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
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
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.
4/A feature of the starting #cell line is the targeted deletion of the B2M gene, which is designed to reduce the immunogenicity of product candidates derived from the lines by inhibiting rejection by CD8+ T cells.
5/@LineageCell & $ERNA intend to initiate additional planned #GeneEditing platforms that may further differentiate the cell line from others currently in use by competitors. Financial terms of the collaboration between $LCTX & $ERNA were not disclosed.
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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
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
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
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
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👇