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Yair Einhorn Profile picture
@yaireinhorn
Mar 4, 2024 • 18 tweets • 7 min read • Read on X
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1/As promised & after reading $NTLA latest Q4 & full year 2023 financial report here is my impression regarding @intelliatx latest corporate status. As always I have focused only on the main issues that I found to be the most interesting & relevant. Here is my 🧵👇 $XBI #BioTech Intellia Therapeutics - a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2023.
2/IMO the most significant corporate event was @intelliatx collaboration signed with @ReCodeTx - which uses tissue-specific delivery to power mRNA & gene correction therapeutics to develop novel medicines for the treatment of Cystic fibrosis based on $NTLA Gene Editing platform In my opinion the most significant corporate event was intellia’s collaboration signed with ReCode therapeutics - which uses tissue-specific delivery to power mRNA and gene correction therapeutics to develop novel medicines for the treatment of Cystic fibrosis based on intellia’s platform
3/Another major development - which was recently presented in the @jpmorgan’s health conference, was @intelliatx’s restructured pipeline & its new 2024-2026 main Strategic Priorities:
1. Trials for its 2 in vivo #CRISPR platforms - $NTLA-2001 & 2002
2. POC for $NTLA new CRISPR-in vivo targeted gene insertion & allogeneic ex vivo program
3. Developing new Gene editing programs outside the liver
4. DNA writing3/Another major development - which was recently presented in the jpmorgan’s health conference, was intelliatx’s restructured pipeline and its new 2024-2026 main Strategic Priorities: 1. Trials for its 2 in vivo CRISPR platforms - NTLA-2001 & 2002 2. POC for NTLA new CRISPR-in vivo targeted gene insertion and allogeneic ex vivo program 3. Developing new Gene editing programs outside the liver 4. DNA writing
@jpmorgan @intelliatx 4/Following the restructuring of its clinical portfolio & focusing on its main Gene editing programs - $NTLA - 2001, 2002 & 3001 - @intelliatx anticipates to reach the following key clinical milestones during 2024👇 Following the restructuring of its clinical portfolio and focusing on its main Gene editing programs NTLA - 2001, 2002 and 3001 - intelliatx anticipates to reach the following key clinical milestones during 2024👇
5/NTLA-2001 is @intelliatx’s In-Vivo CRISPR Cas9 Gene editing program which is aimed to treat Transthyretin Amyloidosis-ATTR. $NTLA-2001 could potentially halt and reverse the disease by knocking out the TTR gene with a single dose-creating a “one & done” treatment for patients NTLA-2001 is intellia In-Vivo CRISPR Cas9 Gene editing program which is aimed to treat Transthyretin Amyloidosis-ATTR. NTLA-2001 could potentially halt and reverse the disease by knocking out the TTR gene with a single dose-creating a “one and done” treatment for patients
@intelliatx 6/@intelliatx is actively enrolling patients, including in the U.S., in the pivotal Phase 3 MAGNITUDE trial. $NTLA is on track to dose the first patient in Q1 2024, continues to open new clinical sites & plans to present updated data from the ongoing Phase 1 study in 2024. intelliatx is actively enrolling patients, including in the U.S., in the pivotal Phase 3 MAGNITUDE trial. $NTLA is on track to dose the first patient in Q1 2024, continues to open new clinical sites & plans to present updated data from the ongoing Phase 1 study in 2024.
@intelliatx 7/@intelliatx’s second program is $NTLA-2002 which aims to knock out the KLKB1 gene in the liver thus reducing total plasma kallikrein protein & activity - a key mediator of HAE hereditary angioedema which affects more than 15,000 patients globally & has a $4B+ market size intellia’s second program is NTLA-2002 which aims to knock out the KLKB1 gene in the liver thus reducing total plasma kallikrein protein and activity - a key mediator of HAE hereditary angioedema which affects more than 15,000 patients globally and has a four billion dollars market size
@intelliatx 8/@intelliatx has announced in January that it had completed the enrollment and dosing in the Phase 2 portion of the Phase 1/2 study in adults with HAE. $NTLA plans to present updated data from the Phase 1 and new data from the Phase 2 portion during 2024. Intellia has announced in January that it had completed the enrollment and dosing in the Phase 2 portion of the Phase 1/2 study in adults with HAE. $NTLA plans to present updated data from the Phase 1 and new data from the Phase 2 portion during 2024.
9/In January @intelliatx reported preliminary positive results from its Phase 1 portion of the Phase 1/2 study of $NTLA-2002 which was published in the @NEJM. The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion & was well tolerated at all dose levels. The most frequent adverse events reported were mild, transient infusion-related reactions and fatigue.In January intellia reported preliminary positive results from its Phase 1 portion of the Phase 1/2 study of NTLA-2002 which was published in The New England Journal of Medicine. The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion and was well tolerated at all dose levels. The most frequent adverse events reported were mild, transient infusion-related reactions and fatigue.
10/@intelliatx $NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. In December 2023, Intellia submitted a Clinical Trial Application (CTA) to initiate a first-in-human, Phase 1 study of NTLA-3001. The Company plans to dose the first patient in 2024.intellia’s NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. In December 2023, Intellia submitted a Clinical Trial Application (CTA) to initiate a first-in-human, Phase 1 study of NTLA-3001. The Company plans to dose the first patient ...
@intelliatx @NEJM 11/One of @intelliatx’s major strengths is its multiple collaborations: 1)the acquisition of Rewrite therapeutics 2)@OnkTherapeutics - development of CRISPR-edited NK Cell therapy for Cancer 3)@KyvernaT - development of KYV-201 - a CD19 CAR-T cell for autoimmune diseases. 11/One of intelliatx’s major strengths is its multiple collaborations: 1)the acquisition of Rewrite therapeutics 2)Onk Therapeutics - development of CRISPR-edited NK Cell therapy for Cancer 3)Kyverna therapeutics - development of KYV-201 - a CD19 CAR-T cell for autoimmune diseases.
12/@intelliatx most important collaboration is the one with @Regeneron. In October 2023, $NTLA & $RGNX announced an expanded research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological & muscular diseases. In addition, Regeneron also exercised its option to extend the existing technology collaboration term with Intellia for two more years. The technology collaboration term now extends to April 2026, and Intellia will receive a $30M payment due in April 2024.12/Intellia’s most important collaboration is the one with Regeneron. In October 2023, Intellia and Regeneron announced an expanded research collaboration to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. In addition, Regeneron also exercised its option to extend the existing technology collaboration term with Intellia for two more years. The technology collaboration term now extends to April 2026, and Intellia will receive a 30 million dollars payment due in April 2024.
13/Another important $NTLA collaboration is the one signed with @SparingVision - a French biotech company focused on developing treatments for ocular Genetic Diseases. SPVN will utilise @intelliatx CRISPR Cas9 platform & will develop new treatments for up to 3 selected targets👇
@SparingVision @intelliatx 14/In 2021 @intelliatx had acquired 10% of @SparingVision’s equity in exchange for up to $200M in future funding around milestones & royalties. @SparingVision will select 3 drug candidates & $NTLA will own 2 of the 3, owning all commercial rights in the 🇺🇸 + 50% of all non US In 2021 intellia had acquired 10% of Sparing Vision equity in exchange for up to 200 million dollars in future funding around milestones & royalties. Sparing Vision will select 3 drug candidates and Intellia ill own 2 of the 3, owning all commercial rights in the United States + 50% of all non US
@SparingVision @intelliatx 15/In September @SparingVision & $NTLA announced that they have selected an undisclosed 2ND target as part of their strategic collaboration to develop novel genomic medicines utilizing @intelliatx CRISPR-based Gene Editing technologies for the treatment of ocular diseases. $XBI In September Sparing Vision and Intellia announced that they have selected an undisclosed second target as part of their strategic collaboration to develop novel genomic medicines utilizing Intellia’s CRISPR-based Gene Editing technologies for the treatment of ocular diseases.
@SparingVision @intelliatx 16/As of 12/31/23 $NTLA had capital resources of $1B & quarterly R&D expenses of $109M. IMO despite the high cash burn, @intelliatx’s current cash position of ~ $1B will enable it to continue in developing its pipeline & to initiate additional acquisitions and collaborations. As of 12/31/23 Intellia had capital resources of 1 billion dollars and quarterly research and development expenses of 109 million dollars. In my opinion despite the high cash burn, Intellia’s current cash position of approximately 1 billion dollars will enable it to continue in developing its pipeline and to initiate additional acquisitions and collaborations.
@SparingVision @intelliatx 17/@intelliatx remains very promising due to its broad collaboration agreements alongside a diverse pipeline. After receiving an IND clearance for $NTLA-2001 thus clearing the way to the US market & advancing $NTLA-2002 into Phase 3 - ‘24 is expected to be a strong year for $NTLA intellia remains very promising due to its broad collaboration agreements alongside a diverse pipeline. After receiving an IND clearance for NTLA-2001 thus clearing the way to the US market and advancing NTLA-2002 into Phase 3 - 2024 is expected to be a super strong year for Intellia
Please feel free to share, retweet or Bookmark this🧵so that those on @X - who are interested in Gene editing, CRISPR, BioTech and Genomics will be able to access this resource and as always I would be more than happy to read your thoughts. $XBI $NTLA

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Yair Einhorn Profile picture
Feb 9
1/🚨WOW! $CRBU has presented updated clinical data from @CaribouBio ongoing CB-011 CaMMouflage phase 1 clinical trial in cancer patients with relapsed or refractory multiple myeloma (r/r MM). The CB-011 CaMMouflage phase 1 translational and clinical data - which was presented at the 2026 Tandem Meetings of ASTCT and CIBMTR, demonstrated deep and durable responses and further support the potential of CB-011 as the best-in-class allogeneic CAR-T Cell Therapy for second-line relapsed or refractory multiple myeloma patients. $XBI 🧵👇WOW! $CRBU has presented updated clinical data from its ongoing CB-011 CaMMouflage phase 1 clinical trial in cancer patients with relapsed or refractory multiple myeloma (r/r MM). The CB-011 CaMMouflage phase 1 translational and clinical data - which was presented at the 2026 Tandem Meetings of ASTCT and CIBMTR, demonstrated deep and durable responses and further support the potential of CB-011 as the best-in-class allogeneic CAR-T Cell Therapy for second-line relapsed or refractory multiple myeloma patients. $XBI
2/Multiple myeloma is a type of blood cancer that develops in the patient’s bone marrow when plasma cells become cancerous and produce abnormal proteins instead of functional antibodies. Common symptoms include persistent bone pain (often back or ribs), fatigue, frequent infections, kidney damage and high calcium levels. While modern treatments (chemotherapy, targeted therapy, stem cell transplants) can manage the disease for years - Multiple myeloma is still considered incurable and present a significant unmet medical need.Multiple myeloma is a type of blood cancer that develops in the patient’s bone marrow when plasma cells become cancerous and produce abnormal proteins instead of functional antibodies. Common symptoms include persistent bone pain (often back or ribs), fatigue, frequent infections, kidney damage and high calcium levels. While modern treatments (chemotherapy, targeted therapy, stem cell transplants) can manage the disease for years - Multiple myeloma is still considered incurable and present a significant unmet medical need.
3/CB-011 is an alogeneic anti-BCMA CAR-T Cell Therapy using $CRBU Cas12a CRISPR hybrid RNA-DNA (chRDNA) technology aimed to insert an anti-BCMA CAR expression into the TRAC gene and thus to knock out B2M to boost the persistence of antitumor activity and to reduce rejection. CB-011 is an alogeneic anti-BCMA CAR-T Cell Therapy using Caribou’s Cas12a CRISPR hybrid RNA-DNA (chRDNA) technology aimed to insert an anti-BCMA CAR expression into the TRAC gene and thus to knock out B2M to boost the persistence of antitumor activity and to reduce rejection.
Read 7 tweets
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

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