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Yair Einhorn Profile picture
@yaireinhorn
Mar 4 • 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
Jan 30
1/@Tome_bio - a #GeneEditing company based on programmable genomic integration (PGI) platform has recently launched & raised $213M in Series A & B funding from leading investors: @a16zBioHealth, @GVteam, @FujifilmUS, #ARCHVenturePartners, @bruker & others. #BioTech #CRISPR $XBI Tome Biosciences - programmable genomic integration company, has launched to usher in a new era of genomic medicines based on programmable genomic integration (PGI). PGI enables the insertion of any DNA sequence, of any size, into any programmed genomic location. The Company has raised 213 million dollars in Series A and B funding from investors: Andreessen Horowitz (a16z) Bio + Health, ARCH Venture Partners, GV, Longwood Fund, Polaris Partners, Bruker Corporation, FUJIFILM Corporation, Alexandria Venture Investments and others.
2/PGI - programmable genomic integration - combines the site-specificity of #CRISPR/Cas9 with enzymes capable of inserting or writing sequences of DNA, including entire genes, without the need for double-strand DNA breaks. @Tome_bio’s most advanced PGI technology, called integrase-mediated PGI (I-PGI), utilizes proprietary integrases & is based on groundbreaking PASTE technology first discovered by Tome’s Co-
Founders, @omarabudayyeh & @jgooten - while at @MIT as investigators.PGI - programmable genomic integration - combines the site-specificity of #CRISPR/Cas9 with enzymes capable of inserting or writing sequences of DNA, including entire genes, without the need for double-strand DNA breaks. Tome bio’s most advanced PGI technology, called integrase-mediated PGI (I-PGI), utilizes proprietary integrases & is based on groundbreaking PASTE technology first discovered by Tome’s Co- Founders, Omar abudayyeh and Jonathan Gootenberg - while at MIT as investigators.
@Tome_bio 3/@Tome_bio’s I-PGI can insert large
DNA sequences anywhere in the genome with unprecedented precision. Thus far, I-PGI has demonstrated insertions of more than 30kb of genetic code with site-specificity in multiple different cell types & can enable complex cell engineering. Tome bio’s I-PGI can insert large DNA sequences anywhere in the genome with unprecedented precision. Thus far, I-PGI has demonstrated insertions of more than 30kb of genetic code with site-specificity in multiple different cell types and can enable complex cell engineering.
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Yair Einhorn Profile picture
Jan 22
1/@PrimeMedicine has issued a corporate update-as part of its #JPM2024 presentation in which $PRME reported: 1)recent business progress 2) its strategic priorities for 2024 3)presentation of the clinical progress it achieved in 2023 #BioTech #CRISPR #GeneEditing #JPM24 🧵👇 PrimeMedicine has issued a corporate update - as part of its JP Morgan healthcare conference presentation in which Prime reported: 1)recent business progress 2) its strategic priorities for 2024 3)presentation of the clinical progress the company achieved in 2023
2/@PrimeMedicine’s proprietary platform - #PrimeEditing is the only #GeneEditing technology which can edit, correct, insert or even delete large DNA sequences in any target tissue - thus making it the most advanced & promising editing technology with $PRME owing its full rights Prime medicine’s proprietary platform - Prime editing is the only Gene editing technology which can edit, correct, insert or even delete large DNA sequences in any target tissue - thus making it the most advanced and promising editing technology with Prime owing its full rights
@PrimeMedicine 3/In my opinion - the most significant statement presented in #JPM2024 was $PRME decision to restructure its pipeline by aligning it into 4 core pillars - #hematology, #Liver, #Ocular & #Neuromuscular - thus extending @PrimeMedicine’s existing financial resources & cash runway In my opinion - the most significant statement presented in JPM2024 was Prime’s decision to restructure its pipeline by aligning it into 4 core pillars - hematology, Liver, Ocular and Neuromuscular - thus extending Prime Medicine’s existing financial resources and cash runway
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Yair Einhorn Profile picture
Jan 19
1/@MeiraGTx announced an asset purchase agreement with @JanssenUS - a @JNJNews company, for its remaining interests in bota-vec for the treatment of XLRP & a technology transfer agreement for bota-vec manufacturing. #BioTech $JNJ #RetinitisPigmentosa #Ophthalmology #Choroideremia MeiraGTx Holdings plc - a vertically integrated, clinical stage gene therapy company, today announced an asset purchase agreement with Janssen Pharmaceuticals - a Johnson and Johnson company, for the remaining interests in bota-vec for the treatment of XLRP, as well as a commercial supply agreement and a technology transfer agreement for bota-vec manufacturing.
2/#RetinitisPigmentosa is a group of rare genetic eye diseases that damage light-sensitive cells in the retina thus leading to loss of sight. In ~10% of RP cases, the gene is passed from the mother to her children resulting in a form of #RP known as #XLRP.
3/@MeiraGTx has already presented very promising data from its phase 1/2 study of AAV-RPGR in patients with XLRP demonstrating significant improvement from baseline compared to untreated eyes and a favourable safety profile. Here is a 🧵which I’ve written for further reading👇
Read 6 tweets
Yair Einhorn Profile picture
Jan 16
1/@VertexPharma announced today that the @US_FDA has approved CASGEVY (exagamglogene autotemcel [exa-cel]), a #CRISPR/Cas9 #GeneEditing #CellTherapy for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. #BioTech @CRISPRTX $CRSP $VRTX Vertex Pharma announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVYTM (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older
2/CASGEVYTM is a non-viral,ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break.CASGEVYTM is a non-viral,ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break.
3/This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen- carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen- carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.
Read 5 tweets
Yair Einhorn Profile picture
Jan 10
1/@BeamTx has issued a corporate update - as part of its #JPM2024 presentation in which $BEAM reported: 1)recent business progress 2)highlighted its strategic priorities for 2024 3)presented the clinical progress the company achieved in 2023. #BioTech #CRISPR #GeneEditing #JPM24 Beam Therapeutics - a biotechnology company developing precision genetic medicines through base editing, today reported progress across the company’s hematology and genetic disease portfolios and provided updates on anticipated upcoming milestones.
2/@BeamTx’s most advanced venture is its Sickle Cell Disease Franchise. By trying to find a long-term treatment - $BEAM has developed a 3 Waves strategy for SCD with the intention to progressively expand the reach of its #BaseEditing platform to broader subsets of SCD patients. Beam is pursuing a long-term, staged development strategy for SCD that has three Waves of innovation intended to progressively expand the reach of our base editing approach to broader subsets of patients.
@BeamTx 3/BEAM-101 is an autologous cell therapy designed to efficiently & uniformly increase fetal hemoglobin (HbF) in red blood cells without relying on double stranded breaks, thus improving HbF induction & lowering residual disease-causing hemoglobin S in #SickleCell patients. BEAM-101 is an autologous investigational cell therapy designed to efficiently and uniformly increase fetal hemoglobin (HbF) in red blood cells without relying on double stranded breaks, offering a potentially best-in-class profile. Preclinical models suggest base editing could lead to improved HbF induction and lower residual disease-causing hemoglobin S compared to existing gene therapy options.
Read 15 tweets
Yair Einhorn Profile picture
Jan 6
1/@intelliatx has issued an update highlighting its strategic priorities & future milestones for 2024. Here’s my 🧵which summarises $NTLA updates of its prioritised pipeline, the progress made in 2023 & its current corporate status. #BioTech #CRISPR #JPM24 #GeneEditing #JPM2024 Intellia Therapeutics - a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced its strategic priorities through 2026 and key anticipated 2024 milestones
2/@intelliatx’s 2024-6 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 #GeneEditing programs outside the liver
4. DNA writing 2024 – 2026 Strategic Priorities A. Complete patient enrollment for pivotal studies of NTLA-2001 and NTLA-2002, including planned biologics license application (BLA) submission for NTLA-2002 in 2026 B. Launch clinical trials for next wave of in vivo and ex vivo programs, validating CRISPR-based in vivo targeted gene insertion and an allogeneic cell engineering solution designed to avoid NK cell-mediated rejection; and C. Expand the range of diseases that can be targeted by deploying new editing and delivery innovations, including advancing gene editing in tissues outside the liver and DNA w...
@intelliatx 3/After restructuring its clinical portfolio by focusing on its main #GeneEditing programs - $NTLA - 2001 / 2002 & 3001 - which will be the Company’s focus in the next three years, $NTLA anticipates reaching the following key clinical milestones during 2024👇 After restructuring its clinical portfolio by focusing on its main Gene editing programs - Intellia - 2001 / 2002 & 3001 - which will be the Company’s focus in the next three years, Intellia anticipates reaching the following key clinical milestones during 2024
Read 11 tweets

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