Yair Einhorn Profile picture
Feb 25 17 tweets 25 min read
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

Feb 23
1/@GraphiteBio announced that after reviewing its business, it decided to discontinue any further development of nulabeglogene autogedtemcel (nula-cel) for #SickleCell disease, to explore a range of strategic alternatives & to reduce 50% in its workforce! #CRISPR #BioTech $GRPH Graphite Bio today announce...
2/@GraphiteBio is also exploring the potential to continue nula-cel development externally. $GRPH intends to continue research activities associated with its early-stage non- genotoxic conditioning program, with the goal of advancing toward potential development candidates.👇
3/In addition, @GraphiteBio’s Board of Directors has approved a corporate restructuring that would reduce the company’s workforce by approximately 50%, among other actions to reduce cash burn while the company explores strategic alternatives. $GRPH Image
Read 5 tweets
Feb 22
1/@LineageCell announced it entered an exclusive option & license agreement with $ERNA for the development of novel B2M deficient induced pluripotent #stem #cell #iPSC by using Eterna’s #GeneEditing technology thus developing therapies for #CNS diseases. #BioTech #CRISPR $LCTX Lineage Cell Therapeutics, ...
2/The new cell lines - to be developed by $ERNA, will support the creation of additional product candidates at $LCTX and specifically for the treatment of central #nervoussystem #CNS disorders & other #neurology targets via Eterna’s IP #CRISPR collaboration with @FactorBio The new cell lines to be de...
3/This agreement will also allow @LineageCell to enter the #CRISPR field and will provide $LCTX an option to obtain an exclusive license to utilize & sublicense $ERNA novel #GeneEditing cell lines for #preclinical, #clinical, & commercial purposes in the field of CNS diseases. This agreement will also al...
Read 5 tweets
Dec 13, 2022
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...
Read 7 tweets
May 21, 2022
1/As promised & after reading $NTLA latest Q1 2022 financial report here is my analysis regarding @intelliatx latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #GeneEditing #BioTech #FinTwit #Genomics👇
2/NTLA-2001 is the most advanced In-Vivo #CRISPR/#Cas9 #GeneEditing program in $NTLA portfolio & is aimed to treat Transthyretin Amyloidosis - (ATTR). NTLA-2001 could potentially halt & reverse the disease in a “one time” treatment by knocking out the TTR gene with a single dose
3/In February $NTLA has presented an updated clinical data from its Phase 1 study of NTLA-2001 which included 15 patients with ATTR amyloidosis. IMO NTLA-2001 readout looks promising & here is a summary that I wrote shortly after @intelliatx’s presentation of NTLA-2001👇
Read 14 tweets

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