CAMP4 Therapeutics and Atsena Therapeutics secure crucial FDA nod

CAMP4 Therapeutics has received the FDA Orphan Drug Designation (ODD) for its clinical-stage therapeutic, CMP-CPS-001, which treats urea cycle disorders (UCDs). The therapy, which employs regulatory RNA-targeting technology, aims to restore healthy protein levels by upregulating the CPS1 enzyme, which is essential in the urea cycle.

CMP-CPS-001 addresses a major unmet need in UCDs, which currently lack disease-modifying treatments. Preclinical studies show that CMP-CPS-001 can help normalize ammonia levels by increasing CPS1 expression, potentially lowering severe neurological and health risks in UCD patients.

On the contrary, the FDA granted Orphan Drug Designation (ODD) to Atsena Therapeutics ATSN-201. ATSN-201, a gene therapy candidate for X-linked Retinoschisis (XLRS). This follows its previous designation as a Rare Pediatric Disease. ATSN-201 employs Atsena’s AAV.SPR capsid, which allows gene expression in photoreceptors while minimizing the risk of foveal detachment.

XLRS affects about 30,000 males in the United States and the European Union, and there are currently no approved treatments. ATSN-201 aims to address this unmet need by upregulating the RS1 gene to reverse retinal damage, potentially preventing progressive vision loss.