RARE Daily

Nanite and SalioGen Collaborate to Develop Non-Viral, Lung-Targeting Nanoparticle for Cystic Fibrosis

May 30, 2024

Rare Daily Staff

Nanite and SalioGen Therapeutics entered a research collaboration to develop a platform that will deliver a cystic fibrosis genetic medicine directly to the lungs.

The collaboration will utilize Nanite’s SAYER delivery platform to develop a lung-targeting polymer nanoparticle designed to integrate the cystic fibrosis transmembrane conductance regulator (CFTR) gene directly into lung cells using SalioGen’s novel Gene Coding technology. Mutations in the CFTR gene cause cystic fibrosis (CF), a chronic and life-threatening condition that progressively damages the lungs, digestive system, and other organs.

“By combining the innovative approaches of both companies, we hope to create a non-viral genetic medicine delivered directly to the lungs and provide the first permanent therapy for people living with CF, regardless of mutation,” said Jason Cole, CEO of SalioGen Therapeutics.

Nanite’s proprietary SAYER platform combines AI-driven polymer design with multiplexed in vivo screening to develop best-in-class polymer nanoparticles (PNPs) for tissue-specific delivery. SAYER has demonstrated strong efficiency and high specificity for targeting the lungs, potentially overcoming the limitations of viral vectors and lipid nanoparticles.

SalioGen’s Gene Coding technology uses a bioengineered transposase enzyme to deliver an optimized construct containing the entire gene of interest, regardless of size, to a precise location in the genome of target cells. In vitro experiments successfully demonstrated the ability to integrate the full-length CFTR gene into human lung cells.

Under the terms of the agreement, Nanite will design a resorbable polymer delivery vehicle for SalioGen’s Gene Coding technology.

“Nanite’s relationship with SalioGen is consequential in discovering safe polymeric nanoparticles for a transformative treatment for CF,” said Sean Kevlahan, Nanite co-founder and CEO. “In addition to ongoing Nanite research supported by the Cystic Fibrosis Foundation’s Path to a Cure, the SalioGen collaboration underscores the power of the SAYER platform’s capacity to design fit-for-purpose delivery vehicles.”

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