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Yair Einhorn Profile picture
@yaireinhorn
Feb 25, 2023 • 17 tweets • 25 min read • Read on X
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1/As promised & after reading $CRSP latest Q4 2022 #financial #report here is my impression regarding @CRISPRTX latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #BioTech #FinTwit #Genomics #GeneEditing 🧵 CRISPR Therapeutics, a biopharmaceutical company focused on
2/IMO the most significant corporate event in Q4 was $CRSP announcement that regulatory Exa-cel submissions of both #SickleCell & #BetaThalassemia validated in the #EU & #UK & that the @US_FDA BLA submission is on track by the end of Q1 ‘23 - possibly reaching the #markets in ‘23 Regulatory submissions complete for exagamglogene autotemcel
3/Exa-cel/CTX001 - the key program in $CRSP portfolio, is an #autologous Ex-Vivo #CRISPR/#Cas9 #GeneEditing therapy aimed for patients suffering from #TDT or severe #SCD. The latest readout for both programs was phenomenal with 42/44 TDT & 31/31(!) demonstrated remarkable results 3/Exa-cel - formally known as CTX001 and the key program in
4/Last December - during #ASH22 @CRISPRTX & $VRTX presented an updated clinical data from the exa-cel trail taken from 75 #patients: 44 TDT+31 #sicklecelldisease & with a long follow-up. The data demonstrates that exa-cel has the potential to be a one-time functional #cure.
5/Exa-cel was submitted to the @US_FDA for BLA rolling review with expected completion of the submission package by the end of Q1 2023. If approved - Exa-cel will be the first ever #CRISPR product to be commercialised & marketed thus making @CRISPRTX the first to sell a product. Exa-cel was submitted to the _FDA for BLA rolling review wit
6/@CRISPRTX second program is CTX110 - a wholly owned donor-derived #GeneEditing #allogeneic antigen receptor T cell #CAR-T #therapy targeting CD19+ B-cell malignancies. CTX110 has also been granted a Regenerative Medicine Advanced Therapy - #RMAT designation from the @US_FDA. CRISPR therapeutics second program is CTX110 - a wholly owne
7/Patients that were treated with CTX110 as part of the @CRISPRTX #CARBON #clinicaltrail have shown good results. CTX110 was well tolerated across all dose levels & patients in CR remain clinically well without receiving any systematic anti-#Cancer #Therapy other than CTX110👇 Patients that were treated with CTX110 as part of the CRISPR
8/@CRISPRTX has recently provided a clinical update for both its Part A & Part B of $CRSP ongoing Phase 1 #CARBON trial evaluating the safety and efficacy of CTX110 its wholly-owned #allogeneic CAR T #celltherapy targeting CD19+ B-#cell malignancies. Here is a 🧵 that I wrote👇
9/@CRISPRTX will initiate clinical trials for CTX112 - next gen CAR T platforms targeting CD19+ B-cell malignancies, in 1H 2023. CTX112 incorporates the edits in CTX110 plus additional edits to the genes encoding Regnase-1 & TGFBRII, thus increasing the potency of the CAR T cells CRISPR Therapeutics will initiate clinical trials for CTX112
10/CTX130 is another $CRSP wholly-owned #allogeneic CAR-T #CellTherapy targeting #CD70, for the treatment of Mycosis Fungoides & #Sézary Syndrome - both types of cutaneous T-cell #lymphoma. In September @CRISPRTX announced that the @US_FDA has granted it RMAT designation. CTX130TM is a wholly-owned allogeneic CAR T cell therapy tar
11/CTX130 has showed overall a good safety profile - patients that were treated with CTX130 as part of the @CRISPRTX #COBALT #clinicaltrail have shown good results with #disease control rate DCR of 90% (N=10), 70% ORR & 30% CR rate. $CRSP CTX130 recent readout was also promising
12/Two interesting collaboration that @CRISPRTX’s has entered recently are: 1)@MoffittNews for developing new #autologous CAT-T #CellTherapy targeting CD83 for treating #AML. 2)@RoswellPark for developing new autologous CAT-T #therapy targeting GPC3 against solid #tumors. #Cancer
13/@ViaCyte & $CRSP have 3 #CRISPR #GeneEditing programs - VCTX210 in which the first patient was dosed as part of a Phase 1 clinical trial. $CRSP expects to move the other two In-Vivo programs - VCTX211 & VCTX212 - both for #diabetes into the clinic in the next 18-24 months👇 Following the previously-announced clearance of the Clinical
14/@CRISPRTX has named recently another 2 new #CardioVascular programs 1)CTX310 for #ANGPTL3 & 2)CTX330 for PCSK9. Regarding both - IMO $VERV is much more advanced & especially after @VerveTx’s recent data & the ongoing #Heart-1 trail it is most likely to dominate this market. CRISPR Therapeutics continues to advance a number of program
15/As of 12/31/22 $CRSP had capital resources of $2.24B & R&D expenses of $103M. IMO @CRISPRTX’s current cash position will enable it to continue to develop its clinical pipeline with hopefully the first #CRISPR commercial product hitting the markets in 2023 & generating revenue Cash Position: Cash, cash equivalents and marketable securit
16/With an highly anticipated first ever @US_FDA approval for a #GeneEditing platform expected in 2023 @CRISPRTX will be the 1ST #CRISPR company with a product in the market & with a strong cash position of $2.24B - @CRISPRTX IMO continues to look very solid & promising. $CRSP With an highly anticipated first ever FDA approval for a Gen
And as always please do feel free to #share & #retweet so that those on #Twitter / #FinTwit who are interested in #GeneEditing, #CRISPR, #BioTech & #Genomics will get this relevant data. Wishing you all a great #Week!

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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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