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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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More from @yaireinhorn

Yair Einhorn Profile picture
Jan 9
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
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
Yair Einhorn Profile picture
Jul 22, 2024
The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver a genetic payload to the exact desired location. Here’s a great Infograph which shows which BioTech companies use Viral delivery methods (AAVs, LVs) compared to non-viral platforms (NLPs). $XBI The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver the genetic payload to the exact desired location. Here is a great Infograph which shows which BioTech companies use Viral (AAVs) delivery methods compared to non-viral platforms (NLPs).
2/Must of the companies - as you can see in the graph m☝️- use a viral based delivery platform for Gene Therapy / Gene Editing treatments, mostly AAV - Adeno-Associated Virus based delivery. AAV is a naturally occurring virus being transformed into a delivery mechanism by replacing its viral DNA with new DNA, thus making it a precisely coded vector & it is no longer considered a virus, as most of its viral components have been replaced.
3/The main problem with AAV delivery system is that it is limited to the size of the payload which can fit into the virus vassal. It could also cause the patient an inflammatory response due to this external virus activity. This led to a search for a non-viral mechanism. $XBI The main problem with AAV delivery system is that it is limited to the size of the payload which can fit into the virus vassal. It could also cause the patient an inflammatory response due to this external virus activity. This led to a search for a non-viral mechanism. $XBI
Read 8 tweets
Yair Einhorn Profile picture
Jul 9, 2024
1/@nvelop_tx has presented its proprietary delivery system - DLVR-M, which has successfully demonstrated improved delivery of CRISPR, base editing & prime editing platforms to different human cell types. DLVR-M could be the key in overcoming the obstacle of CRISPR delivery. $XBI nvelop therapeutics has presented its proprietary delivery system - DLVR-M, which has successfully demonstrated improved delivery of CRISPR, base editing & prime editing platforms to different human cell types. DLVR-M could be the key in overcoming the obstacle of CRISPR delivery
2/Every Gene Therapy is combined from 2 components - the genetic payload which is the mechanism for fixing the genetic disease or the cure itself & a delivery vehicle which needs to deliver the “package” to a specific human tissue & location. Just Imagine a pickup truck & a box. Image
3/As of today the only approved delivery platform for Gene Therapy / Gene Editing is AAV - Adeno-Associated Virus based delivery. AAV is a naturally occurring virus being transformed into a delivery mechanism by replacing its viral DNA with new DNA thus making it a precisely coded vector & it is no longer considered a virus, as most of its viral components have been replaced
Read 12 tweets
Yair Einhorn Profile picture
May 29, 2024
1/WOW! hC Bioscience announces its first ever tRNA-based program for Duchenne muscular dystrophy (DMD). hC is developing anticodon engineered tRNAs as a potential treatment for DMD patients with shortened & nonfunctional dystrophin due to premature termination codons (PTCs). $XBI WOW! hC Bioscience announces its first ever tRNA-based program for Duchenne muscular dystrophy (DMD). hC is developing anticodon engineered tRNAs as a potential treatment for DMD patients with shortened & nonfunctional dystrophin due to premature termination codons (PTCs).
2/Duchenne is a severe progressive disease which rapidly worsening children’s muscle function often using a wheelchair by early adolescence & eventually needing artificial ventilation to breathe. DMD is caused by mutations to dystrophin that affect about 300,000 males worldwide & PTCs account for approximately 26% of cases.Duchenne is a severe progressive disease which rapidly worsening children’s muscle function often using a wheelchair by early adolescence & eventually needing artificial ventilation to breathe. DMD is caused by mutations to dystrophin that affect about 300,000 males worldwide & PTCs account for approximately 26% of cases.
3/A nonsense mutation or premature termination codons (PTCs) is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in DuchenneA nonsense mutation or premature termination codons (PTCs) is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne
Read 7 tweets

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