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Yair Einhorn Profile picture
@yaireinhorn
May 21, 2022 • 14 tweets • 14 min read • Read on X
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1/As promised & after reading $NTLA latest Q1 2022 financial report here is my analysis regarding @intelliatx latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #GeneEditing #BioTech #FinTwit #Genomics👇
2/NTLA-2001 is the most advanced In-Vivo #CRISPR/#Cas9 #GeneEditing program in $NTLA portfolio & is aimed to treat Transthyretin Amyloidosis - (ATTR). NTLA-2001 could potentially halt & reverse the disease in a “one time” treatment by knocking out the TTR gene with a single dose
3/In February $NTLA has presented an updated clinical data from its Phase 1 study of NTLA-2001 which included 15 patients with ATTR amyloidosis. IMO NTLA-2001 readout looks promising & here is a summary that I wrote shortly after @intelliatx’s presentation of NTLA-2001👇
4/NTLA-2002 is aimed to knock out the KLKB1 gene in the #liver thus reducing total plasma kallikrein protein & activity, a key mediator of #HAE - hereditary #angioedema. In 12/1/21 $NTLA has dosed the 1ST patient & will publish interim data from the Phase 1/2 study in 2H of 2022.
5/Two more In-Vivo programs are $NTLA 3001 & 2003: NTLA-3001 is aimed for AATD-associated #Lung disease & IND will be filled during 2023. NTLA-2003 is aimed for AATD-associated #Liver disease & @intelliatx is advancing towards IND-enabling activities for this program as well.
6/NTLA-5001 is an autologous T-cell therapy aimed for the treatment of all genetic subtypes of #AML. In March @intelliatx announced that the first patient was dosed in its Phase 1/2a study & that @US_FDA has granted orphan drug designation to NTLA-5001 for the treatment of #AML👇
7/IMO the most important development was the designation of NTLA-6001 - an allogeneic CAR-T program for treating hematologic #cancer including Hodgkin #lymphoma (cHL). NTLA-6001 is the first program that was developed using @intelliatx’s proprietary cell engineering platform👇
8/One of @intelliatx’s major strengths is its multiple collaborations: 1)acquisition of Rewrite 2)@OnkTherapeutics - the co - development of #CRISPR-edited NK #CellTherapy for #Cancer. 3)@KyvernaT - the development of KYV-201 - a CD19 CAR-T cell for an #autoimmune diseases.👇
9/As of 3/31/22 $NTLA had capital resources of $994.7M & R&D expenses of $133.1M. IMO although the cash burn has increased by $94M, @intelliatx cash position of $994M will enable it to both continue to develop its pipeline & to initiate additional acquisitions & collaborations.
10/Upcoming Milestones:
1.NTLA-2001 - ATTR:
ATTRv-PN arm - Phase 1 data in June 2022
ATTR-CM arm - Phase 1 data in 2H 2022
2.NTLA-2002 - HAE:
Phase 1/2 data in 2H 2022 3.NTLA-3001 - AATD:
File an IND in 2023
4.NTLA-5001 - AML:
Enrolment of patients in Phase 1/2a during 2022
11/@intelliatx is definitely an impressive & promising #GeneEditing company. Both $NTLA proprietary pipeline & its systematic strategy of collaborations & acquisitions are the key factors which defines $NTLA as a leading #CRISPR and #GeneEditing company.
on a personal note: one of my main interests in $NTLA was its collaboration with @SparingVision - aimed to develop #Ocular #GeneEditing platforms for the treatment of #IRDs. As Of Q1 22 no progress was published yet & I eagerly await to see what progress this agreement will bring
And as always please feel free to share and retweet so that those on @Twitter or #FinTwit who are interested in #GeneEditing, #CRISPR, #BioTech & #Genomics will get this relevant data. Enjoy the rest of your #Weekend!
@ArielDa58728507

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More from @yaireinhorn

Yair Einhorn Profile picture
Jan 14
1/@PrimeMedicine’s recent #JPM26 presentation emphasised its strategic priorities & the company’s planned milestones for 2026-7. Here’s my🧵👇which focuses on PRIME’s new prioritised pipeline, the progress $PRME has made during 2025 & its current corporate status. $XBI #JPM2026 Prime Medicine’s recent JPM26 presentation emphasised its strategic priorities & the company’s planned milestones for 2026-7. Here’s my post which focuses on PRIME’s new prioritised pipeline, the progress Prime has made during 2025 and its current corporate status.
2/@PrimeMedicine’s proprietary platform - Prime Editing is the only Gene Editing platform which can edit, correct, insert or even delete large DNA sequences in any target tissue. This makes it the most advanced & promising editing technology with $PRME owing its full IP rights. Prime Medicine’s proprietary platform - Prime Editing is the only Gene Editing platform which can edit, correct, insert or even delete large DNA sequences in any target tissue. This makes it the most advanced & promising editing technology with Prime owing its full IP rights.
3/IMO the most important key takeaway from @primemedicine’s #JPM26 presentation is that in 2026 $PRME intends to submit INDs and to initiate clinical trails for both of its key programs - PM577 for Wilson’s disease and PM647 for AATD. Moving forward with its liver franchise and providing positive human clinical data are a threshold for $PRME to enter into a significant collaboration with a big Pharma company.Image
Read 11 tweets
Yair Einhorn Profile picture
Aug 24, 2025
1/🚨WOW! According to this excellent Economist’s article 🧵👇 it seems that the 🇬🇧 big Pharma company AstraZeneca - one of Europe’s and England’s last remaining BioTech and Pharma moguls, is moving its business away from England! $AZN leaving the UK is a huge negative milestone in the deterioration process of both the British and the European BioTech ecosystems which are rapidly collapsing and losing ground to the American BioTech ecosystem - following President Trump’s Most-Favored-Nation (MFN) policy as well as to the Chinese one which has made an exponential progress. $XBIWOW! According to this excellent Economist’s article it seems that the big Pharma company AstraZeneca - one of Europe’s and England’s last remaining BioTech and Pharma moguls, is moving its business away from England! AstraZeneca leaving the UK is a huge negative milestone in the deterioration process of both the British and the European BioTech ecosystems which are rapidly collapsing and losing ground to the American BioTech ecosystem - following President Trump’s Most-Favored-Nation (MFN) policy as well as to the Chinese one which has made an exponential progress.
2/The British government has always considered its life science industry as one of the Crown Jewels of its economy and rightfully so - a 145 billion dollars industry which employs over 300,000 high skilled employees is crucial for the British economy. The importance of the Pharma and Biotech industry has led the British government to a present a new governmental initiative to support the industry and that was introduced by the Labor government just 3 months ago.The British government has always considered its life science industry as one of the Crown Jewels of its economy and rightfully so - a 145 billion dollars industry which employs over 300,000 high skilled employees is crucial for the British economy. The importance of the Pharma and Biotech industry has led the British government to a present a new governmental initiative to support the industry and that was introduced by the Labor government just 3 months ago.
3/But it seems that the British BioTech & Pharma sector is about to face its biggest challenge ever after AstraZeneca - Britain’s biggest and leading Pharma company is shifting its corporate strategy including a possible relocation of $AZN entire operations to the U.S. including mass investments being made outside England!But it seems that the British BioTech and Pharma sector is about to face its biggest challenge ever after AstraZeneca - Britain’s biggest and leading Pharma company is shifting its corporate strategy including a possible relocation of AZN entire operations to the U.S. including mass investments being made outside England!
Read 13 tweets
Yair Einhorn Profile picture
May 13, 2025
1/🚨WOW! A huge earthquake in the CRISPR & Gene Editing field happened last night after $EDIT has announced that the U.S Federal Court of Appeals has vacated (!) the Patent Trial & Appeal Board’s (PTAB’s) previous decision🧵👇which granted all of the rights for CRISPR/Cas9 Gene Editing to the Broad institute and ruled against University of California, the University of Vienna & Nobel Prize winner Emmanuelle Charpentier - the co-inventor of CRISPR Cas9 and the co-founder of CRISPR Therapeutics. $CRSP $NTLA $XBIWOW! A huge earthquake in the CRISPR & Gene Editing field happened last night after EDITAS has announced that the U.S Federal Court of Appeals has vacated (!) the Patent Trial and Appeal Board’s (PTAB’s) previous decision which granted all of the rights for CRISPR/Cas9 Gene Editing to the Broad institute and ruled against University of California, the University of Vienna & Nobel Prize winner Emmanuelle Charpentier - the co-inventor of CRISPR Cas9 and the co-founder of CRISPR Therapeutics.
2/In February of 2022 the U.S. Patent & Trademark Office has issued a crucial decision in favour of the Broad Institute, which validated its patents for CRISPR/Cas9 Gene Editing in human cells. This provided $EDIT with strong IP rights & gave it a huge commercial advantage. $XBI In February of 2022 the U.S. Patent and Trademark Office has issued a crucial decision in favour of the Broad Institute, which validated its patents for CRISPR/Cas9 Gene Editing in human cells. This provided $EDIT with strong IP rights & gave it a huge commercial advantage.
3/But the losing side in this dispute over the patents for CRISPR/Cas9 editing in human cells - the University of California, the University of Vienna & Emmanuelle Charpentier - the co-founder of $CRSP refused to accept this ruling and instead filed an appeal.
Read 7 tweets
Yair Einhorn Profile picture
Jan 9, 2025
1/Here’s an excellent article - published in the recent @WIRED issue, by Nobel prize winner Jennifer Doudna about how AI & machine learning are amplifying the impact of CRISPR & Gene editing in all walks of life - medicine, agriculture, climate change & research landscape.🧵👇 Here’s an excellent article - published in the recent WIRED issue, by Nobel prize winner Jennifer Doudna about how AI and machine learning are amplifying the impact of CRISPR and Gene editing in all walks of life - medicine, agriculture, climate change and research landscape.
2/Jennifer Doudna is an American biochemist who discovered CRISPR Cas9 as a Gene Editing tool & had received the 2020 Nobel Prize in Chemistry with Emmanuelle Charpentier for their discovery. She also founded several BioTech companies like $NTLA, $CRBU, Scribe, Mammoth & others. Jennifer Doudna is an American biochemist who discovered CRISPR Cas9 as a Gene Editing tool & had received the 2020 Nobel Prize in Chemistry with Emmanuelle Charpentier for their discovery. She also founded several BioTech companies like Intellia therapeutics, Caribou Biosciences, Scribe therapeutics, Mammoth biosciences and others.
3/According to Jennifer Doudna - 2025 will be a pivotal year in which the ever growing use of Artificial Intelligence and machine learning will amplify the effects and reach of CRISPR and Gene Editing in many aspects of our lives - curative medicines, agriculture & lab research. According to Jennifer Doudna - 2025 will be a pivotal year in which the ever growing use of Artificial Intelligence and machine learning will amplify the effects and reach of CRISPR and Gene Editing in many aspects of our lives - curative medicines, agriculture and lab research.
Read 8 tweets
Yair Einhorn Profile picture
Jan 8, 2025
1/🚨WOW! $SANA announced initial positive results from its first-in-human study of UP421 - an allogeneic primary islet Cell Therapy engineered with Sana’s proprietary hypoimmune (HIP) technology for patients with type 1 Diabetes, without the use of any immunosuppression $XBI 🧵👇Sana Biotechnology - a company focused on changing the possible for patients through engineered cells, today announced initial results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with Sana’s hypoimmune (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.
2/Type 1 diabetes is a chronic (life-long) autoimmune disease that prevents the patient’s pancreas from making Insulin - an important hormone that regulates the amount of glucose (sugar) in the blood. Type 1 diabetes affects both children and adults & requires daily management with insulin injections and blood sugar monitoring.
3/Type 1 Diabetes affects roughly 8.4M people worldwide. The number of patients is constantly rising and the prevalence of type 1 diabetes is expected to double over the next decade. Interestingly enough almost 80% of T1D patients are from high-income countries with insurance. Image
Read 8 tweets
Yair Einhorn Profile picture
Aug 18, 2024
1/This great @WSJ’s 📊 shows how after spending tens of billions of dollars for a single BioTech company, big Pharma moguls such as $ABBV, $AZN, $BMY, $AMGN, $PFE & $VRTX have all shifted to acquiring smaller targets of >$5B - for both regulatory & financials reasons. $XBI 🧵👇 After shelling out tens of billions of dollars for single biotech companies, pharma- ceutical giants such as AbbVie, AstraZeneca and Merck have shifted to smaller targets cost- ing $5 billion or less. Many of the companies getting taken out are private. The reason, according to ex- ecutives, bankers and lawyers: Smaller deals are just easier to do in the current regulatory environment, and the sector is looking pretty picked over.
2/All 17 deals made by big Pharma during the first 6 months of ‘24 were $5B or less. During the same period last year, big Pharma made 9 deals, including 2 that were $10B or more. 9 of the 17 deals were for privately held companies, compared to 1 during the same period in 2023. All 17 deals announced by big pharmaceutical companies during the first six months of the year were valued at 5 billion dollars or less, according to re- search firm DealForma. During the same period last year, big drugmakers agreed to nine deals, including two that were 10 billion dollars or larger. Nine of the 17 deals were for privately held companies, compared with one during the same period last year, DealForma said.
3/Last year’s big acquisitions were led by @pfizer’s $43B purchase of cancer biotech $SGEN ❤️👇. By contrast & to demonstrate how things have changed, the biggest deal so far in 2024 was @VertexPharma $4.9B purchase of Alpine Immune Sciences & its experimental kidney drug. $PFE
Read 14 tweets

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