The U.S. Food and Drug Administration approved Casgevy, a $2.2 million per patient treatment for use in patients with sickle cell disease.

Casgevy, which utilizes CRISPR technology to edit a person’s genes, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

CNBC reports:

The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the treatment could be challenging given the potential hurdles — including cost — of administering the complex therapy.

Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’s genes to treat disease. The treatment was approved by U.K. regulators last month.

Sickle cell, an inherited blood disorder, causes red blood cells to become misshapen half moons that get stuck inside blood vessels, restricting blood flow and causing what are known as pain crises. About 100,000 Americans are estimated to have the disease.

Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients.

The FDA approved the treatment for people 12 years and older.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.

“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” she added.

“According to a 2012 paper that first announced the technology, CRISPR is basically a cheap and easy way to edit genes and create new genetically modified organisms (GMOs) – in this case, out of human beings,” Natural News wrote.

Natural News discussed the risks involved with CRISPR gene-editing technology:

Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the technology was approved after just one study that followed only 29 out of 45 total participants for 16 months. Twenty-eight out of these 29 supposedly had no pain after one year, Nature reported.

In a clinical trial for beta thalassemia, 39 of the 42 trial participants tracked did not end up needing a routine red blood cell transfusion for a full 12 months at least – without Casgevy, these same beta thalassemia patients require a blood transfusion every three to five weeks.

While the media and the companies they back that produce this kind of stuff are celebrating these amazing recoveries, what they are not telling people is that CRISPR comes with serious side effects, not the least of which include unexpectedly large deletions that result in even more serious genetic mutations.

Advertisement

“It is well established that CRISPR / Cas9 gene editing is not only prone to off-target genetic damage but also a wide range of unintended mutations even at the intended edit site,” said Michael Antoniou, PhD., head of the Gene Expression & Therapy Group at King’s College London. “This can negatively impact the function of multiple genes, which can lead to cancer.”

“It is thus vital that those responsible for administering the gene editing therapy conduct unbiased genome-wide analysis of treated patients for potentially life-damaging DNA mutations. This is essential as it only takes one cell within the large pool of edited cells to go wrong and cause cancer.”

In the meantime, a U.S. company is pushing a different but similar CRISPR technology called “base editing” that was reportedly designed to artificially reduce people’s cholesterol levels.

Join The Conversation. Leave a Comment.


We have no tolerance for comments containing violence, racism, profanity, vulgarity, doxing, or discourteous behavior. If a comment is spam, instead of replying to it please click the ∨ icon below and to the right of that comment. Thank you for partnering with us to maintain fruitful conversation.