Prime Editing has potential to be best-in-class approach for AATD ---- Previously undisclosed program now emerging from within liver platform; ...

Prime Editing has potential to be best-in-class approach for AATD -- -- Previously undisclosed program now emerging from within liver platform; leverages proprietary, universal liver LNP -- -- High ...

Completed dosing of first two cohorts in Phase 1/2a REWRITE Clinical Study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD); Interim ...

"WVE-007 is also Wave's first GalNAc-siRNA to enter the clinic and utilizes our proprietary chemistry. Our expected clinical data this year will provide us with an early look into WVE-007's ...

Korro expects to nominate a development candidate for its rare metabolic disorder program targeting the liver (GalNAc) with subcutaneous delivery to create de novo protein variants by the end of 2025.

The LNP used is the same as for Verve-102, the GalNAc-LNP. A first patient has been dosed in the Pulse-1 open-label Phase 1b clinical trial (enrollees must require additional LDL-C lowering in ...

And second, GalNAc-conjugates allow for a highly specific and efficient targeting deliver cells in a binary silencing leads to lower active and E levels, resulting in higher adipose like policies ...

This molecule, called GalNAc is able to pass into liver cells, called hepatocytes, where huge numbers of proteins are manufactured. As well as developing this delivery process, the company also ...

The results come from the phase 1b/2a RestorAATion-2 study of WVE-006, a GalNAc-conjugated oligonucleotide that is designed to correct a mutation in mRNA associated with AATD that prevents ...

Alnylam Pharmaceuticals has developed GalNAc conjugates such as Amvuttra (vutrisiran), which enable targeted delivery of ...

Several biotech companies are exploring tissue-targeting approaches to improve drug delivery. Alnylam Pharmaceuticals has developed GalNAc conjugates such as Amvuttra (vutrisiran), which enable ...