YolTech Therapeutics, a clinical-stage in vivo gene editing company, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to YOLT-203 for the treatment of primary hyperoxaluria type 1 (PH1) in September. Earlier that month, YOLT-203 received Rare Pediatric Disease Designation (RPDD) from the FDA.  

After reviewing YolTech's submission, the FDA determined that the therapy is not only appropriate for primary hyperoxaluria type 1 (PH1), but also applicable to the broader category of primary hyperoxaluria (PH). As a result, the designations were granted for PH, reflecting the expanded scope of the therapy's potential impact. These dual designations underscore the transformative potential of YOLT-203 to address this rare and life-threatening genetic disorder.

The Rare Pediatric Disease Designation (RPDD) program encourages the development of new drugs and biologics for rare pediatric conditions. This designation makes YolTech eligible to receive a priority review voucher (PRV) upon potential FDA approval of YOLT-203. Similarly, the Orphan Drug Designation (ODD) program provides benefits including tax credits, exemption from certain FDA fees, and seven years of market exclusivity upon regulatory approval.

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.

 

Source: prnewswire.com

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