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Yair Einhorn Profile picture
@yaireinhorn
Dec 13, 2022 • 7 tweets • 11 min read • Read on X
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1/@GraphiteBio presented preclinical results supporting the use of a single-#cell #RNA #sequencing method to assess #gene correction outcomes in #patients treated with nulabeglogene autogedtemcel (nula-cel) - GPH-101. #BioTech #CRISPR #GeneEditing #Genomics $GRPH #ASH22 GraphiteBio presented precl...
2/GPH101 (nula-cel) is a #CRISPR #Cas9 #GeneEditing autologous #stem #cell-based #therapy in clinical development aimed to treat #SickleCellDisease. GPH101 is designed to directly correct the underlying mutation, thereby decreasing HbS production & restoring HbA expression #ASH22 GPH101 (nula-cel) is a CRIS...
3 $GRPH gene correction platform involves editing hematopoietic #stem cells found in the #bonemarrow that develop into various types of #blood #cells. Since red blood cells lose their genomic DNA during maturation-tracking #GeneEditing in mature cells via sequencing is impossible Graphite bio’s gene correct...
4/The problem of the inability to mesure the efficacy of #GeneEditing in #SCD patients was solved by using immature red blood cells called #reticulocytes which retain their RNA and can be sequenced in order to assess the #gene correction levels after using nula-cel. #ASH22 The problem of the inabilit...
5/Based on this knowledge, $GRPH sought to develop a single-cell RNA sequencing method that could measure #GeneEditing outcomes in reticulocytes. It first measured the genetic makeup of reticulocytes from healthy donors-AA, people with SCD trait-AS, & with #sicklecelldisease-SS. Based on this knowledge, gr...
6/Results from both experiments demonstrated the single-cell RNA sequencing method’s ability to precisely and reproducibly measure and differentiate the healthy donors - AA, people with SCD trait - AS and #SickleCellDisease patients - SS. #ASH22 Results from both experimen...
7/These data support the use of Single-cell #RNA sequencing of #reticulocytes to determine initial #GeneEditing outcomes in #SickleCellDisease patients treated with nula-cel (GPH101) thus supporting the clinical development of @GraphiteBio’s investigational #therapy. #ASH22 These data support the use ...

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More from @yaireinhorn

Yair Einhorn Profile picture
Mar 2
🚨🚨WOW! $NTLA has just announced that the FDA has removed the clinical hold on @intelliatx’s Investigational New Drug application (IND) for its MAGNITUDE Phase 3 clinical trial of nexiguran ziclumeran (nex-z) for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) after it has already lifted the clinical hold from Intellia’s MAGNITUDE-2 Phase 3 clinical trial for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This is a huge win for the entire CRISPR and Gene Editing! $XBIWOW! NTLA announced that the FDA has removed the clinical hold on intellia’s Investigational New Drug application (IND) for its MAGNITUDE Phase 3 clinical trial of nexiguran ziclumeran (nex-z) for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) after it has already lifted the clinical hold from Intellia’s MAGNITUDE-2 Phase 3 clinical trial for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This is a huge win for the entire CRISPR and Gene Editing! $XBI
2/ATTR amyloidosis (ATTR) is a rare, progressive disease characterized by the abnormal buildup of amyloid deposits composed of misfolded transthyretin protein in the body’s organs and tissues. Because symptoms of ATTR amyloidosis are often similar to those of other diseases, the condition can be difficult to recognize and diagnose properly. However, ATTR amyloidosis is fatal if untreated, with a life-expectancy ranging between 3-15 years after the onset of symptoms, so early identification, diagnosis, and treatment are critical.Image
3/Nex-z or $NTLA-2001 is a CRISPR/Cas9-based in vivo Gene Editing therapy which has the potential to become the first ever one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. $XBI Nex-z or NTLA-2001 is a CRISPR/Cas9-based in vivo Gene Editing therapy which has the potential to become the first ever one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein.
Read 6 tweets
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
Aug 11, 2025
1/Here’s a great @nytimes story about the rise & fall of Novo Nordisk - from a huge success story with its first GLP-1 blockbuster drug - Ozempic, leading the Obesity market with 13M prescriptions in the U.S into losing it all to its nemesis - Eli Lilly! $LLY $NVO $HIMS $XBI 🧵👇 Here is a great New York Times story about the rise & fall of Novo Nordisk - once an amazing success story with its GLP-1 blockbuster drug - Ozempic, reigning the obesity market with 13M prescriptions in the U.S, but lost it all to its nemesis - Eli Lilly.
2/There is no doubt that $NVO was the first company which significantly developed the Obesity and weigh-loss field by introducing its breakthrough drug - Ozempic. Not only did Ozempic succeed in reducing weight but it also successfully reduced the risk of heart attacks and other health risks thus causing $NVO value to increase.There is no doubt that Novo Nordisk was the first company which significantly developed the Obesity and weigh-loss field by introducing its breakthrough drug - Ozempic. Not only did Ozempic succeed in reducing weight but it also successfully reduced the risk of heart attacks and other health risks thus causing Novo Nordisk’s value to increase.
3/But it seems that $NVO golden age is over. A decisive loss in its fierce competition against $LLY 🧵👇- who has managed to successfully introduce more efficient & reliable obesity drugs into the market, has led to a 50% fall in Novo Nordisk’s share price and to the departure of its CEO. $XBIBut it seems that Novo Nordisk golden age is over. A decisive loss in its fierce competition against Eli Lilly who has managed to successfully introduce more efficient and reliable obesity drugs into the market, has led to a 50% fall in Novo Nordisk’s share price and to the departure of its CEO.
Read 15 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

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