YolTech Therapeutics Receives U.S. FDA Rare Pediatric Disease Designation for YOLT-203 in Treating Primary Hyperoxaluria Type 1

SHANGHAI, Sept. 4, 2024 /PRNewswire/ -- YolTech Therapeutics, a pioneering gene editing company focused on developing in vivo gene editing medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to YOLT-203 for the treatment of Primary Hyperoxaluria Type 1 (PH1).

Primary hyperoxaluria is a genetic disorder associated with hepatic oxalate overproduction, leading to increased renal oxalate excretion. PH1 as the most commonly diagnosed subtype, is a childhood-onset autosomal recessive disease form caused by AGXT gene mutations and absence or dysfunction of alanine-glyoxylate aminotransferase. Most patients ultimately progress to renal failure and require intensive hemodialysis as a bridge to a dual liver/kidney transplant to resolve the metabolic defect in the liver and replace the terminally damaged kidneys.

YOLT-203 is the world's first in vivo gene editing therapy specifically developed for PH1, utilizing YolTech's proprietary YolCas12™ gene editing technology. This groundbreaking treatment not only targets and corrects the genetic mutations responsible for the disease, aiming to reduce the harmful build-up of oxalate in the kidneys and other organs, but also marks the first-ever in vivo gene editing clinical trial globally for a pediatric rare disease. This innovation has the potential to significantly mitigate severe kidney damage and systemic complications, especially in children.

The investigator-initiated clinical study of YOLT-203 commenced on August 5th, 2024, with the first adult subject dosed, followed by the first pediatric patient on August 20th, 2024. Early data from preclinical models have shown promising reductions in oxalate levels.

The RPDD program is designed to encourage the development of new drugs and biologics for the treatment of rare diseases that primarily affect children. Under this designation, YolTech is eligible to receive a priority review voucher (PRV) upon potential FDA approval of YOLT-203. This voucher can be used to expedite the review of another drug or sold to another company.

About YOLT-203

YOLT-203 is an innovative in vivo gene editing therapy designed using YolTech's proprietary YolCas12™ system, a novel CRISPR/Cas tool developed through the High-Throughput Evolution Platform (HEPDONE®). This therapy is encapsulated within lipid nanoparticles (LNPs) that facilitate targeted delivery to the liver, where it addresses the underlying genetic mutations responsible for primary hyperoxaluria type 1 (PH1). Upon intravenous administration, the LNPs are recognized and internalized by liver cells. Once inside, the YolCas12™ editor protein, guided by the corresponding guide RNA (gRNA), targets and corrects mutations in the AGXT gene, aiming to reduce harmful oxalate levels in the blood, thereby offering a potential one-time, curative treatment for PH1.

About YolTech

YolTech Therapeutics is a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines. Our approach combines innovative gene editing technologies with an advanced lipid nanoparticle (LNP) delivery system, creating a versatile platform designed to address a wide range of serious diseases. Central to our mission is the development of internal capabilities, including end-to-end manufacturing, to ensure the highest standards of quality and scalability. Our lead candidate, targeting ATTR, marks a significant milestone as China's first LNP-mediated in vivo gene editing therapy to enter clinical development. With promising early clinical outcomes, YolTech is also advancing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1). As a company dedicated to transforming the treatment landscape, YolTech continues to push the boundaries of what is possible in gene editing.

For more information, please visit: www.yoltx.com or follow YolTech on LinkedIn https://www.linkedin.com/company/yoltech-therapeutics/

Contacts

Colin LIN
Business Development & Investor Relations
[email protected]
+86 180 2100 7750

SOURCE YolTech Therapeutics