July 23, 2024

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FDA: First CRISPR gene editing therapy NTLA-2001 Granted as orphan drug

FDA: First CRISPR gene editing therapy NTLA-2001 Granted as orphan drug


FDA: First CRISPR gene editing therapy NTLA-2001 Granted as orphan drug.


Intellia Therapeutics is committed to promoting CRISPR gene editing therapy deep into the human body. The company co-founded by Nobel Prize winner Jennifer Doudna announced that the FDA has awarded NTLA-2001 the title of orphan drug, which is used to treat transthyretin (ATTR) amyloidosis.


In Intellia Therapeutics and its partner Regeneron Pharmaceuticals, for the first time, clinical evidence proves that CRISPR is safe and effective for gene editing in vivo. After nearly four months, Intellia announced that the FDA has granted NTLA-2001 the title of Orphan Drug.


The experimental drug NTLA-2001 is under development to treat transthyretin (ATTR) amyloidosis, a rare and life-threatening protein misfolding disease caused by specific mutations in the transthyretin (TTR) gene.

In disease pathology, proteins usually dissolve and fold on themselves, causing a series of problems, including damage to nerves (polyneuropathy), kidneys, and eyes. When the protein is deposited in the heart by mistake, the patient may also have cardiomyopathy (ATTR-CM).

Approximately 50,000 people worldwide suffer from inherited ATTR, and the prognosis is very poor. Patients usually die of the disease within 2 to 15 years after the onset of symptoms.


In June of this year, the clinical data of the first phase of Intellia study showed that after a systemic infusion of NTLA-2001 in the body, the pathogenic protein was greatly reduced. The results published in the New England Journal of Medicine are based on six patients in the first two groups of the study.

The results of the study showed that the clinical trial data included 6 ATTR patients who received treatment in the phase 1 clinical trial, of which 3 received NTLA-2001 at a dose of 0.1 mg/kg, and the other 3 received a dose of 0.3 mg. /kg of NTLA-2001 treatment. The test on the 28th day of treatment showed that NTLA-2001 can dose-dependently reduce the TTR level in the patient’s serum.

The 0.1 mg/kg dose group decreased TTR by an average of 52%, and the 0.3 mg/kg dose group decreased by an average of 87%. One patient’s TTR level decreased by 96%. This is the first batch of clinical data to support the safety and effectiveness of CRISPR therapy in vivo, which is expected to usher in a new era of medicine.


Intellia President and CEO Dr. John Leonard said in a statement: “The designation of orphan drugs underscores the FDA’s recognition of NTLA-2001 as a single-dose new treatment for ATTR amyloidosis with its potential promise.”


The U.S. Food and Drug Administration’s Orphan Drug Designation grants investigational drugs designed to treat, diagnose, or prevent rare diseases that affect less than 200,000 people in the United States.

It gives drug sponsors certain advantages, such as waivers for prescription drug users and seven years of marketing exclusivity if the drug is approved.

NTLA-2001 has been awarded the title of orphan drug for ATTR amyloidosis in the European Union, which was approved by the European Commission (EC) in March.


Intellia’s early summer milestone kicked off a breakthrough in gene editing in 2021. On September 30, Editas Medicine released preliminary data from the Phase I/II brilliant trial of EDIT-101, which was developed for a type of hereditary blindness called Leber congenital amaurosis type 10 (LCA10). This is the second evidence of the success of CRISPR gene editing in vivo.


Many people hope for gene editing in the body because it has the potential to permanently cure rare genetic diseases. Although these research therapies are still in the early stages of the field, they provide the possibility of “one-off” treatments for patients who would otherwise have no viable treatment or depend on expensive and cumbersome repetitive treatments for life.




FDA: First CRISPR gene editing therapy NTLA-2001 Granted as orphan drug

(source:internet, reference only)

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