A New Era Begins! Intellia Reports First Clinical Proof for Direct CRISPR Genome Editing in Humans

by Rajaneesh K. Gopinath
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June 26th, 2021 – Intellia Therapeutics, a gene-editing company co-founded by Nobel laureate, Jennifer Doudna, created history today, revealing the first-ever clinical proof for directly editing human genomes using the revolutionary CRISPR technology.

Along with development partner Regeneron Pharmaceuticals, the Cambridge, MA-based startup reported positive data from a landmark Phase 1 study that evaluates a potential transthyretin (ATTR) amyloidosis treatment. NTLA-2001, its lead in vivo genome editing candidate, is the first CRISPR-based therapy to be administered intravenously for precise editing of a human gene in vivo. If approved, it could be the first permanent curative treatment for ATTR.

The encouraging results were presented today at the 2021 Peripheral Nerve Society (PNS) Annual Meeting and simultaneously published in The New England Journal of Medicine.

“These are the first-ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR. The interim results support our belief that NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose,” said John Leonard, President and CEO of Intellia.


Transformative Cures for Debilitating Genetic Diseases

The results obtained with NTLA-2001 would serve as a bedrock for similar therapies against other genetic diseases. Presently, Intellia is also developing NTLA-2002, a second in vivo knockout therapeutic candidate to treat hereditary angioedema (HAE). Besides, it has another CRISPR/Cas9-based in vivo therapy pipeline for Hemophilia A and B under the collaboration with Regeneron.

“Solving the challenge of targeted delivery of CRISPR/Cas9 to the liver, as we have with NTLA-2001, also unlocks the door to treating a wide array of other genetic diseases with our modular platform, and we intend to move quickly to advance and expand our pipeline. With these data, we believe we are truly opening a new era of medicine,” Leonard added.

Related Article: New Era in Precision Medicine? Introduction & Overview of RNA Editing

A Potential One Time Treatment for ATTR Amyloidosis

ATTR amyloidosis is a rare, life-threatening genetic disease caused by the misfolding of transthyretin (TTR) protein. The amyloid deposits accumulate in multiple tissues, including the heart, nerves, and GI tract leading to complications such as polyneuropathy (ATTRv-PN) and cardiomyopathy. Hereditary ATTR (hATTR) is autosomal dominant, meaning the disease occurs even if one copy of the TTR gene is defective.

The most prevalent treatment for this progressive disease is an orthotopic liver transplant. In 2018, Alnylam’s RNAi drug, Onpattro, and Ionis’ antisense oligonucleotide inhibitor, Tegsedi, were FDA approved as treatments for hATTR. In 2019, Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) also bagged the drug regulator’s approval for cardiomyopathy caused by ATTR. Presently, Ionis is evaluating AKCEA-TTR-LRx, an antisense drug candidate.

However, NTLA-2001 is the only one-time treatment in development for the disease. The CRISPR therapy is designed to inactivate the TTR gene in liver cells and prevent the production of misfolded TTR protein.

Interim results reported at the meeting were from six ATTRv-PN patients who received either 0.1 mg/kg (n=3) or 0.3 mg/kg (n=3)of NTLA-2001. Results showed that NTLA-2001 led to dose-dependent reductions in serum TTR. Three patients in the 0.1 mg/kg dose group registered a reduction of 52%, while the other three in the 0.3 mg/kg dose group showed 87%. One patient who received the highest dose showed a 96% reduction. In comparison, chronic dosing of treatment standard yielded approximately 80% TTR reductions.

“ATTR amyloidosis is a progressive and fatal disease that usually requires chronic, lifelong treatment. These interim Phase 1 data support NTLA-2001 as the only one-time treatment either on the market or in development,” said the study’s national coordinating investigator, Dr. Julian Gillmore, a Professor at the Royal Free Hospital, UK.

“As the first-ever systemically administered CRISPR therapy candidate, NTLA-2001 shows strong potential to stop the production and accumulation of the misfolded TTR protein by inactivating the TTR gene at the root of the disease. This approach could deliver life-changing, lifelong benefits to patients with all forms of ATTR amyloidosis, who continue to experience debilitating symptoms and complications of disease while on the standard of care. While further investigation is needed, these results are highly encouraging.”

Intellia Therapeutics was featured as one of GeneOnline’s 10 top gene therapy companies to look out for in 2021.

Related Article: Novartis’ Expensive Gene Therapy for SMA Reasserts its Efficacy in Two Clinical Trials

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