Last Updated 2/24/2023

Intellia Therapeutics


Intellia is one of the original CRISPR CAS9 companies. They use the original CAS9 enzyme to do gene editing. Due to the possible risks of using a double stranded break in a person, they have focused on using the technology for editing cells outside of humans like stem cell and T cell therapies. They also proved safety and efficacy in human gene knockout. Now they are progressing toward in-vivo editing. They are working in the T cell space with cell editing. They are building their own versions of base editing and possibly gene writing from their acquisition of Rewrite. This gives them a good balance of current technology and future technology.


John Leonard has only been the CEO for a short time, but I think he has done a great job. He really turned this company around which got me to sell out of CRISPR Therapeutics and move into Intellia. He bought Recode which gives them technology toward second generation editors. He has done a good job at managing the cash with a strong balance sheet. They are generating good early clinical data and started working toward US IND filings.


NTLA-2001 is their first therapy for in-vivo gene knockout of the ATTR gene which they are focusing on the Cardiomyopathy segment of the patient population. This therapy is designed to remove the unwanted ATTR proteins that are made from the mutated genes. This is moving into phase 2 outside the US. The data showed up to 93% knock down of the unwanted ATTR protein in serum. They plan to file the US IND by mid year for CM with hopes to start the trial by year end. The phase 1 trial of the Polyneuropathy arm is complete and they are evaluating the phase 3 design.

NTLA-2002 is their second therapy targeting the Kallikrein gene for knockout. The historical data from HAE shows that the knock down of Kallikrein in the plasma can reduce the attacks from HAE. This program started phase 2 outside the US. They submitted the IND for the US and plan to start a phase 2 if they are cleared by the FDA. They have some initial data showing up to 92% reduction in Kallikrein. They also showed an attack free event time improvement.

NTLA-2003 is their therapy for AATD in the Liver where they attempt to knock out the SERPINA1 gene. I suspect this would work very much like RNAi knockout programs for this target, but in a permanent manner.It would compete with RNAi in a competitive space though. They plan to submit the IND for this program this year.

NTLA-3001 is their therapy for AATD in the Lung where they attempt to insert the SERPINA1 gene. They are using Homology Directed Repair using a donor DNA template strand. I find this program extremely concerning as so much could go wrong with this program.

NTLA-6001 is allogeneic CAR-T therapy targeting CD30 in Lymphomas. They are testing out a potential immune evasion strategy where they knock out the HLA-A but match the HLA-B and HLA-C. I honestly do not see the value to this. It seems like a really weak attempt at immune evasion. The whole point of knocking out HLA is so you don't have to match. I wait and see the data though. My guess is it gets dropped.


Cash $1.3 billion

NTLA-2001 is shared 75% Intellia and 25% Regeneron. Their first program is for hATTR CM. This is about 50,000 worldwide. I think they could do about 2,000 patients per year for at least $2 million price as RNAi alternatives cost nearly $600,000 per year. I could see at least up to $4 billion sales. That would entitle Intellia to $3 billion in sales. With good phase 1 data, I would put this multiple at 1 right now. That makes it worth $3 billion value.

NTLA-2002 is a gene knock out therapy targeting HAE. This is about 7,000 patients in the US each year. I think they could do a few hundred a year. This could be worth over $1 billion in sales. I would also give this program .5 multiple based on the early data. That makes it worth $500 million value.

NTLA-3001 is expected to fail. I don't think I will give this program any value at all.

NTLA-2003 is their gene knockout for AATD. There are estimated to be about 60,000 AATD patients in the US. I think they could capture a few thousand of them for a $2 million price. That could make this program worth $2 billion or more in revenues. I would put a .1 multiplier on that for preclinical. That makes it worth $200 million.

NTLA-6001 is a CAR-T therapy for Lymphoma. I don't think I will give this program any value at all.

All in, that is a $5 billion market cap. Based on the 88 million shares outstanding, that comes to $56.81.


Licensed their CAS9 technology from Caribou. They issued shares to Caribou which they have since sold in the market. It does not appear they own anything to Caribou any longer. They even won a patent dispute over chRDNA for CAS9 with Caribou. They are entitled to royalties on CB010 which still uses chRDNA CAS9.

Regeneron partnership for delivery to the Liver where Intellia gets 75% of sales and Regeneron gets 25% of sales. Their first program covers NTLA-2001 in aTTR.

Regeneron partnership for delivery to the Liver where Intellia gets 50% of sales and Regeneron gets 50% of sales. This program covers Hemophilia A and Hemophilia B.

SparingVision partnership for therapies in the eye. They are entitled to $200 million milestones for each program. They can even get royalties on any products.

Kyverna partnership for inflammatory disorders. Intellia has the opt-in right to participate up to 50% in the US.

ONK Therapeutics for NK cells where they can get $184 million in milestones per product and mid single digits royalty on any products.


NTLA-2001 Planning Phase 3 with regulatory authorities for PN.

NTLA-2001 Expect US IND submission in mid 2023 for CM.

NTLA-2002 IND submission in US and start of phase 2 in 1H 2023

NTLA-3001 IND submission 2H of 2023

NTLA-2003 Complete IND activities by end of 2023

NTLA-6001 Looking for collaboration opportunities

Data Readouts

NTLA-2001 Phase 1 data

NTLA-2002 Phase 1 data

Company Website Links

Press Releases

Events & Presentations

SEC Filings

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* These are my Opinions and Estimates. They should not be considered financial advice.