Aron Jaffe
@jaffeab.bsky.social
SVP @ https://curie.bio/; Former SVP Head of Research @chromamedicine; EIR @ThirdRockV; Reg Med, uBiome group leader/Resp DA co-lead @NovartisScience. Opinions my own.
First game of the season with the boy. Never gets old.
April 6, 2025 at 6:13 PM
First game of the season with the boy. Never gets old.
This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9
9/9
February 10, 2025 at 2:05 PM
This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9
9/9
Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, & we demonstrated that a single administration led to rapid and sustained, 90% reduction of PCSK9 protein, resulting in a clinically meaningful 70% reduction in LDL-C in non-human primates
7/9
7/9
February 10, 2025 at 2:05 PM
Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, & we demonstrated that a single administration led to rapid and sustained, 90% reduction of PCSK9 protein, resulting in a clinically meaningful 70% reduction in LDL-C in non-human primates
7/9
7/9
Not only was this methylation-driven silencing durable in a homeostatic liver, but both silencing and methylation were fully maintained during liver regeneration, indicating durability through cell division in vivo!
6/9
6/9
February 10, 2025 at 2:05 PM
Not only was this methylation-driven silencing durable in a homeostatic liver, but both silencing and methylation were fully maintained during liver regeneration, indicating durability through cell division in vivo!
6/9
6/9
We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse!
5/9
5/9
February 10, 2025 at 2:05 PM
We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse!
5/9
5/9
We designed an mRNA-based #epigeneticeditor: DNA-binding domain fused to a DNA methyl-transferase domain & a transcriptional repressor domain which, together w/ human PCSK9-targeted gRNA, deeply & specifically methylated & silenced PCSK9 in primary human hepatocytes in vitro
4/9
4/9
February 10, 2025 at 2:05 PM
We designed an mRNA-based #epigeneticeditor: DNA-binding domain fused to a DNA methyl-transferase domain & a transcriptional repressor domain which, together w/ human PCSK9-targeted gRNA, deeply & specifically methylated & silenced PCSK9 in primary human hepatocytes in vitro
4/9
4/9
This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9
9/9
February 10, 2025 at 12:43 PM
This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9
9/9
Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, and we demonstrated that a single administration led to rapid and sustained, 90% reduction of PCSK9 protein, resulting in a clinically meaningful 70% reduction in LDL-C
7/9
7/9
February 10, 2025 at 12:43 PM
Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, and we demonstrated that a single administration led to rapid and sustained, 90% reduction of PCSK9 protein, resulting in a clinically meaningful 70% reduction in LDL-C
7/9
7/9
Not only was this methylation-driven silencing durable in a homeostatic liver, but both silencing and methylation were fully maintained during liver regeneration, indicating durability through cell division in vivo!
6/9
6/9
February 10, 2025 at 12:43 PM
Not only was this methylation-driven silencing durable in a homeostatic liver, but both silencing and methylation were fully maintained during liver regeneration, indicating durability through cell division in vivo!
6/9
6/9
We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein by 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse!
5/9
5/9
February 10, 2025 at 12:43 PM
We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein by 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse!
5/9
5/9
We designed an mRNA-based #epigeneticeditor: DNA-binding domain fused to a DNA methyl-transferase domain & a transcriptional repressor domain which, together w/ human PCSK9-targeted gRNA, deeply & specifically methylated & silenced PCSK9 in primary human hepatocytes in vitro
4/9
4/9
February 10, 2025 at 12:43 PM
We designed an mRNA-based #epigeneticeditor: DNA-binding domain fused to a DNA methyl-transferase domain & a transcriptional repressor domain which, together w/ human PCSK9-targeted gRNA, deeply & specifically methylated & silenced PCSK9 in primary human hepatocytes in vitro
4/9
4/9