**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Zi Jun Emma Wang
YolTech Therapeutics
Shanghai, China

**Main Body of the Abstract**
YOLT-201 is an innovative therapeutic solution for treating Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) and Cardiomyopathy (ATTR-CM). YOLT-201 utilizes state-of-the-art in vivo lipid nanoparticle (LNP) technology to deliver the CRISPR/Cas9 gene editing tool to the liver precisely. By knocking out the TTR gene, YOLT-201 effectively suppresses the production of abnormal TTR protein and reduces its deposition in essential organs, especially the nervous system and cardiac muscle, known as transthyretin amyloidosis polyneuropathy (ATTR-PN) and fatal cardiomyopathy (ATTR-CM), giving severe health impact to patients. The primary goal of YOLT-201 is to alleviate clinical symptoms and extend their lifespan. Currently, YOLT-201 has undergone animal model experiments as well as preliminary clinical trials: a. GLP toxicity preclinical in vivo pharmacokinetic/pharmacodynamic studies demonstrated a rapid, safe, and significant reduction in serum prealbumin levels with targeted liver editing by YOLT-201 administration; b. Off-target assay and genome safety assay showed that YOLT-201 did not result in off-target editing (over 500 loci) or chromosomal structural abnormalities at genomic loci outside of the target site, nor did it introduced tumor-associated mutations (over 1,300 loci); c. in an investigator-initiated clinical trial study with two dose cohorts, a significant and rapid reduction in serum TTR protein levels was observed in ATTR-CM patients after intravenous administration of YOLT-201.



Treating Transthyretin Amyloidosis with CRISPR/Cas9 via LNP Delivery for in vivo TTR Knockdown PC99 (#82)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.