Discover and read the best of Twitter Threads about #Autologous

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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
Read 17 tweets
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...
(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?
Read 30 tweets
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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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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Read 13 tweets

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