Category Archives: Gene Editing

FDA approves landmark sickle cell gene therapy treatment

Posted on by • Posted in Casgevy, Clinical Research, Clinical Trials, CRISPR Gene Therapy, CRISPRs, Food and Drug Administration (FDA), Gene Editing, Gene Therapy, Health Insurance Coverage Adequacy, Health Insurance Denial, Lyfgenia, Sickle Cell Disease • Leave a comment

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Last week, the Food and Drug Administration (FDA) approved two gene therapy treatments for sickle cell disease, Casgevy and Lyfgenia.

Casgevy, jointly developed by Boston, MA-based Vertex Pharmaceuticals and Switzerland-based CRISPR Therapeutics, is the first approved treatment of any kind available to US patients that uses CRISPR’s gene-editing capabilities.

Lyfgenia, made by Somerville, MA-based Bluebird Bio, uses a more common retrovirus technique for genetic modification. The FDA estimates that about 20K Americans with sickle cell disease will be eligible for the therapies, limited to those patients 12 and older who have had episodes of debilitating pain.

Both treatments will only be available at a small number of facilities nationwide, priced between $2-3M, and require a patient to endure months of hospitalization as well as intensive chemotherapy. Around 100K mostly Black Americans suffer from sickle cell disease, which causes intense pain, organ damage, and reduced life expectancy. Previously, the only curative treatment was a bone marrow transplant.

The Gist: The approval of these drugs represents a milestone moment for those suffering from sickle cell disease, while Casgevy also fulfills the revolutionary promise scientists have seen in CRISPR since it first received broad attention in 2005.

However, now that gene-editing therapies have graduated from the domain of scientific possibility into the realities of our healthcare delivery system, the new challenge becomes ensuring accessibility and equity, as many Americans who most stand to benefit from it also experience barriers in access to care and insurance coverage. (We’d expect insurer pushback similar to that seen when the first highly effective, but extremely costly, hepatitis C treatments like Solvaldi hit the market a decade ago this month.)

While the clinical trial patients who received Casgevy report having “a new lease on life”, sky–high costs, questions of insurance coverage, and the arduous, time-intensive nature of the procedure stand in the way of a population-wide cure for sickle cell disease.

CRISPR Shows Promise in Gene-Editing Therapy During Clinical Trial

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CRISPR-Cas9 Gene Therapy Shows Promise in Angelman Mouse Model

Scientists at UCL National Amyloidosis Centre at the Royal Free Hospital, London are hoping their gene editing therapy using CRISPR will be a breakthrough for patients suffering from hereditary transthyretin (ATTR) amyloidosis. In a phase 1 clinical trial, the first six patients have shown positive interim results for gene-editing treatment.

The CRISPR breakthrough comes in treating transthyretin amyloidosis, a mutation in the transthyretin (TTR) gene. Those with this mutation produce an abnormal protein, which gradually builds up in the heart and nerves. Symptoms can include numbness in the hands and feet, loss of control of the bowel and bladder, and loss of mobility.

Hereditary transthyretin amyloidosis gets progressively worse and is fatal. Up until this point, most of the treatment options available to patients have included management of the symptoms and prevention of progression.

Those taking part in the trial have received a molecule knows as CRISPR/Cas9 via one-off infusion. The purpose of this is to deactivate the incorrect gene within the liver cell.

“With the gene no longer active in the liver, it is expected that the patient will only produce negligible levels of the harmful transthyretin protein,” UCL stated in a press release. 

Scientists saw in the first six patients a reduced production of the harmful transthyretin protein by up to 96 percent, 28 days after the treatment. Additionally, there were no serious adverse effects witnessed. This data was published in the New England Journal of Medicine.

“As the trial progresses, patients will be given higher doses of the gene editing therapy with the hope that will drive the levels of toxic protein even lower,” UCL explained. 

CRISPR/Cas9, a Nobel Prize-winning technology, has been used to edit cells outside the body in the past. However, UCL is presenting the first clinical data which CRISPR/Cas9 is being used as medicine itself for a potential therapy.

“This is wonderful news for patients with this condition. If this trial continues to be successful, the treatment may permit patients who are diagnosed early in the course of the disease to lead completely normal lives without the need for ongoing therapy,” Professor Julian Gillmore, the trial lead, of the UCL National Amyloidosis Centre, part of the UCL Centre for Amyloidosis and Acute Phase Proteins said in a press release.

“Until very recently, the majority of treatments we have been able to offer patients with this condition have had limited success. If this trial continues to go well, it will mean we can offer real hope and the prospect of meaningful clinical improvement to patients who suffer from this condition,” Gillmore continued.

The global trial includes patients from the Royal Free London and a hospital located in Auckland, New Zealand. The investigational therapy, designated NTLA-2001, is being developed by Intellia Therapeutics; a biotechnology company based in the United States.

 This could be a big step forward in using CRISPR as gene therapy. Typically, the therapy is injected into the site of illness. However, this newest approach injects CRISPR directly into the bloodstream, which could revolutionize how clinicians treat certain illnesses.

Another new first for CRISPR

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Image result for crispr gene editing

For the first time, scientists have used the gene-editing technique CRISPR inside the body of an adult patient, in an effort to cure congenital blindness, Bryan reports.

Why it matters: CRISPR has already been used to edit cells outside a human body, which are then reinfused into the patient.

  • But the new study could open the door to using gene editing to treat incurable conditions that involve cells that can’t be removed from the body, like Huntington’s disease and dementia.

Details: The research was sponsored by biotech companies Editas Medicine of Cambridge, Massachusetts, and Allergan of Dublin, Ireland, and was carried out at Oregon Health and Science University.

  • Scientists led by Eric Pierce of Harvard Medical School injected microscopic droplets carrying a benign virus into the eye of a nearly blind patient suffering from the genetic disorder Leber congenital amaurosis.
  • The virus had been engineered to instruct the cells to create CRISPR machinery. The hope is that CRISPR will edit out the genetic defects that cause blindness, restoring at least some vision.
  • “We literally have the potential to take people who are essentially blind and make them see,” Charles Albright, chief scientific officer at Editas, told AP.

“It gives us hope that we could extend that to lots of other diseases — if it works and if it’s safe,” National Institutes of Health director Francis Collins told NPR.