Top Tweets for #yoltech
YOLT-101
Another single-infusion #BaseEditor therapy for Heterozygous Familial Hypercholesterolemia
From #YolTech Therapeutics 🇨🇳
0.6 mg/kg N=3
Being effective in 1 wk
Sustained 70% ⬇️plasma PCSk9 & 50% ⬇️ plasma LDL-C till 16-wk
medRxiv 2025
https://t.co/QsKaf8gjOh
🇨🇳 #YolTech Therapeutics, a clinical-stage biotech company pioneering in vivo genome editing therapies, today announced the closing of its approximately $45 million (over RMB 300 million) Series B #financing led by the AstraZeneca-CICC healthcare investment fund. https://t.co/UkDstmu0tx #CGT
To date, YolTech Therapeutics has cumulatively raised over RMB 575 million.
Founded in 2021, YolTech is advancing next-generation in vivo gene-editing therapies designed for one-time treatment. The company has built a fully integrated platform encompassing proprietary CRISPR nucleases (YolCas), base editors (YolBE), and a cutting-edge lipid nanoparticle delivery system (Yol-LNPs), enabling precise, efficient, and tissue-specific gene editing across a broad range of therapeutic areas.
4 product of YolTech have entered the clinical stage:
1. YOLT-101, in vivo base editing therapy based on adenine base editor YolBE hpABE5 and a novel LNP delivery system. For reduce high LDL-C levels in patients with familial hypercholesterolemia (FH)
YOLT-101 has been approved for clinical trials in China, with 🇨🇳 Salubris obtaining the rights for development, manufacturing, and commercialization in China.
2. lead asset: YOLT-201, achieved excellent data in the IIT clinical study for treating transthyretin amyloid cardiomyopathy (ATTR-CM). It is a next-generation in vivo gene editing drug that delivers the CRISPR-Cas gene editing RNA system via lipid nanoparticles (LNP) to achieve efficient and specific editing of the transthyretin (TTR) target gene in the human liver.
3. YOLT-202 is based on YolTech's YolBE, for the treatment of alpha-1 antitrypsin deficiency (AATD).
4. YOLT-203 is an in vivo gene editing therapy that employs YolTech' CRISPR/Cas gene editing tool YolCas12HF and is precisely delivered to hepatocytes via the company's self-developed lipid nanoparticle (LNP) system. It targets the editing of the HAO1 gene to inhibit glycolate oxidase (GO) expression, thereby reducing oxalate production at the source for the treatment of patients with primary hyperoxaluria type 1 (PH1).
🇨🇳 #YolTech Therapeutics recently published a study titled "In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery" in Nature Biomedical Engineering, detailing its latest mouse study results. https://t.co/aKVh1mAo17
LNP-168 is a novel bone marrow-targeting lipid nanoparticle (LNP) carrier developed by screening an ionizable lipid library and optimizing the formulation.
This carrier efficiently delivers adenine base editor (ABE8e) mRNA and sgRNA targeting the γ-globin (HBG1/2) promoter region to hematopoietic stem cells (HSCs).
The platform eliminates the need for stem cell harvesting or chemotherapy-based myeloablative preconditioning.
In Vivo CRISPR Effectively Edits Human Blood Stem Cells Engrafted in Mice
If translated to the clinic, this strategy could provide a less invasive, potentially one-time treatment for inherited #blooddisorders
#YolTech #CRISPR #HSCs
https://t.co/Xe38dQ2euv
🇨🇳 #YolTech Therapeutics, a clinical-stage biotech developing in vivo genome editing therapies, announced that China #NMPA has cleared the company’s #(IND application for YOLT-101, an in vivo base editing therapy targeting #PCSK9 for the treatment of heterozygous familial hypercholesterolemia (HeFH). #GeneTherapy #CGT #BaseEditing
This marks YOLT-101’s second regulatory clearance—following FDA approval in June 2025—and establishes it as the first in vivo base editing therapy for cardiovascular disease to advance into clinical trials in both the U.S. and China.
YOLT-101 is an investigational in vivo base editing therapy designed to be a single-dose treatment to durably reduce blood LDL-C.
It is based on YolTech’s proprietary adenine base editor, YolBE—specifically hpABE5—which comprises nCas and a novel deaminase evolved from Hafnia paralvei.
For delivery, YOLT-101 utilizes YolTech’s innovative lipid nanoparticle (LNP) delivery system. Unlike traditional CRISPR/Cas9 systems that rely on DNA double-strand breaks (DSBs), hpABE5 enables precise A•T to G•C base conversion without introducing DSBs, thereby significantly reducing risks of chromosomal abnormalities and off-target effects.
YOLT-101 is being evaluated in ongoing investigator-initiated trial (IIT), where it has demonstrated a favorable safety profile and robust LDL-C–lowering effects. https://t.co/ikFWRXbsyy
🇨🇳 #YolTech Therapeutics, a clinical-stage biotec developing in vivo genome editing therapies, announced that the #FDA has cleared the #IND application for YOLT-101, an in vivo base editing therapy targeting #PCSK9 for the treatment of heterozygous familial hypercholesterolemia (HeFH). https://t.co/ybZIUv20Ov
About YolTech: https://t.co/h4eLV58AdA
YOLT-101, already licensed to another 🇨🇳 #Salubris, total price ￥1b 🧵 https://t.co/lpaQEfBE2s
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