakers have pursued treatments for a common subtype, but ignored others that are more rare, according to Tresa Warner, executive director of the National Urea Cycle Disorders Foundation. This potential Crispr trial could change that.
“Knowing this technology can be implemented in other subtypes is really a ray of sunshine,” she said.