Discover and read the best of Twitter Threads about #celltherapy
Most recents (14)
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 🧵 
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
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
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👇
Really chuffed to share our latest work from Sanjana Lab out in @nature today (nature.com/articles/s4158…).
We tested >12,000 genes to find positive regulators of T cell proliferation to be used for next-gen #immunotherapies.
A thread...
We tested >12,000 genes to find positive regulators of T cell proliferation to be used for next-gen #immunotherapies.
A thread...
(2/28) T cell therapies have shown the potential to cure patients from #cancer – but even in B cell cancers, relapses outnumber cures… One of the problems is limited T cell persistence. #celltherapy #CARTcell
(3/28) We wanted to find new genes (including genes never expressed in T cells) that could improve T cell persistence. Now, the question was: should we use #crispr activation or directly deliver target genes on a #lentivirus?
1/ Excited to share how T cell therapies kill #leukemia!! multi-omics + new #computational #singlecell tools for longitudinal analysis 👉unexpected answer! cell.com/cell-reports/f…
*👏* @elhamazizi! 🙏 @dpeer Cathy Wu @MDAndersonNews @CPRITTexas @ColumbiaBME @sloan_kettering
*👏* @elhamazizi! 🙏 @dpeer Cathy Wu @MDAndersonNews @CPRITTexas @ColumbiaBME @sloan_kettering
2/ We studied donor lymphocyte infusion (DLI) - an #Immunotherapy for relapsed #leukemia after #BMT & the #og of #celltherapy. Previously, we showed DLI reversed T cell exhaustion - but didn't know why/how/which T cells were responsible...
ashpublications.org/blood/article/…
ashpublications.org/blood/article/…
3/ To address these ?'s, we modeled intraleukemic T cell dynamics by integrating longitudinal, multimodal data from ~100K T cells (!) during response (R) or resistance (NR: nonresponder) to DLI.
The chase for an #HIV cure has been elusive for decades with as many failures as seen with CNS diseases. $SGMO has been dedicated to this effort tho are now supporting sponsors given the cost of the effort. This stream will provide an recap. #Autologous #CellTherapy
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$SGMO began this clinical effort in 2009. They have worked with UPenn, City of Hope, Case Western University. NAID and CIRM have helped with funding among others. We should see some critical updates this year.
#HIV
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#HIV
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The first trial (sponsored by UPenn) SB-728 began in 2009 focused on HAART treated patients and was focused on safety and comparison/persistence of CD4/CD8 T cells after ZFN modifed autologous T cells were infused.
#HIV #CellTherapy
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#HIV #CellTherapy
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$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A
#GeneTherapy
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#GeneTherapy
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$SNY #GMU head of Rare and Neurologic Disease Research is Christian Mueller. Video into is pretty good
Addl heads include Bruno Figueroa (CMC) and Cate O'Riordan (translational unit)
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Addl heads include Bruno Figueroa (CMC) and Cate O'Riordan (translational unit)
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$SNY #GMU Principal Scientist Robert Jackson profile says he is working on: Huntingtin's, MLD, PKU, Neurophathies, T1Diabetes Hypertension, Nephrophaty, vascular disease, Rheumatoid Arthritis and sensorineural hearing loss
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Part II of the $CRSP $SGMO comparison will hopefully be a bit less dry now that Part I updates are in place. This thread will focus more on valuation, outlook and why the valuation cycle should matter to investors.
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Let's start with the concept of #Disruptive technologies. The originator of disruptive innovation theory, Clayton Christensen worked with HBR in 2015 to revisit the past 20 years.
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hbr.org/2015/12/what-i…
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hbr.org/2015/12/what-i…
#GenomicMedicine is disrupting Big Pharma who has begun to respond by spinning off old product lines and jumping into #GeneTherapy.
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This is the third edition of my tweetstorms on #GeneTherapy #CellTherapy #GeneEditing. This one compares $SGMO to $CRSP and attempts to look at #GenomeMedicine from the perspectives laid out by C.Woods at ARK This is part one of the series. Pt 2 tomorrow AM 1/20
The charts of both are attached and useful context. $CRSP went public in 2016. The weekly chart is attached and notes the pct ownership of $ARKG $ARKK since CY18. Prior the stock was stagnant below the support/resistance levels show. 2/20
I'm including two charts for $SGMO. Wkly and Mthly. The weekly shows they were pulled along with the #geneediting group The monthly shows this rally/bust cycle is not unique. Compare the SGMO 2017-2018 to CRSP. Pretty similar. More later. 3/20
$ARKG Tweet series
This series of tweets is my opinion only. Cathie Wood has created a group of ETFs that target the disruptive innovation that is both happening and accelerating. These funds have had stellar performance based upon innovation trends and economic theory.
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This series of tweets is my opinion only. Cathie Wood has created a group of ETFs that target the disruptive innovation that is both happening and accelerating. These funds have had stellar performance based upon innovation trends and economic theory.
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The $ARKG fund description is a good place to start. This has changed recently to a #CRISPR theme but started more with the sequencing cost declines which would drive diagnostics adoption and precision medicine
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What's up with $SGMO valuation? This thread will try to show some history and the setup for what I still hope to be an outsized move. First worth noting that news will always trump technicals. First here is a month chart I've been using for the company 1/
This being a 10 year chart, it doesn't show how long $SGMO has been around but it does show virtually no trading volume and a 3 yr ceiling on the price as they were in early development stage while trying to get ill-chosen indications into pre/clinic 2/
The first wave $SGMO rally started in 2013 when the ceiling was broken. The company was talking up #GeneEditing #GeneRegulation #HIVcure and #diabetes None were ready but the volume picked up and the first big rally ensued going from around $9 to $20 3/
Time to catch up on some #Senescence papers, starting with beautiful work by @corina_amor_MD, Judith Freucht & @JosefLeibold. They used #CART #CellTherapy to clear #Senescent cells, and you'll never guess what happened next! nature.com/articles/s4158…
Just kidding, you guessed it: disease model mice got better. Props to the authors for A) inducing #Senescence in 3 different ways, B) using 2 models of #NASH/liver #Fibrosis, and C) validating their senescence observations in human samples of #Cancer and #Atherosclerosis.
The linchpin of the paper was identifying a specific membrane marker on #Senescent cells, uPAR. They used bulk #Transcriptomics to identify candidates, then narrowed down with #Proteomic data. Go #Omics!
They didn't explore whether uPAR is causative for the #Senescent phenotype.
They didn't explore whether uPAR is causative for the #Senescent phenotype.
Good review of natural killer (NK) cells for #cancer immunotherapy from @NatRevDrugDisc: nature.com/articles/s4157…
#CellTherapy $TAK $FATE $NK @CellMedica & Century Therapeutics
#CellTherapy $TAK $FATE $NK @CellMedica & Century Therapeutics
@NatRevDrugDisc @CellMedica A Ph 1/2 study from @MDAndersonNews showed promising clinical data of HLA-mismatched anti-CD19 CAR-NK #CellTherapy in NHL/CLL #cancer patients (n=11): 73% ORR & 64% CR(!)
@NEJM paper: nejm.org/doi/full/10.10…
$TAK has licensed this product. Competitively relevant to $FATE.
@NEJM paper: nejm.org/doi/full/10.10…
$TAK has licensed this product. Competitively relevant to $FATE.
@NatRevDrugDisc @CellMedica @MDAndersonNews @NEJM $GILD Gilead, Kite and oNKo-innate announced a research collaboration to discover cancer immunotherapies focused on natural killer (NK) cells.
Gilead will pay an undisclosed upfront and milestones. $KITE @oNKo_innate
Relevant to $FATE $JNJ $TAK $NK
gilead.com/news-and-press…
Gilead will pay an undisclosed upfront and milestones. $KITE @oNKo_innate
Relevant to $FATE $JNJ $TAK $NK
gilead.com/news-and-press…
Not sure what to expect at the 2019 BHP Research Showcase? Our free public event will be held at @qehbham on Fri 17 May, where you can get hands-on with our health research. If you want to #BePartOfResearch, take a look through our exhibitor info THREAD > birminghamhealthpartners.co.uk/research-showc…
Our #cardiology stand will focus on current studies that are changing & saving the lives of our patients. Our cardiology team is one of the largest in the UK and works closely with @ICVS_UoB to treat every type of #heartdisease
#BePartOfResearch
#BePartOfResearch
.@UmbrellaHealth is the home of #sexualhealth services for Brum & Solihull. They'll be here with interactive displays highlighting research into #HIV and #STIs, including #antimicrobialresistance and bacterial vaginosis >> umbrellahealth.co.uk
#BePartOfResearch
#BePartOfResearch
Excellent overview of the cell therapy landscape from Piper Jaffray
$GILD $NVS $CELG $JNJ $AMGN $ALLO $BLUE $IOVA $AUTL $SGMO $ATRA $CRSP $FATE $CLLS $NTLA $DTIL $ZIOP $MRKR $CYAD $BLCM $TCRR $ADAP $MBIO $NTGN $UMRX $MXCT $GMDA $SRNE $KDS $MDGEF $NK $ANIX $KITE $JUNO #ASGCT19
$GILD $NVS $CELG $JNJ $AMGN $ALLO $BLUE $IOVA $AUTL $SGMO $ATRA $CRSP $FATE $CLLS $NTLA $DTIL $ZIOP $MRKR $CYAD $BLCM $TCRR $ADAP $MBIO $NTGN $UMRX $MXCT $GMDA $SRNE $KDS $MDGEF $NK $ANIX $KITE $JUNO #ASGCT19
New article from @NatRevDrugDisc looks at the global #CellTherapy landscape: nature.com/articles/d4157…
Now, the global cell therapy pipeline includes 1,011 active agents, 258 more than a year ago.
#ASCO19
Now, the global cell therapy pipeline includes 1,011 active agents, 258 more than a year ago.
#ASCO19
@NatRevDrugDisc Guggenheim highlights next-generation #CellTherapy approaches
1st gen: $ADAP $AMGN $ATRA $AUTL $BLCM $BLUE $CELG $CBMG $CYAD $FBIO $GILD $GSK $XON $IOVA $JNJ $MXCT $MDGEF $NVS $SRNE $TCRR $ZIOP
Next-gen: $ALLO $ATRA $CLLS $DTIL CYAD $BLCM $EVTCY $FATE $EDIT $RUBY $ERYP
#ASCO19
1st gen: $ADAP $AMGN $ATRA $AUTL $BLCM $BLUE $CELG $CBMG $CYAD $FBIO $GILD $GSK $XON $IOVA $JNJ $MXCT $MDGEF $NVS $SRNE $TCRR $ZIOP
Next-gen: $ALLO $ATRA $CLLS $DTIL CYAD $BLCM $EVTCY $FATE $EDIT $RUBY $ERYP
#ASCO19
